Lipid-based formulations and lipid nanoparticles are administration strategies of increasing importance in the pharmaceutical world. On the way to a successful drug product registration, there are numerous challenges to be overcome – from formulation development to meeting regulatory requirements and submission. In particular, care must be taken to choose appropriate lipids with respect to their type, source and quality, as these factors greatly affect the performance of the final formulation and also play a role in financial considerations.
In this webinar, you will:
● Explore critical aspects relating to synthetic lipids as raw materials for lipidic formulations
● Learn strategies to minimize risk when choosing lipidic raw materials for drug product development
● Gain insights into tailored manufacturing and lipids with optimized properties such as conjugation of targeting moieties
From preclinical to commercial: The evolution of ADC manufacturing expertise ...MilliporeSigma
ADCs have evolved since the initial approval of MYLOTARG® in 2000, with respect to both conjugation, linker and toxin chemistries as well as processing. With the current availability of four commercially approved drugs and approximately 80 programs in various clinical trials, there has been a significant interest in simplifying the complex supply chain to make manufacturing efficient. With approximately 80% of the programs outsourced to Contract Development and Manufacturing Organizations (CDMOs), a transparent and integrated supply chain is critical for the success of ADC projects.
MilliporeSigma has been involved with the development and manufacturing of ADCs since 2008. This webinar will discuss how chemistry and manufacturing have evolved over the past 10 years globally and how MilliporeSigma, as a CDMO has adapted to these changes to meet customers needs.
In this webinar, you will learn:
● How ADC manufacturing has evolved over the past decade
● The challenges that this evolution implies
● Why an integrated supply chain has become increasingly important for the success of ADC projects
Hot melt extrusion with PVA – solubility enhancement, supersaturation perform...Merck Life Sciences
Hot melt extrusion has successfully emerged as an innovative manufacturing technology in pharmaceutical industry for the creation of amorphous solid dispersions (ASDs).
In this webinar you will learn about the potential of hot melt extrusion to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA) as a matrix polymer. We will provide an overview about different types of solid dispersions and their evolution in the pharmaceutical field. A brief introduction in hot melt extrusion processing will be given as well as actual formulation trends. You will get insights in potential down-stream options to create your final dosage form and you will gain ideas on how to speed up your formulation development.
A detailed background of PVA will be provided including its physical properties as well as its regulatory status. PVA is more than a polymer. Due to its amphiphilic structure it has the potential to improve the supersaturation of low soluble APIs and to prevent precipitation after release. This highlights the versatility of PVA as an advanced polymer for HME applications and we will guide you through our latest research activities so that you can leverage our knowledge to improve your formulations.
This webinar includes:
- The current status and further potential of HME in pharmaceutical industry
- Advantages of PVA in the field of ASDs: Solubility improvement, impact on supersaturation potential, stability data generated on sample formulations & downstream options
- Deep dive into latest research activities: Permeation studies with Caco-2 cell membranes, pH shift studies to investigate supersaturation potential, ongoing research activities to get to know a more detailed understanding of matrix systems and their intermolecular interactions
In this webinar, you will learn:
- which potential hot melt extrusion has, to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA)
- why PVA is more than just a polymer
- how to create your final dosage form and speed up your formulation development
Excipients selection for high risk formulations Smita RajputMerck Life Sciences
Are you choosing the right excipients for your high risk application? Find out how to select the right excipients and enable your process optimization to improve the total cost of ownership.
In this webinar, you will learn:
• Selection of right excipients for high risk formulation is very critical step
• Low Endotoxin and low bioburden limits are important aspect while selecting raw materials
• Strong regulatory support is crucial for high risk formulation
Excipients selection for high risk formulations like parenteral and ophthalmic applications is very challenging. Excipients should be inert with high purity for such dosage forms because trace amounts of impurities present in excipients can interact with active pharmaceutical ingredient (API) which results in instability of the formulation. This presentation discusses how to select the right excipients for high-risk applications and gives guidance for process optimization by choosing the best combination of filters and excipients to improve the total cost of ownership.
The Future of Bioprocessing – What you need to know.MilliporeSigma
One of the hottest topics in the biopharmaceutical industry today is “continuous bioprocessing”. Buzzwords such as “process intensification”, “next generation bioprocessing”, “process optimization”, and “integrated, connected manufacturing” are prevalent in industry conference programs and biopharma trade publications. This is no surprise considering that by 2025, it is expected that 20 percent of revenue from molecules that are still in the pipeline today will come from drugs manufactured with next generation technologies. It is estimated that roughly 35 percent of today’s commercial molecules will utilize process intensification methods within the next 5-10 years.
But what actually is next generation bioprocessing? How are industry players defining next generation bioprocessing and how will they pursue and successfully implement this approach? And is continuous bioprocessing always the ultimate end goal drug manufacturers around the globe are, or should be, striving for?
In this dedicated supplement, experts from both biologics manufacturers and from MilliporeSigma provide their insights and perspectives on the on-going paradigm shift towards next generation bioprocessing occurring in today’s and tomorrow’s biopharma landscape.
Process Impurities: Don’t Let PEI or HCP Derail Your BioTherapyMerck Life Sciences
View our webinar here: https://bit.ly/2lKNdWX
Many different impurities are present in or generated during biotherapy manufacturing. This webinar will address how process contaminates can arise from raw input materials, occur as residual processing agents, or form as reaction by-products. We will review strategies within product characterization to de-risk the manufacturing process, including the use of routine and high complexity assays; and the recommended testing to meet regulatory requirements for clinical submission. Learn methods to avoid costly pitfalls and implement procedures to expedite product quality decisions at critical junctures in your development plan. We will discuss two types of therapies:
Cell & Gene Therapies
Polyethylenimine (PEI) is a transfection agent used in nearly all cell and gene therapy products. We will review the regulations and the liquid chromatography with charged aerosol detection (LC-CAD) methodology to demonstrate PEI removal during the production process.
Monoclonal Antibodies (mAb) and Cell & Gene Therapies
During mAb manufacturing and inherent to Cell & Gene Therapies, a significant proportion of process impurities arise from the host cell used to express the drug. Host cell protein (HCP) impurities, present at PPM-levels, are a major immunogenicity risk because they can elicit an unpredictable immune response in patients. We will review why their complex and diverse nature makes them challenging to monitor, and theho best practices, specifically HCP identification by mass spectrometry, for detection.
Learning points:
1. Accurate detection and characterization of residual PEI in cell and gene therapy products
2. Effective detection and characterization of residual host cell proteins (HCP) in mAbs
3. Available technology and assays for quantifying process impurities
4. Current regulatory requirements for detecting, quantifying, and removing process impurities during biotherapy manufacturing
Streamlining Biopharmaceutical Cell Line Development - Reducing risk and decr...Merck Life Sciences
CHO cells with their unique characteristics, represent the major expression system within the biopharmaceutical industry. However, one of the major challenges in cell line development is to identify those rare, high-producing clones in a huge population of non-expressing or low-expressing cell lines. This leads to laborious and time consuming cell line development processes. This webinar will educate the audience about challenges faced with traditional expression systems and how the CHO cell line with the glutamine synthethase knock-out via Zinc Finger Nucleases provides benefits for fast and efficient cell line development as well as stable and high titer expression. We will explore additional cell line engineering targets that can be modified to engineer a cell line that mitigates risks and removes bottlenecks throughout the biopharmaceutical process.
In this webinar, you will learn:
• What are the benefits of using an optimized/engineered expression system?
• What can be done throughout the cell line development process to mitigate risks and remove bottlenecks?
• Applications of cell line engineering for further upstream biopharmaceutical enhancements.
Next Generation Bioprocessing adoption for mAbs – BioContinuum™ Platform Info...Merck Life Sciences
Learn more on the opportunities and hurdles of intensified, connected or continuous processing. 30 US and European biomanufacturers were interviewed to understand the likely future adoption of ‘Next Generation Bioprocessing’.
Discover the animated version! http://www.merckmillipore.com/INTL/en/20180329_155610?bd=1&tab=2
Visit the BioContinuum™ Platform webpage now! http://www.merckmillipore.com/biocontinuum
From preclinical to commercial: The evolution of ADC manufacturing expertise ...MilliporeSigma
ADCs have evolved since the initial approval of MYLOTARG® in 2000, with respect to both conjugation, linker and toxin chemistries as well as processing. With the current availability of four commercially approved drugs and approximately 80 programs in various clinical trials, there has been a significant interest in simplifying the complex supply chain to make manufacturing efficient. With approximately 80% of the programs outsourced to Contract Development and Manufacturing Organizations (CDMOs), a transparent and integrated supply chain is critical for the success of ADC projects.
MilliporeSigma has been involved with the development and manufacturing of ADCs since 2008. This webinar will discuss how chemistry and manufacturing have evolved over the past 10 years globally and how MilliporeSigma, as a CDMO has adapted to these changes to meet customers needs.
In this webinar, you will learn:
● How ADC manufacturing has evolved over the past decade
● The challenges that this evolution implies
● Why an integrated supply chain has become increasingly important for the success of ADC projects
Hot melt extrusion with PVA – solubility enhancement, supersaturation perform...Merck Life Sciences
Hot melt extrusion has successfully emerged as an innovative manufacturing technology in pharmaceutical industry for the creation of amorphous solid dispersions (ASDs).
In this webinar you will learn about the potential of hot melt extrusion to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA) as a matrix polymer. We will provide an overview about different types of solid dispersions and their evolution in the pharmaceutical field. A brief introduction in hot melt extrusion processing will be given as well as actual formulation trends. You will get insights in potential down-stream options to create your final dosage form and you will gain ideas on how to speed up your formulation development.
A detailed background of PVA will be provided including its physical properties as well as its regulatory status. PVA is more than a polymer. Due to its amphiphilic structure it has the potential to improve the supersaturation of low soluble APIs and to prevent precipitation after release. This highlights the versatility of PVA as an advanced polymer for HME applications and we will guide you through our latest research activities so that you can leverage our knowledge to improve your formulations.
This webinar includes:
- The current status and further potential of HME in pharmaceutical industry
- Advantages of PVA in the field of ASDs: Solubility improvement, impact on supersaturation potential, stability data generated on sample formulations & downstream options
- Deep dive into latest research activities: Permeation studies with Caco-2 cell membranes, pH shift studies to investigate supersaturation potential, ongoing research activities to get to know a more detailed understanding of matrix systems and their intermolecular interactions
In this webinar, you will learn:
- which potential hot melt extrusion has, to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA)
- why PVA is more than just a polymer
- how to create your final dosage form and speed up your formulation development
Excipients selection for high risk formulations Smita RajputMerck Life Sciences
Are you choosing the right excipients for your high risk application? Find out how to select the right excipients and enable your process optimization to improve the total cost of ownership.
In this webinar, you will learn:
• Selection of right excipients for high risk formulation is very critical step
• Low Endotoxin and low bioburden limits are important aspect while selecting raw materials
• Strong regulatory support is crucial for high risk formulation
Excipients selection for high risk formulations like parenteral and ophthalmic applications is very challenging. Excipients should be inert with high purity for such dosage forms because trace amounts of impurities present in excipients can interact with active pharmaceutical ingredient (API) which results in instability of the formulation. This presentation discusses how to select the right excipients for high-risk applications and gives guidance for process optimization by choosing the best combination of filters and excipients to improve the total cost of ownership.
The Future of Bioprocessing – What you need to know.MilliporeSigma
One of the hottest topics in the biopharmaceutical industry today is “continuous bioprocessing”. Buzzwords such as “process intensification”, “next generation bioprocessing”, “process optimization”, and “integrated, connected manufacturing” are prevalent in industry conference programs and biopharma trade publications. This is no surprise considering that by 2025, it is expected that 20 percent of revenue from molecules that are still in the pipeline today will come from drugs manufactured with next generation technologies. It is estimated that roughly 35 percent of today’s commercial molecules will utilize process intensification methods within the next 5-10 years.
But what actually is next generation bioprocessing? How are industry players defining next generation bioprocessing and how will they pursue and successfully implement this approach? And is continuous bioprocessing always the ultimate end goal drug manufacturers around the globe are, or should be, striving for?
In this dedicated supplement, experts from both biologics manufacturers and from MilliporeSigma provide their insights and perspectives on the on-going paradigm shift towards next generation bioprocessing occurring in today’s and tomorrow’s biopharma landscape.
Process Impurities: Don’t Let PEI or HCP Derail Your BioTherapyMerck Life Sciences
View our webinar here: https://bit.ly/2lKNdWX
Many different impurities are present in or generated during biotherapy manufacturing. This webinar will address how process contaminates can arise from raw input materials, occur as residual processing agents, or form as reaction by-products. We will review strategies within product characterization to de-risk the manufacturing process, including the use of routine and high complexity assays; and the recommended testing to meet regulatory requirements for clinical submission. Learn methods to avoid costly pitfalls and implement procedures to expedite product quality decisions at critical junctures in your development plan. We will discuss two types of therapies:
Cell & Gene Therapies
Polyethylenimine (PEI) is a transfection agent used in nearly all cell and gene therapy products. We will review the regulations and the liquid chromatography with charged aerosol detection (LC-CAD) methodology to demonstrate PEI removal during the production process.
Monoclonal Antibodies (mAb) and Cell & Gene Therapies
During mAb manufacturing and inherent to Cell & Gene Therapies, a significant proportion of process impurities arise from the host cell used to express the drug. Host cell protein (HCP) impurities, present at PPM-levels, are a major immunogenicity risk because they can elicit an unpredictable immune response in patients. We will review why their complex and diverse nature makes them challenging to monitor, and theho best practices, specifically HCP identification by mass spectrometry, for detection.
Learning points:
1. Accurate detection and characterization of residual PEI in cell and gene therapy products
2. Effective detection and characterization of residual host cell proteins (HCP) in mAbs
3. Available technology and assays for quantifying process impurities
4. Current regulatory requirements for detecting, quantifying, and removing process impurities during biotherapy manufacturing
Streamlining Biopharmaceutical Cell Line Development - Reducing risk and decr...Merck Life Sciences
CHO cells with their unique characteristics, represent the major expression system within the biopharmaceutical industry. However, one of the major challenges in cell line development is to identify those rare, high-producing clones in a huge population of non-expressing or low-expressing cell lines. This leads to laborious and time consuming cell line development processes. This webinar will educate the audience about challenges faced with traditional expression systems and how the CHO cell line with the glutamine synthethase knock-out via Zinc Finger Nucleases provides benefits for fast and efficient cell line development as well as stable and high titer expression. We will explore additional cell line engineering targets that can be modified to engineer a cell line that mitigates risks and removes bottlenecks throughout the biopharmaceutical process.
In this webinar, you will learn:
• What are the benefits of using an optimized/engineered expression system?
• What can be done throughout the cell line development process to mitigate risks and remove bottlenecks?
• Applications of cell line engineering for further upstream biopharmaceutical enhancements.
Next Generation Bioprocessing adoption for mAbs – BioContinuum™ Platform Info...Merck Life Sciences
Learn more on the opportunities and hurdles of intensified, connected or continuous processing. 30 US and European biomanufacturers were interviewed to understand the likely future adoption of ‘Next Generation Bioprocessing’.
Discover the animated version! http://www.merckmillipore.com/INTL/en/20180329_155610?bd=1&tab=2
Visit the BioContinuum™ Platform webpage now! http://www.merckmillipore.com/biocontinuum
Key to Successful Formulation Development for Lipid Based RNA Delivery and Va...MilliporeSigma
In this webinar, we will discuss:
• The application of RNA therapeutics and the different drug delivery routes used in the clinic.
• Design principles for developing lipids-based RNA formulations.
• Critical parameters to consider for cost effective development and consistent performance of RNA therapeutics and vaccines.
RNA therapeutics are changing the way we address diseases. Applications range from gene therapy, oncology, to vaccines for infectious diseases such as COVID-19.
The performance of RNA therapeutics critically depends on its formulation. Key decisions have to be made early on in the drug development process; choosing the appropriate drug delivery method and novel excipients. Raw material source and judicious choice of chemistry, ultimately determine the quality of novel lipid excipients which, in turn, has a big impact on the performance, reproducibility, costs, and regulatory approval timelines. This webinar will propose solutions to maximize the probability of success while formulating RNA therapeutics and vaccines.
Participate in the interactive webinar now: https://bit.ly/2xXMZlm
Explore our webinar library: www.emdmillipore.com/webinars
Promises and Challenges of Manufacturing and Testing Viral Producer Cell LinesMerck Life Sciences
To date, manufacturing of lentivirus (LV) vectors for gene therapy commonly relies on transient transfection of adherent HEK293 cells. This method is costly, time-consuming, difficult to scale-up and poorly reproducible, rendering large-scale applicability to fulfill increasing demand of LV in clinical pipelines cumbersome. The use of suspension-adapted transient producer cell lines for LV production has overcome some of these challenges. Furthermore, successful creation of stable producer cell lines would allow creation of master and working cell banks easily amenable to commercial production. The ideal producer cell lines should demonstrate stability in growth and gene expression, and be easily adaptable to chemically defined culture conditions and optimized for high-titer virus production. The availability of more robust producer cell lines thus represents an important scalable first step towards manufacturing processes that are conducive to large-scale production. Ultimately, these producer cell lines must be screened to satisfy various biosafety and regulatory implications.
In this webinar, you will learn:
• Process development for transient and stable producer cell lines
• Screening of cellular gene targets via CRISPR to improve LV production from producer cell lines
• cGMP and Regulatory readiness: Cell line characterization and release testing through BioReliance® global service offering
The Butterfly Effect: How to see the impact of small changes to your ADCMilliporeSigma
Watch this webinar here: https://bit.ly/31PRr2z
Small changes to the design of antibody-drug conjugate can have a dramatic effect on its structure and biological activity. Effective product characterization is essential to understanding the impact of these changes. Here we discuss methods to provide insight at critical junctures in ADC development.
There are many different design considerations facing developers of antibody-drug conjugates: these variables must be tuned to achieve the right balance of efficacy and safety. For example, the choice of linker can influence an ADC's potency, toxicity and pharmacokinetics.
In this webinar we explore the influence of various PEG linkers on the structure of a model ADC by identifying specific sites of conjugation by peptide mapping, investigating changes in higher order structure by HDX mass spectrometry, and examining the impact on binding by SPR spectroscopy.
We demonstrate that employing a range of orthogonal methods is critical to understanding the structure-function relationships of an ADC.
In this webinar, you will learn about:
• How the choice of linker can influence an ADC's activity
• Information-rich methods to probe ADC structure and function
• Effective strategies for thorough characterization of ADC products
Employing Innovative Platform Manufacturing and Biosafety Testing for your Ge...MilliporeSigma
Watch the webinar here: https://event.on24.com/wcc/r/2003970/F5AFA4FE6C60AD00635D4D15BADB5D8E?partnerref=slideshare
As gene therapies and gene-modified cell therapies show increasing promise, the need for innovative and proficient viral vector manufacturing continues to grow. Concurrently, increased regulatory guidance governing the manufacturing and testing of viral vectors adds complexity and increases the timelines to successfully produce high-quality virus ready for clinical use.
This webinar will address how the implementation of both manufacturing templates and platform characterization and safety assays can increase the likelihood of success in process validation and reduce risk in the timeline to commercialization for your gene therapy product. Using adeno-associated virus (AAV) as a case study, we will demonstrate how our validated, templated process for production can reduce the need for qualification inherent in niche manufacturing workflows and anticipate forthcoming needs for process performance qualification. This webinar will also highlight benefits from a new, platform assay offering for characterization and safety testing of AAV. Because these assays are pre-qualified, they reduce the variability inherent in assay validation and subsequently the time needed to establish readiness for regulatory compliance.
While these developments increase the standardization across the manufacturing and testing workflows, they remain flexible to clients' needs and are created to be scalable and as future-proof as possible, allowing for adaptability as the regulatory landscape of gene therapies evolves.
In this webinar, you will learn:
● The unit operations in AAV manufacturing that are ideal for templating
● How the manufacturing workflow can be targeted to reduce variability in testing and improve readiness for commercial production
● How platform assays can ease the burden of assay qualification and improve overall commercialization timelines
USP <665> draft standard : A rational risk-based approach to characterization...MilliporeSigma
This webinar will cover risk-based characterization of filters and single-use systems used in biopharmaceutical manufacturing according to USP <665>.
Novel innovative biomanufacturing systems such as single-use assemblies often comprise of polymeric materials. There is a lack of standards for characterization of these polymeric systems. USP <665> draft standard is the first standard in development addressing this topic. This chapter recommends risk assessment with respect to patient safety, risk level assignment and risk level appropriate characterization of components.
In this webinar we will discuss:
● Risk assessment to assign a risk level
● Risk level based testing
● Our approach for compliance
● Emprove™ Dossiers for Filters and Single-use systems
Polymer based drug delivery systems for parenteral controlled release: from s...Merck Life Sciences
This webinar, presented by two world-class experts in polymer based parenteral controlled-release drug delivery technologies, will provide insights into formulation technologies from small molecules up to biologics.
There is an increasing interest in long-acting injectables as drugs administered through injection help to increase patient compliance due to reduced frequency of administration while providing the same therapeutic efficiency. Depending from the nature of the drug, the optimum polymer technology is to be selected.
Prof. Dr. Mäder focus on how to select the appropriate PLA/PLGA polymer for small drug molecule applications. He will provide an overview of drug delivery systems, most important formulation techniques and appropriate characterization methods along with application examples.
Alternative polymer systems are required for peptide and protein controlled-release formulations. Dr. Rob Steendam introduces InnoCore´s SynBioSys® biodegradable polymer system demonstrating excellent safety, control over release kinetics and effective preservation of structural integrity and bioactivity of biologics. InnoCore Pharmaceuticals and SynBioSys® multi-block polymer introduction, challenges in development of controlled-release formulations of biological therapeutics including various examples and development and cGMP manufacturing at InnoCore are key elements of his presentation.
In this webinar, you will learn:
• drug delivery systems
• most important formulation techniques
• appropriate characterization methods along with application examples
Webinar: Benefits of Monodisperse Activated PEGs in ADC DevelopmentMilliporeSigma
Watch the webinar here: http://bit.ly/PEGsWebinar
As the field of antibody-drug conjugation chemistry has advanced, the use of linkers to impart "drugability" has also been growing. Recent literature shows a variety of linear and branched monodisperse PEGs being called on to modify the PK/PD profile of some of the most active but lipophilic payloads. In this webinar, we will survey the types of PEGs that are used in ADCs and give examples of successful implementation to change the biophysical properties of the construct. In addition, we will focus on PEGs and why activated PEGs are widely used to improve the pharmacokinetics of drugs (such as pegylated proteins, peptides etc.). Raw materials used in this field may have a variety of polydispersity. However, when it comes to the use of activated PEGs as linkers for ADCs, the requirements are significantly higher. Therefore, the choice and control of the PEG linker is crucial for the successful development and accelerated time to market for your ADC.
This webinar addresses critical success factors such as:
• Survey of current PEG enhanced ADCs
• Why PEGs are used designing ADCs
• Critical parameters for aPEGs used as linkers
• Requirements in terms of analytical capabilities
Upcoming USP 665 - Level of Characterization of Single-Use Systems Today and ...MilliporeSigma
Register for the interactive, on-demand webinar now: https://bit.ly/USP665
Single-use plastic systems are being utilized more frequently especially for COVID-19 vaccine manufacturing. However, there are issues regarding standardization of quality information that limits implementation efficiencies. One of the challenges is the evaluation of leachables derived from a variety of different plastic components in a timely manner.
Since the USP <665> highlights a risk assessment approach with no typical pass/fail limit, approaches to decision-making based on the extractables data package will be reviewed. In addition, we will highlight legacy testing requirements which may not be necessary once USP <665> is implemented.
In this webinar, we will discuss:
- Regulatory expectations of extractables and leachables assessment today and tomorrow
- The right criteria that need to be assessed to select the type and quality of plastic materials for use in biopharmaceutical manufacturing
In this webinar, you will learn:
Sources of endotoxin contamination
Contamination control strategy
Endotoxin removal strategies
Detailed description:
Endotoxin, a lipopolysaccharide (LPS), is a type of pyrogen and is a component of the exterior cell wall of Gram-negative bacteria. To ensure safety on patient’s endotoxin content in the drug should always be controlled. In a biological processing it may emanate from facility, utility, raw materials, process, and personnel. In this webinar we discuss the regulatory norms, strategies for prevention & removal of endotoxin to ensure that the final drug product is safe.
The Role of BPOG Extractables Data in the Effective Adoption of Single-Use Sy...Merck Life Sciences
The successful adoption of single-use technologies in a biopharmaceutical process largely relies on confidently selecting the right components for use in the fluid path of a product, within a specific process. An important step in choosing such components requires generating an extractables profile, which can be done by carefully selecting the solvent streams and extraction conditions to model the product and process steps complemented with the right analytical strategy.
In this webinar, you will learn:
● An approach to adopt the BioPhorum Operations Group (BPOG) extractables protocol as a baseline testing strategy.
● How to apply extractables data to a specific process followed by a systematic, risk-based safety assessment approach used for comparing known safety concern thresholds.
● The important stages in the risk assessment process as demonstrated by case studies from typical drug manufacturing processes where single-use components were used.
Process equipment characterization – how standardized extractables data suppo...Merck Life Sciences
View the recording here: https://bit.ly/35KIwBb
Biopharmaceutical Industry recently increased adoption of Single-Use systems and components in manufacturing process operations. Drug manufacturers are responsible for the characterization of SU components and systems used for the production to ensure patient safety. SUS Suppliers are encouraged by BPOG and BPSA to provide comprehensive extractables data package to support drug manufacturer’s E&L assessments.
This webinar will give an overview of the E&L evaluation workflow and practical study approaches from both supplier and end-user perspective, in accordance with the latest industry’s standards and upcoming USP <665> requirements. Case studies will be presented on how the data from suppliers are used to mitigate risk associated to SU materials, highlighting the key role of collaboration between the supplier and the drug manufacturer.
Challenges using Multiple Single-use Systems: Functionality versus Extractabl...Merck Life Sciences
As single-use technologies continue to expand in pharmaceutical manufacturing processes, the risk assessment for extractables and leachables becomes increasingly complex. Join this webinar to obtain guidance on how to perform risk evaluation on a process with multiple single-use components.
A Single-Use System (SUS) is typically designed for a specific process step. In many cases, single-use components are chosen based on their functionality. The challenge arises when there are multiple processing steps-- as the different applications and product matrices are evaluated, the complexity of the risk assessment increases. Complexity includes component evaluation, process conditions, and model solvents streams which ultimately relates to the patient safety risk.
This webinar will evaluate the different single-use components with respect to compatibility and extractables and leachables. A case study will be used to demonstrate the complexity and potential concerns when performing a risk evaluation on the manufacturing process.
In this webinar, you will learn:
- Risk assessment of extractables
- Single-use component evaluation
- Complexity when evaluating multiple assemblies
The Emprove® Program: Introduction of New Portfolio AdditionsMerck Life Sciences
Watch the presentation of this webinar here: https://bit.ly/3wT5Irt
The Emprove® Program is constantly expanding and updating. Find out about the recently launched Emprove® CCM category for our cell culture media portfolio, the new Emprove® API Information Packages, and the latest updates on available TUPPs for our Emprove® Chemicals portfolio.
This virtual user group will introduce the two latest additions to the Emprove® Program:
1. Our latest category Emprove® CCM for our cell culture media with wave 1 focus on our Cellvento® CHO cell culture media portfolio, looking also into the content of our three Emprove® Dossiers.
2. The new Emprove® API Information Packages that are currently created for our Emprove® APIs, looking into the content and showing how to access.
Additionally, we'll give an overview of latest Technically Unavoidable Particle Profiles (TUPPs) for our Emprove® Chemicals.
In this virtual user group, you will learn:
• Emprove® CCM and its documentation for our cell culture media
• Content and availability of the new Emprove® API Information Package
• Update on available TUPPs for our Emprove® Chemicals
Webinar: Post Approval Changes in Biologics Manufacturing - A Practical Asses...MilliporeSigma
Participate in the interactive webinar: http://bit.ly/PACWebinar
Post-approval changes for biologics manufacturing processes are complicated and challenging with the current global diverse regulatory environment. Here, we will present approaches to make these changes more efficient using a risk-based approach.
Explore our webinar library: www.emdmillipore.com/webinars
Excipients selection for high risk formulations Smita RajputMilliporeSigma
Are you choosing the right excipients for your high risk application? Find out how to select the right excipients and enable your process optimization to improve the total cost of ownership.
In this webinar, you will learn:
• Selection of right excipients for high risk formulation is very critical step
• Low Endotoxin and low bioburden limits are important aspect while selecting raw materials
• Strong regulatory support is crucial for high risk formulation
Excipients selection for high risk formulations like parenteral and ophthalmic applications is very challenging. Excipients should be inert with high purity for such dosage forms because trace amounts of impurities present in excipients can interact with active pharmaceutical ingredient (API) which results in instability of the formulation. This presentation discusses how to select the right excipients for high-risk applications and gives guidance for process optimization by choosing the best combination of filters and excipients to improve the total cost of ownership.
Key to Successful Formulation Development for Lipid Based RNA Delivery and Va...Merck Life Sciences
In this webinar, we will discuss:
• The application of RNA therapeutics and the different drug delivery routes used in the clinic.
• Design principles for developing lipids-based RNA formulations.
• Critical parameters to consider for cost effective development and consistent performance of RNA therapeutics and vaccines.
RNA therapeutics are changing the way we address diseases. Applications range from gene therapy, oncology, to vaccines for infectious diseases such as COVID-19.
The performance of RNA therapeutics critically depends on its formulation. Key decisions have to be made early on in the drug development process; choosing the appropriate drug delivery method and novel excipients. Raw material source and judicious choice of chemistry, ultimately determine the quality of novel lipid excipients which, in turn, has a big impact on the performance, reproducibility, costs, and regulatory approval timelines. This webinar will propose solutions to maximize the probability of success while formulating RNA therapeutics and vaccines.
Participate in the interactive webinar now: https://bit.ly/2xXMZlm
Explore our webinar library: www.merckmillipore.com/webinars
Liposomal Delivery Systems in Cancer Therapy - Creative BiolabsCreative-Biolabs
The lipid-based drug delivery system is a newly developed drug carrier that can be applied for various cancer-targeted treatments with many superiorities. This video briefly introduces various types of liposomes, the principles of liposomal drug delivery systems for cancer therapy, and liposome development services and products provided by Creative Biolabs.
Key to Successful Formulation Development for Lipid Based RNA Delivery and Va...MilliporeSigma
In this webinar, we will discuss:
• The application of RNA therapeutics and the different drug delivery routes used in the clinic.
• Design principles for developing lipids-based RNA formulations.
• Critical parameters to consider for cost effective development and consistent performance of RNA therapeutics and vaccines.
RNA therapeutics are changing the way we address diseases. Applications range from gene therapy, oncology, to vaccines for infectious diseases such as COVID-19.
The performance of RNA therapeutics critically depends on its formulation. Key decisions have to be made early on in the drug development process; choosing the appropriate drug delivery method and novel excipients. Raw material source and judicious choice of chemistry, ultimately determine the quality of novel lipid excipients which, in turn, has a big impact on the performance, reproducibility, costs, and regulatory approval timelines. This webinar will propose solutions to maximize the probability of success while formulating RNA therapeutics and vaccines.
Participate in the interactive webinar now: https://bit.ly/2xXMZlm
Explore our webinar library: www.emdmillipore.com/webinars
Promises and Challenges of Manufacturing and Testing Viral Producer Cell LinesMerck Life Sciences
To date, manufacturing of lentivirus (LV) vectors for gene therapy commonly relies on transient transfection of adherent HEK293 cells. This method is costly, time-consuming, difficult to scale-up and poorly reproducible, rendering large-scale applicability to fulfill increasing demand of LV in clinical pipelines cumbersome. The use of suspension-adapted transient producer cell lines for LV production has overcome some of these challenges. Furthermore, successful creation of stable producer cell lines would allow creation of master and working cell banks easily amenable to commercial production. The ideal producer cell lines should demonstrate stability in growth and gene expression, and be easily adaptable to chemically defined culture conditions and optimized for high-titer virus production. The availability of more robust producer cell lines thus represents an important scalable first step towards manufacturing processes that are conducive to large-scale production. Ultimately, these producer cell lines must be screened to satisfy various biosafety and regulatory implications.
In this webinar, you will learn:
• Process development for transient and stable producer cell lines
• Screening of cellular gene targets via CRISPR to improve LV production from producer cell lines
• cGMP and Regulatory readiness: Cell line characterization and release testing through BioReliance® global service offering
The Butterfly Effect: How to see the impact of small changes to your ADCMilliporeSigma
Watch this webinar here: https://bit.ly/31PRr2z
Small changes to the design of antibody-drug conjugate can have a dramatic effect on its structure and biological activity. Effective product characterization is essential to understanding the impact of these changes. Here we discuss methods to provide insight at critical junctures in ADC development.
There are many different design considerations facing developers of antibody-drug conjugates: these variables must be tuned to achieve the right balance of efficacy and safety. For example, the choice of linker can influence an ADC's potency, toxicity and pharmacokinetics.
In this webinar we explore the influence of various PEG linkers on the structure of a model ADC by identifying specific sites of conjugation by peptide mapping, investigating changes in higher order structure by HDX mass spectrometry, and examining the impact on binding by SPR spectroscopy.
We demonstrate that employing a range of orthogonal methods is critical to understanding the structure-function relationships of an ADC.
In this webinar, you will learn about:
• How the choice of linker can influence an ADC's activity
• Information-rich methods to probe ADC structure and function
• Effective strategies for thorough characterization of ADC products
Employing Innovative Platform Manufacturing and Biosafety Testing for your Ge...MilliporeSigma
Watch the webinar here: https://event.on24.com/wcc/r/2003970/F5AFA4FE6C60AD00635D4D15BADB5D8E?partnerref=slideshare
As gene therapies and gene-modified cell therapies show increasing promise, the need for innovative and proficient viral vector manufacturing continues to grow. Concurrently, increased regulatory guidance governing the manufacturing and testing of viral vectors adds complexity and increases the timelines to successfully produce high-quality virus ready for clinical use.
This webinar will address how the implementation of both manufacturing templates and platform characterization and safety assays can increase the likelihood of success in process validation and reduce risk in the timeline to commercialization for your gene therapy product. Using adeno-associated virus (AAV) as a case study, we will demonstrate how our validated, templated process for production can reduce the need for qualification inherent in niche manufacturing workflows and anticipate forthcoming needs for process performance qualification. This webinar will also highlight benefits from a new, platform assay offering for characterization and safety testing of AAV. Because these assays are pre-qualified, they reduce the variability inherent in assay validation and subsequently the time needed to establish readiness for regulatory compliance.
While these developments increase the standardization across the manufacturing and testing workflows, they remain flexible to clients' needs and are created to be scalable and as future-proof as possible, allowing for adaptability as the regulatory landscape of gene therapies evolves.
In this webinar, you will learn:
● The unit operations in AAV manufacturing that are ideal for templating
● How the manufacturing workflow can be targeted to reduce variability in testing and improve readiness for commercial production
● How platform assays can ease the burden of assay qualification and improve overall commercialization timelines
USP <665> draft standard : A rational risk-based approach to characterization...MilliporeSigma
This webinar will cover risk-based characterization of filters and single-use systems used in biopharmaceutical manufacturing according to USP <665>.
Novel innovative biomanufacturing systems such as single-use assemblies often comprise of polymeric materials. There is a lack of standards for characterization of these polymeric systems. USP <665> draft standard is the first standard in development addressing this topic. This chapter recommends risk assessment with respect to patient safety, risk level assignment and risk level appropriate characterization of components.
In this webinar we will discuss:
● Risk assessment to assign a risk level
● Risk level based testing
● Our approach for compliance
● Emprove™ Dossiers for Filters and Single-use systems
Polymer based drug delivery systems for parenteral controlled release: from s...Merck Life Sciences
This webinar, presented by two world-class experts in polymer based parenteral controlled-release drug delivery technologies, will provide insights into formulation technologies from small molecules up to biologics.
There is an increasing interest in long-acting injectables as drugs administered through injection help to increase patient compliance due to reduced frequency of administration while providing the same therapeutic efficiency. Depending from the nature of the drug, the optimum polymer technology is to be selected.
Prof. Dr. Mäder focus on how to select the appropriate PLA/PLGA polymer for small drug molecule applications. He will provide an overview of drug delivery systems, most important formulation techniques and appropriate characterization methods along with application examples.
Alternative polymer systems are required for peptide and protein controlled-release formulations. Dr. Rob Steendam introduces InnoCore´s SynBioSys® biodegradable polymer system demonstrating excellent safety, control over release kinetics and effective preservation of structural integrity and bioactivity of biologics. InnoCore Pharmaceuticals and SynBioSys® multi-block polymer introduction, challenges in development of controlled-release formulations of biological therapeutics including various examples and development and cGMP manufacturing at InnoCore are key elements of his presentation.
In this webinar, you will learn:
• drug delivery systems
• most important formulation techniques
• appropriate characterization methods along with application examples
Webinar: Benefits of Monodisperse Activated PEGs in ADC DevelopmentMilliporeSigma
Watch the webinar here: http://bit.ly/PEGsWebinar
As the field of antibody-drug conjugation chemistry has advanced, the use of linkers to impart "drugability" has also been growing. Recent literature shows a variety of linear and branched monodisperse PEGs being called on to modify the PK/PD profile of some of the most active but lipophilic payloads. In this webinar, we will survey the types of PEGs that are used in ADCs and give examples of successful implementation to change the biophysical properties of the construct. In addition, we will focus on PEGs and why activated PEGs are widely used to improve the pharmacokinetics of drugs (such as pegylated proteins, peptides etc.). Raw materials used in this field may have a variety of polydispersity. However, when it comes to the use of activated PEGs as linkers for ADCs, the requirements are significantly higher. Therefore, the choice and control of the PEG linker is crucial for the successful development and accelerated time to market for your ADC.
This webinar addresses critical success factors such as:
• Survey of current PEG enhanced ADCs
• Why PEGs are used designing ADCs
• Critical parameters for aPEGs used as linkers
• Requirements in terms of analytical capabilities
Upcoming USP 665 - Level of Characterization of Single-Use Systems Today and ...MilliporeSigma
Register for the interactive, on-demand webinar now: https://bit.ly/USP665
Single-use plastic systems are being utilized more frequently especially for COVID-19 vaccine manufacturing. However, there are issues regarding standardization of quality information that limits implementation efficiencies. One of the challenges is the evaluation of leachables derived from a variety of different plastic components in a timely manner.
Since the USP <665> highlights a risk assessment approach with no typical pass/fail limit, approaches to decision-making based on the extractables data package will be reviewed. In addition, we will highlight legacy testing requirements which may not be necessary once USP <665> is implemented.
In this webinar, we will discuss:
- Regulatory expectations of extractables and leachables assessment today and tomorrow
- The right criteria that need to be assessed to select the type and quality of plastic materials for use in biopharmaceutical manufacturing
In this webinar, you will learn:
Sources of endotoxin contamination
Contamination control strategy
Endotoxin removal strategies
Detailed description:
Endotoxin, a lipopolysaccharide (LPS), is a type of pyrogen and is a component of the exterior cell wall of Gram-negative bacteria. To ensure safety on patient’s endotoxin content in the drug should always be controlled. In a biological processing it may emanate from facility, utility, raw materials, process, and personnel. In this webinar we discuss the regulatory norms, strategies for prevention & removal of endotoxin to ensure that the final drug product is safe.
The Role of BPOG Extractables Data in the Effective Adoption of Single-Use Sy...Merck Life Sciences
The successful adoption of single-use technologies in a biopharmaceutical process largely relies on confidently selecting the right components for use in the fluid path of a product, within a specific process. An important step in choosing such components requires generating an extractables profile, which can be done by carefully selecting the solvent streams and extraction conditions to model the product and process steps complemented with the right analytical strategy.
In this webinar, you will learn:
● An approach to adopt the BioPhorum Operations Group (BPOG) extractables protocol as a baseline testing strategy.
● How to apply extractables data to a specific process followed by a systematic, risk-based safety assessment approach used for comparing known safety concern thresholds.
● The important stages in the risk assessment process as demonstrated by case studies from typical drug manufacturing processes where single-use components were used.
Process equipment characterization – how standardized extractables data suppo...Merck Life Sciences
View the recording here: https://bit.ly/35KIwBb
Biopharmaceutical Industry recently increased adoption of Single-Use systems and components in manufacturing process operations. Drug manufacturers are responsible for the characterization of SU components and systems used for the production to ensure patient safety. SUS Suppliers are encouraged by BPOG and BPSA to provide comprehensive extractables data package to support drug manufacturer’s E&L assessments.
This webinar will give an overview of the E&L evaluation workflow and practical study approaches from both supplier and end-user perspective, in accordance with the latest industry’s standards and upcoming USP <665> requirements. Case studies will be presented on how the data from suppliers are used to mitigate risk associated to SU materials, highlighting the key role of collaboration between the supplier and the drug manufacturer.
Challenges using Multiple Single-use Systems: Functionality versus Extractabl...Merck Life Sciences
As single-use technologies continue to expand in pharmaceutical manufacturing processes, the risk assessment for extractables and leachables becomes increasingly complex. Join this webinar to obtain guidance on how to perform risk evaluation on a process with multiple single-use components.
A Single-Use System (SUS) is typically designed for a specific process step. In many cases, single-use components are chosen based on their functionality. The challenge arises when there are multiple processing steps-- as the different applications and product matrices are evaluated, the complexity of the risk assessment increases. Complexity includes component evaluation, process conditions, and model solvents streams which ultimately relates to the patient safety risk.
This webinar will evaluate the different single-use components with respect to compatibility and extractables and leachables. A case study will be used to demonstrate the complexity and potential concerns when performing a risk evaluation on the manufacturing process.
In this webinar, you will learn:
- Risk assessment of extractables
- Single-use component evaluation
- Complexity when evaluating multiple assemblies
The Emprove® Program: Introduction of New Portfolio AdditionsMerck Life Sciences
Watch the presentation of this webinar here: https://bit.ly/3wT5Irt
The Emprove® Program is constantly expanding and updating. Find out about the recently launched Emprove® CCM category for our cell culture media portfolio, the new Emprove® API Information Packages, and the latest updates on available TUPPs for our Emprove® Chemicals portfolio.
This virtual user group will introduce the two latest additions to the Emprove® Program:
1. Our latest category Emprove® CCM for our cell culture media with wave 1 focus on our Cellvento® CHO cell culture media portfolio, looking also into the content of our three Emprove® Dossiers.
2. The new Emprove® API Information Packages that are currently created for our Emprove® APIs, looking into the content and showing how to access.
Additionally, we'll give an overview of latest Technically Unavoidable Particle Profiles (TUPPs) for our Emprove® Chemicals.
In this virtual user group, you will learn:
• Emprove® CCM and its documentation for our cell culture media
• Content and availability of the new Emprove® API Information Package
• Update on available TUPPs for our Emprove® Chemicals
Webinar: Post Approval Changes in Biologics Manufacturing - A Practical Asses...MilliporeSigma
Participate in the interactive webinar: http://bit.ly/PACWebinar
Post-approval changes for biologics manufacturing processes are complicated and challenging with the current global diverse regulatory environment. Here, we will present approaches to make these changes more efficient using a risk-based approach.
Explore our webinar library: www.emdmillipore.com/webinars
Excipients selection for high risk formulations Smita RajputMilliporeSigma
Are you choosing the right excipients for your high risk application? Find out how to select the right excipients and enable your process optimization to improve the total cost of ownership.
In this webinar, you will learn:
• Selection of right excipients for high risk formulation is very critical step
• Low Endotoxin and low bioburden limits are important aspect while selecting raw materials
• Strong regulatory support is crucial for high risk formulation
Excipients selection for high risk formulations like parenteral and ophthalmic applications is very challenging. Excipients should be inert with high purity for such dosage forms because trace amounts of impurities present in excipients can interact with active pharmaceutical ingredient (API) which results in instability of the formulation. This presentation discusses how to select the right excipients for high-risk applications and gives guidance for process optimization by choosing the best combination of filters and excipients to improve the total cost of ownership.
Key to Successful Formulation Development for Lipid Based RNA Delivery and Va...Merck Life Sciences
In this webinar, we will discuss:
• The application of RNA therapeutics and the different drug delivery routes used in the clinic.
• Design principles for developing lipids-based RNA formulations.
• Critical parameters to consider for cost effective development and consistent performance of RNA therapeutics and vaccines.
RNA therapeutics are changing the way we address diseases. Applications range from gene therapy, oncology, to vaccines for infectious diseases such as COVID-19.
The performance of RNA therapeutics critically depends on its formulation. Key decisions have to be made early on in the drug development process; choosing the appropriate drug delivery method and novel excipients. Raw material source and judicious choice of chemistry, ultimately determine the quality of novel lipid excipients which, in turn, has a big impact on the performance, reproducibility, costs, and regulatory approval timelines. This webinar will propose solutions to maximize the probability of success while formulating RNA therapeutics and vaccines.
Participate in the interactive webinar now: https://bit.ly/2xXMZlm
Explore our webinar library: www.merckmillipore.com/webinars
Liposomal Delivery Systems in Cancer Therapy - Creative BiolabsCreative-Biolabs
The lipid-based drug delivery system is a newly developed drug carrier that can be applied for various cancer-targeted treatments with many superiorities. This video briefly introduces various types of liposomes, the principles of liposomal drug delivery systems for cancer therapy, and liposome development services and products provided by Creative Biolabs.
Solid lipid nanoparticles were prepared by using the solvent evaporation method. The prepared solid lipid nanoparticles of
felodipine from different batches were weighed accurately and filled into appropriate-sized capsules for further evaluation.
The prepared nanoformulation was tested in vitro for drug entrapment efficiency found to be 97-99% for the formulation
with soya lecithin and glyceryl mono-stearate and percentage yield. The results of the in vitro drug release study for all
formulations performed at gastric pH 1.2 indicated that the drug was released in an immediate manner within 60 minutes,
which could be due to a burst effect or a faster rate of drug release due to smaller particle size SLN13 the concentration of
Glycerol Monosterate- 576.522mg and Poloxamer 188. In vitro release data from standardised nanoformulations was fitted
to various release kinetics models for the drug release mechanism. The optimised formulation demonstrated improved drug
release and entrapment efficiency, and in vitro release data were fitted to various kinetic models to better understand the
drug release mechanism. In vitro assay results were ranged between 76.5 to 96%, Zeta potential -40.75 ± 2.33 (mV) ± SD,
entrapment efficiency 99.32. Accelerated stability studies for standardised nanoformulations were performed for 6 months in
accordance with standard ICH guidelines, and no appreciable changes in the physical appearance, drug content, and in vitro
drug release rates of the nanoformulations were observed. As a result of the study's evidence, it is concluded that felodipineloaded solid lipid nanoparticles are the best choice for hypertension.
Sterile filtration of complex injectables by Partha BanerjeeMilliporeSigma
Sterile filtration and filter validation remain a critical segment during the development of these segments of products. Let's find the same by understanding a checklist and visualize certain case studies.
As the sterile injectable market continues to see rapid growth (~10% to 15% per annum) – outpacing the growth of oral products – it is natural to see the diversity of parenteral product formulations increasing in parallel. The definition of complexity in parenteral formulation development is broad. It varies based on the stage of development and the specific nature of the challenge. A notionally simple, stable reproducible laboratory formulation may carry a level of complexity in aseptic control if routine means of sterilization are unavailable.
Sterile filtration process intensification can bring significant benefits to manufacturers in terms of manufacturing flexibility, reduction of risks, better turn around time, thus achieving significantly higher productivity. We will identify these scenarios with case studies to reduce complications in manufacturing and process development.
Sterile filtration of complex injectables by Partha BanerjeeMerck Life Sciences
Sterile filtration and filter validation remain a critical segment during the development of these segments of products. Let's find the same by understanding a checklist and visualize certain case studies.
As the sterile injectable market continues to see rapid growth (~10% to 15% per annum) – outpacing the growth of oral products – it is natural to see the diversity of parenteral product formulations increasing in parallel. The definition of complexity in parenteral formulation development is broad. It varies based on the stage of development and the specific nature of the challenge. A notionally simple, stable reproducible laboratory formulation may carry a level of complexity in aseptic control if routine means of sterilization are unavailable.
Sterile filtration process intensification can bring significant benefits to manufacturers in terms of manufacturing flexibility, reduction of risks, better turn around time, thus achieving significantly higher productivity. We will identify these scenarios with case studies to reduce complications in manufacturing and process development.
Sterile filtration of complex injectables by Partha BanerjeeMerck Life Sciences
Sterile filtration and filter validation remain a critical segment during the development of these segments of products. Let's find the same by understanding a checklist and visualize certain case studies.
As the sterile injectable market continues to see rapid growth (~10% to 15% per annum) – outpacing the growth of oral products – it is natural to see the diversity of parenteral product formulations increasing in parallel. The definition of complexity in parenteral formulation development is broad. It varies based on the stage of development and the specific nature of the challenge. A notionally simple, stable reproducible laboratory formulation may carry a level of complexity in aseptic control if routine means of sterilization are unavailable.
Sterile filtration process intensification can bring significant benefits to manufacturers in terms of manufacturing flexibility, reduction of risks, better turn around time, thus achieving significantly higher productivity. We will identify these scenarios with case studies to reduce complications in manufacturing and process development.
Oral lipid drug delivery system for poor water soluble drugsTanvi Shetty
The presentation talks about recent advancement in the delivery mode for many poorly water soluble drugs. Introduces you to various available Lipid based systems and a easy guide to use a specific mode through detailed description of pros & cons they hold.
Its will serve as very essential for researchers working on BCS class II drug to increase its systemic availability.
Specialized in custom synthesis of organic molecules. Broad experience of working on multiple business models - FTEs, FFS, Milestone services of pharmaceutical industry.
Commited to deliver highest quality products in a timely manner and value to the customers.
Diethyl Chlorophosphate Market is highly diverse, with a range of services and specialties, including quantitative and qualitative research, brand research, product testing, customer satisfaction research, and competitive analysis.
Hot melt extrusion with PVA – solubility enhancement, supersaturation perform...MilliporeSigma
Hot melt extrusion has successfully emerged as an innovative manufacturing technology in pharmaceutical industry for the creation of amorphous solid dispersions (ASDs).
In this webinar you will learn about the potential of hot melt extrusion to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA) as a matrix polymer. We will provide an overview about different types of solid dispersions and their evolution in the pharmaceutical field. A brief introduction in hot melt extrusion processing will be given as well as actual formulation trends. You will get insights in potential down-stream options to create your final dosage form and you will gain ideas on how to speed up your formulation development.
A detailed background of PVA will be provided including its physical properties as well as its regulatory status. PVA is more than a polymer. Due to its amphiphilic structure it has the potential to improve the supersaturation of low soluble APIs and to prevent precipitation after release. This highlights the versatility of PVA as an advanced polymer for HME applications and we will guide you through our latest research activities so that you can leverage our knowledge to improve your formulations.
This webinar includes:
- The current status and further potential of HME in pharmaceutical industry
- Advantages of PVA in the field of ASDs: Solubility improvement, impact on supersaturation potential, stability data generated on sample formulations & downstream options
- Deep dive into latest research activities: Permeation studies with Caco-2 cell membranes, pH shift studies to investigate supersaturation potential, ongoing research activities to get to know a more detailed understanding of matrix systems and their intermolecular interactions
In this webinar, you will learn:
- which potential hot melt extrusion has, to overcome challenges in API solubility and bioavailability by using polyvinyl alcohol (PVA)
- why PVA is more than just a polymer
- how to create your final dosage form and speed up your formulation development
Xcelience is a contract research organization that has provided formulation development and clinical trial manufacturing solutions for pharmaceutical companies since 1997. The company is renowned for reliably expediting early development activities to speed potential drugs to clinical trials while applying stage-specific scientific knowledge and experience. Core services include: API Characterization, Analytical Development and Stability Services, Formulation Development, and Clinical Trial Manufacturing, Packaging and Labeling. For more detailed information about Xcelience, visit www.xcelience.com
Similar to Successful Drug Development with Synthetic Lipids: Critical Aspects and Strategies (20)
The Viscosity Reduction Platform: Viscosity-reducing excipients for improveme...MilliporeSigma
Protein viscosity is a major challenge in preparing highly concentrated protein formulations suitable for subcutaneous injection. Recently, the Viscosity Reduction Platform (VRP) was introduced and its technical key features and benefits for formulations were discussed. However, highly viscous solutions do not only pose a challenge when administering a drug to a patient, they can also impose technical limitations in the manufacturing process.
This white paper evaluates the effect of the excipients in the Viscosity Reduction Platform on ultrafiltration processes used to produce a highly concentrated formulation of a monoclonal antibody (mAb). Two filtration methods are demonstrated in this work.
Find more information about the Viscosity Reduction Platform on our website: https://www.sigmaaldrich.com/products/pharma-and-biopharma-manufacturing/formulation/viscosity-reduction-platform
Use of Excipients in Downstream Processing to Improve Protein PurificationMilliporeSigma
Excipients are used to improve the stability of protein-based therapeutics by protecting the protein against a range of stress conditions such as temperature changes, pH changes, or agitation. Similar stresses are applied to proteins during downstream purification. Shifts in pH during Protein A chromatography, subsequent incubations at low pH for virus inactivation, and changes in conductivity in ion exchange chromatography can lead to aggregation, fragmentation, or other chemical modifications of the therapeutic protein. Given the potential impact on the protein’s structural integrity, there is a need for approaches to reduce the risk presented by the conditions during downstream processing. For example, integration of a solution to prevent aggregation of proteins would be a more efficient strategy than implementing steps to remove multimeric forms.
This white paper highlights the results from a recent paper by Stange et. al., in which protein stabilizing excipients such as polyols, sugars, and polyethylene glycol (PEG4000) were used as buffer system additives. Effect of the excipients on elution patterns, stabilization of the monomer antibody, host-cell protein removal, virus inactivation rates and binding capacity of cation exchange chromatography were explored.
Exploring the protein stabilizing capability of surfactants against agitation...MilliporeSigma
Agitation of therapeutic protein solutions during manufacturing, shipping and handling is one of the major initiators for protein aggregation and particle formation during the life history of a protein drug. Adsorption of protein molecules to liquid-air interfaces leads to the formation of highly concentrated protein surface films. The rupture of these protein films due to various mechanical processes can then result in the appearance of protein aggregates and particles in the bulk solution phase.
One technique to stabilize proteins against stress induced by liquid-air interfaces is the use of non-ionic surfactants. About 91% of antibody formulations commercially available in 2021 contained a surfactant. Polysorbate 20 and 80, composed of a hydrophilic polyoxyethylene sorbitan and hydrophobic fatty acid esters, made up the largest part being employed in 87% of said formulations.
Despite their frequent use in parenteral drug products, concerns have been raised for decades about the application of polysorbates as surfactants in biopharmaceutical formulations. Autoxidation of polysorbate, caused by residual peroxides in polysorbates, can damage the proteins and can further drive the oxidative degradation of polysorbate. Chemical and enzymatic hydrolysis of polysorbate may lead to the formation of free fatty acid particles, which may become visible; and both mechanisms eventually lead to the reduction in polysorbate concentration. Therefore, the purpose of the current study was to compare various molecules for their capabilities to reduced agitation-induced protein aggregation and particle formation; and furthermore, investigate their underlying protein stabilizing mechanisms.
The Viscosity Reduction Platform: Viscosity Reducing Excipients for Protein F...MilliporeSigma
Protein viscosity is one of the major obstacles in preparing highly concentrated protein formulations suitable for subcutaneous injection.
This whitepaper examines how combining an amino acid with a second viscosity-reducing excipient circumvents adverse effects on protein stability and improves viscosity-reducing capacity.
To find more information about the Viscosity Reduction Platform, please visit our website: https://sigmaaldrich.com/products/pharma-and-biopharma-manufacturing/formulation/viscosity-reduction-platform
Characterization of monoclonal antibodies and Antibody drug conjugates by Sur...MilliporeSigma
Watch the presentation of this webinar: https://bit.ly/3Pjpjvr
Highlights of this webinar:
- Surface plasmon resonance as a powerful tool for biologic characterization including mAbs and ADCs.
- SPR allows rapid binding analysis in real time without using labels for SARS-CoV-2 receptor binding domain mutations.
- Kinetic data is indicative of possible neutralizing activity allowed assessment of neutralizing ability of therapeutic monoclonal antibodies.
- The application can provide preliminarily efficacy information and facilitated mAbs/ACDs candidate selection process
Detailed description:
Characterization of therapeutic monoclonal antibodies (mAbs) or Antibody drug conjugates (ADCs) is challenging due to their ability to bind to a variety of proteins via their Fc and Fab domains, giving rise to diverse biological functions associated with each domain. The Fc domain of mAbs interacts with Fc receptors with varying affinities, which can influence biological processes such as Complement-dependent cytotoxicity (CDC) and Antibody-dependent cellular cytotoxicity (ADCC), transcytosis, phagocytosis, and/or serum half-life.
An important characteristic of an antibody is its Fc effector function. Antibodies can be engineered to obtain desired binding of the Fc region to Fc receptors expressed on effector cells. Hence, it is crucial to evaluate the binding interaction of mAbs/ADC with Fc receptors in the early phase of drug development to understand the potential biological activity of the product in vivo.
Surface Plasmon Resonance (SPR) is a powerful technique to establish binding kinetics in real-time, label free, and high sensitivity with low sample consumption. Along with target antigen binding, it is crucial to evaluate the binding interaction of antibodies and ADCs with Fc receptors. Our SPR case studies investigated the impact on binding kinetics of ADCs with different linkers and the binding interactions of SARS-CoV-2 spike protein variants and evaluated the neutralizing ability of therapeutic mAbs. SPR characterisation can be facilitated in all stages of the product life cycle to ensure the quality and safety of mAbs and ADCs.
The Role of BioPhorum Extractables Data in the Effective Adoption of Single-U...MilliporeSigma
Regulatory expectation does require patient safety evaluations with supporting data for manufacturing components that directly come into contact with drug manufacturing process streams. Readily available extractables data can help manufacturers using singleuse technology to accelerate product qualifications, risk assessments and process optimization
This white paper guides you on how to save time and resources with supplier-provided single-use system extractables data and gives you an overview about the overall strategy for Extractables & Leachables. At the end you will find a case study.
Find more information about filters and single-use components on our website: https://www.sigmaaldrich.com/DE/en/services/product-services/emprove-program/emprove-filter-and-single-use-component-portfolio
The Future of Pharma- and Biopharmaceutical AuditsMilliporeSigma
Watch the recording of this presentation here: https://bit.ly/3zTOpe4
Detailed description:
SARS-CoV-2 showed us that technology supports us during our inspection activity even if on-site visits are not possible. Travel restrictions of various kinds will remain a risk in the future. The use of new technologies has shown that inspections and audits can be carried out despite these restrictions. We will focus on what possibilities the new technologies offer and take a look at the future of inspections and audits.
In this webinar, you will learn:
• Regulatory overview of remote audits
• The technologies needed to support the audit process
• What types of inspections are possible with the use of these technologies
• How audits may look in the future
Presented by:
Daniel Buescher, Product Manager - Digital Solutions
Moving your Gene Therapy from R&D to IND: How to navigate the Regulatory Land...MilliporeSigma
Watch the recording of this presentation here: https://bit.ly/3SqOsoP
Novel therapies, including cell and gene therapies, continue to be central to innovation in healthcare and represent the fastest growing area of therapeutic medicine. As a consequence, the number of gene therapies undergoing clinical trials has increased significantly in the last five years.
Manufacturing processes for these novel therapeutics are very complex with a high risk of contamination. Regulatory agencies world-wide have responded by issuing guidance to outline their expectations for development and manufacture of cell and gene therapies. Currently, regulatory guidance is not harmonized globally and can often lead to confusion within industry and increased risk of non-compliance.
In this webinar, we'll answer:
• Which regulatory guidelines do you need to comply for your INDs?
• When do you start implementing GMPs and validated assays?
• How do you get your QC testing strategy ‘right the first time’?
• How do you ensure testing is not your rate limiting step for the IND submission?
Presented by:
Manjula Aysola, Senior Regulatory Consultant
Dr. Alison Armstrong, Sr. Director, Technical and Scientific Solutions
Identity testing by NGS as a means of risk mitigation for viral gene therapiesMilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3RijkHC
Detailed description:
Imagine you’ve just completed a manufacturing run for your viral vector. Identity testing is performed to confirm the vector sequence. But when the results come back the data reveals unexpected sequence variants! With an appropriate risk mitigation testing strategy, this situation can be prevented.
The situation described above is not hypothetical, and happens more that you think, costing valuable time and resources.
Investigatory testing has shown that sequence variants present in starting materials (e.g. plasmids) are likely to make their way to the final product. Adequate identification of low-level variants with an appropriately sensitive method is critical in ensuring the quality of the final product. A risk-based testing strategy, in the context of identity, for viral vector manufacturing will be presented, focusing on key testing points. NGS assays for identity and variant detection will be highlighted due to their extremely sensitive nature compared to traditional approaches.
In this webinar, we'll explore:
• Regulatory requirements for identity testing
• NGS applications for identity testing as compared to traditional methods
• A case study on the impact of not establishing a proper risk-based testing strategy
Presented by: Bradley Hasson, Director of Lab Operations for NGS Services
Latest advancements of melt based 3D printing technologies for oral drug deli...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3A2WcH4
The application of polymer excipients in 3D printing manufacturing is usually limited due to the concerns of filament strength, high processing temperature and large scale manufacturing.
Latest technology developments are targeting a direct melt deposition to simplify the process and enable a constant and efficient process. Two different processing approaches will be presented:
The advanced melt drop deposition, where individual three dimensional geometries can be created by depostition of polymer droplets and the MED® 3D printing technology which allows by precise layer-by-layer deposition to produce objects with well-designed geometric structures.
In this webinar, you will learn:
• Latest advancements of melt based 3D printing approaches
• Application examples for the individual technologies
• Deep dive in the MED® 3D printing technology to design dedicated drug release profiles
Presented by:
Dr. Thomas Kipping, Head of Drug Carriers
Dr. Xianghao Zuo, Deputy Director of R&D, Triastek
CAR-T Manufacturing Innovations that Work - Automating Low Volume Processes a...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3NDNIKe
Automated, fit-for-purpose tools are essential in CAR-T processing to support sustainable manufacturing of clinical and market-approved cell therapy products. This webinar will discuss how the ekko™ Acoustic Cell Processing System uses acoustic technology as a touchless approach to manipulate cells, enabling a modular tool across the CAR-T manufacturing workflow. Typical performance of templated ekko™ System processes for DMSO washout of leukapheresis material, low volume and high cell concentrate for electroporation preparation, and harvest of expanded T cells will be reviewed.
This webinar will also give an early glimpse at the ekko™ Select System for unmatched T cell selection.
In this webinar, you will:
• Uncover how the ekko™ System supports the broad industrialization of cell therapy, with particular focus on how to achieve low volume, high concentrate cell product for critical transduction and transfection steps
• Discover how ekko™ System for wash and concentrate processes throughout the cell therapy workflow achieve high cell recovery, viability, and effective residual removal
• Preview to ekko™ Select, our cell therapy selection platform, to achieve unmatched ease-of-use with direct processing from leukopaks reducing the need for preparation steps
Presented by:
Benjamin Ross-Johnsrud, Acoustic Technology Expert
Robert Scott, Mechanical Engineer III
How does the ICH Q5A revision impact viral safety strategies for biologics?MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3t7X9tg
How does the ICH Q5A revision impact viral safety strategies for biologics?
Biologics continue to grow at a fast pace. Manufactured using cell lines of human or animal origin, these are at risk of viral contamination making safety strategies critical. A comprehensive risk mitigation strategy using multiple orthogonal measures is a regulatory expectation. ICH Q5A, the globally-harmonized guideline outlines the expectations. ICH Q5A is currently being revised to address recent scientific advancements including novel therapeutic modalities, new manufacturing paradigms, updates in viral clearance applications, and alternate detection technologies. We’ll discuss the expected changes and potential impact on viral safety strategies with case studies and examples.
In this webinar, you will learn about:
• The Importance of virus testing in biologics products
• Regulatory landscape, expectations for the Q5A revision
• What's new and changing
• Examples of alternate testing schedules, impact on viral clearance
Presented by:
Manjula Aysola, Senior Regulatory Consultant
Alison Armstrong, PhD, Sr. Director, Technical and Scientific Solutions
Improve Operational Efficiency by Over 30% with Product, Process, & Systems A...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3adaxWh
When implementing new automation systems, organizations must consider things like deployment time, user adoption, and costs.
They must also consider the cost of doing nothing – that is, what competitive advantage is lost in standing still? What time and quality is lost in repetitive, manual tasks rather than an automated, digital workflow? What operational efficiencies are lost?
In this webinar we examine how a product, process, and system agnostic automation platform can be deployed faster than traditional system specific software while bringing greater operational efficiencies (in many cases over 30% improvement).
To remain competitive in the market, biopharma manufacturers must adopt automation and digital technologies, but most plants still have island of automation consisting of independently functioning, standalone unit operations. This results in operational inefficiency, regulatory concerns, and a poor understanding of the process and product life cycle.
Taking the first, right step must include considering risks, costs, timelines, and technology alternatives. Traditional automation approaches tied to specific systems, processes, and products are, by their nature, limited; while an agnostic platform will address current biomanufacturing business challenges and ensure future readiness. With the right platform, a phased automation implementation can yield operational efficiency gains of up to 30% and improved product quality and regulatory compliance.
In this webinar, let's explore:
• Challenges of automation and digital technology adoption
• What a product, process, and system agnostic platform entails
• Applications and benefits of a process orchestration platform
• Ensuring future readiness with process orchestration
Presented by:
Braj Nandan Thakur, Global Product Manager - Automation
Insights from a Global Collaboration Accelerating Vaccine Development with an...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3Nbb5ug
Get insights and best practices from a multinational team establishing a platform for vaccine production. See how a long-term collaboration on a bench-scale process used to produce a Virus Like Particle (VLP) vaccine for SARS-CoV-2 was successfully converted to a robust GMP-compatible, scalable process.
The COVID-19 pandemic further emphasized the need for collaboration in the development of urgently needed vaccines and therapeutics. In this webinar, we take you behind the scenes of our collaboration with Technovax and Innovative Biotech in which a scalable VLP vaccine platform was optimized for use in a production facility in Nigeria in response to the need for local production of SARS-CoV-2 vaccines. The flexibility and robustness of the platform will enable its rapid deployment to support the West African pandemic readiness program. Initial development of the VLP process began in late 2019 and by March 2020, was already adapted for production of a SARS-CoV-2 vaccine.
In this webinar, you will learn:
• About building a priceless collaborative network with integrated solutions
• Virus-Like Particle Vaccines
• Process Development Overview and Challenges
• Pre-clinical Results and Next Steps
Presented by:
Jose M. Galarza, PhD,
President and Founder of TechnoVax
Naomi Baer,
Business development consultant, Emerging Biotech, BioProcess division
Youssef Gaabouri, Eng. ,
Associate Director, Head of Sales Middle East & Africa, BioProcess division
Risk-Based Qualification of X-Ray Sterilization for Single-Use SystemsMilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3vQf0qv
In the single-use bioprocess industry, X-ray irradiation warrants consideration as an alternate sterilization technology. Using a risk-based qualification testing strategy is important when evaluating and implementing equivalent ionizing irradiation sterilization methods.
The urgent need for life-saving therapies as a result of the global pandemic has reinforced the criticality of flexibility in pharmaceutical manufacturing, including sterilization. The single-use bioprocess industry traditionally has employed gamma irradiation sterilization. X-ray irradiation is being considered as an additional sterilization technology for business and supply continuity. We will share a risk-based qualification testing strategy including Extractables and data generated to support comparability of gamma irradiation and X-ray irradiation as equivalent ionizing irradiation sterilization methods.
In this webinar, you will learn about:
• The comparison of gamma and X-ray irradiation sterilization
• A risk-based qualification test strategy
• Data evaluation of gamma versus X-ray sterilized single-use components
Presented by:
Monica Cardona,
Global Senior Program Manager
Paul Killian, Ph.D.,
R&D Director, Analytical Technologies
Rapid Replication Competent Adenovirus (rRCA) Detection: Accelerate your Lot ...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3MJ4u9V
Testing for presence of replication competent adenovirus (RCA) is a key component to ensure patient safety and a requirement for all biologicals manufactured using adenoviral vectors. For many adenoviral-based products, the RCA assay is a rate-limiting assay for lot release.
Join this webinar to learn about a rapid RCA detection assay currently in development, which combines a 7-day culture assay with a highly sensitive molecular endpoint specific for RCA. The method can detect presence of as little as 1 RCA in adenoviral vector material at an approximate concentration of 5x107 - 2x108 vector particles (VP)/mL, making it a suitable method to meet regulatory requirements while accelerating your lot release timelines.
In this webinar, you will learn about:
• Regulatory framework for adenoviral vector products
• Considerations for lot release testing of adenoviral-based therapies
• Advantages of a rapid method for RCA testing on production lot material
Presented by:
Axel Fun, Ph.D.,
Principal Scientist
Alberto Santana, MBA,
Product Manager, Biologics Biosafety Testing
The High Intensity Sweeteners Neotame and Sucralose: 2 Ways to ace the Patien...MilliporeSigma
Watch the presentation of this webinar here: https://bit.ly/3vQyN7K
Bitter medicines are an important issue, especially for pediatric applications. As several APIs have bitter tasting components, high intensity sweeteners for taste optimization are of great interest. Join our webinar to discover our new sweetener toolbox enabling safe and stable formulations.
Mask bitter aftertaste for a sweeter pill to swallow! Patients’ compliance and the therapeutic benefit are supported by a pleasant taste of pharmaceutical formulations. With the high intensity sweeteners Neotame and Sucralose, you have efficient tools at hand which are superior to other sweeteners in many aspects:
• excellent sugar-like taste profile
• outstanding sweetness factors
• use effectiveness
• enhanced stability
We will present our new toolbox of two high performance sweeteners and focus on aspects of stability, safety, the application in various dosage forms, and market perception.
In this webinar, you will learn:
• How to optimize the patients' taste experience of your pharmaceuticals
• How sweeteners can be differentiated by their sensory profiles and features
• How our new product offering Neotame can be effectively used in your targeted formulations
Presented by:
Almut von der Brelie,
Senior Manager Strategic Marketing, Excipients for Solid Applications
The Developability Classification System (DCS): Enabling an Optimized Approac...MilliporeSigma
This whitepaper by Dr. Daniel Joseph Price outlines how poorly soluble drug formulations can be designed using the developability classification system (DCS).
The DCS identifies the root cause of low solubility and enables lean, cost-effective and effective formulations to be developed.
#solubility #pharmaceuticalmanufacturing #oralsoliddosage #drugdevelopment
How to Accelerate and Enhance ADC TherapiesMilliporeSigma
In this webinar, you will learn about:
The advantages of using advanced intermediates to develop ADC therapies
How to increase ADC solubility and efficiency
Fast, small-scale ADC library generation
Seamless supply chain with reduced complexity and regulatory support
The ADCore product line offers versatile intermediates that simplify the synthesis of common ADC payloads (dolastatins, maytansinoids, and PBDs) by greatly reducing the number of synthetic steps. This translates to savings in development and manufacturing costs and shorter timelines to the clinic. To address the poor solubility of many ADC payloads, ChetoSensar™ was developed to significantly increase the hydrophilicity of the drug linker, which has been shown to also substantially increase the efficacy of ADCs and broaden the therapeutic window.
Lastly, the ADC Express™ service leverages conjugation chemistry and analytical expertise to help design and quickly synthesize sets of potential ADC therapies suitable for screening to simplify candidate selection and get ADC therapies to market faster.
Explore our infographic on 'Essential Metrics for Palliative Care Management' which highlights key performance indicators crucial for enhancing the quality and efficiency of palliative care services.
This visual guide breaks down important metrics across four categories: Patient-Centered Metrics, Care Efficiency Metrics, Quality of Life Metrics, and Staff Metrics. Each section is designed to help healthcare professionals monitor and improve care delivery for patients facing serious illnesses. Understand how to implement these metrics in your palliative care practices for better outcomes and higher satisfaction levels.
R3 Stem Cells and Kidney Repair A New Horizon in Nephrology.pptxR3 Stem Cell
R3 Stem Cells and Kidney Repair: A New Horizon in Nephrology" explores groundbreaking advancements in the use of R3 stem cells for kidney disease treatment. This insightful piece delves into the potential of these cells to regenerate damaged kidney tissue, offering new hope for patients and reshaping the future of nephrology.
Defecation
Normal defecation begins with movement in the left colon, moving stool toward the anus. When stool reaches the rectum, the distention causes relaxation of the internal sphincter and an awareness of the need to defecate. At the time of defecation, the external sphincter relaxes, and abdominal muscles contract, increasing intrarectal pressure and forcing the stool out
The Valsalva maneuver exerts pressure to expel faeces through a voluntary contraction of the abdominal muscles while maintaining forced expiration against a closed airway. Patients with cardiovascular disease, glaucoma, increased intracranial pressure, or a new surgical wound are at greater risk for cardiac dysrhythmias and elevated blood pressure with the Valsalva maneuver and need to avoid straining to pass the stool.
Normal defecation is painless, resulting in passage of soft, formed stool
CONSTIPATION
Constipation is a symptom, not a disease. Improper diet, reduced fluid intake, lack of exercise, and certain medications can cause constipation. For example, patients receiving opiates for pain after surgery often require a stool softener or laxative to prevent constipation. The signs of constipation include infrequent bowel movements (less than every 3 days), difficulty passing stools, excessive straining, inability to defecate at will, and hard feaces
IMPACTION
Fecal impaction results from unrelieved constipation. It is a collection of hardened feces wedged in the rectum that a person cannot expel. In cases of severe impaction the mass extends up into the sigmoid colon.
DIARRHEA
Diarrhea is an increase in the number of stools and the passage of liquid, unformed feces. It is associated with disorders affecting digestion, absorption, and secretion in the GI tract. Intestinal contents pass through the small and large intestine too quickly to allow for the usual absorption of fluid and nutrients. Irritation within the colon results in increased mucus secretion. As a result, feces become watery, and the patient is unable to control the urge to defecate. Normally an anal bag is safe and effective in long-term treatment of patients with fecal incontinence at home, in hospice, or in the hospital. Fecal incontinence is expensive and a potentially dangerous condition in terms of contamination and risk of skin ulceration
HEMORRHOIDS
Hemorrhoids are dilated, engorged veins in the lining of the rectum. They are either external or internal.
FLATULENCE
As gas accumulates in the lumen of the intestines, the bowel wall stretches and distends (flatulence). It is a common cause of abdominal fullness, pain, and cramping. Normally intestinal gas escapes through the mouth (belching) or the anus (passing of flatus)
FECAL INCONTINENCE
Fecal incontinence is the inability to control passage of feces and gas from the anus. Incontinence harms a patient’s body image
PREPARATION AND GIVING OF LAXATIVESACCORDING TO POTTER AND PERRY,
An enema is the instillation of a solution into the rectum and sig
Antibiotic Stewardship by Anushri Srivastava.pptxAnushriSrivastav
Stewardship is the act of taking good care of something.
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
WHO launched the Global Antimicrobial Resistance and Use Surveillance System (GLASS) in 2015 to fill knowledge gaps and inform strategies at all levels.
ACCORDING TO apic.org,
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
ACCORDING TO pewtrusts.org,
Antibiotic stewardship refers to efforts in doctors’ offices, hospitals, long term care facilities, and other health care settings to ensure that antibiotics are used only when necessary and appropriate
According to WHO,
Antimicrobial stewardship is a systematic approach to educate and support health care professionals to follow evidence-based guidelines for prescribing and administering antimicrobials
In 1996, John McGowan and Dale Gerding first applied the term antimicrobial stewardship, where they suggested a causal association between antimicrobial agent use and resistance. They also focused on the urgency of large-scale controlled trials of antimicrobial-use regulation employing sophisticated epidemiologic methods, molecular typing, and precise resistance mechanism analysis.
Antimicrobial Stewardship(AMS) refers to the optimal selection, dosing, and duration of antimicrobial treatment resulting in the best clinical outcome with minimal side effects to the patients and minimal impact on subsequent resistance.
According to the 2019 report, in the US, more than 2.8 million antibiotic-resistant infections occur each year, and more than 35000 people die. In addition to this, it also mentioned that 223,900 cases of Clostridoides difficile occurred in 2017, of which 12800 people died. The report did not include viruses or parasites
VISION
Being proactive
Supporting optimal animal and human health
Exploring ways to reduce overall use of antimicrobials
Using the drugs that prevent and treat disease by killing microscopic organisms in a responsible way
GOAL
to prevent the generation and spread of antimicrobial resistance (AMR). Doing so will preserve the effectiveness of these drugs in animals and humans for years to come.
being to preserve human and animal health and the effectiveness of antimicrobial medications.
to implement a multidisciplinary approach in assembling a stewardship team to include an infectious disease physician, a clinical pharmacist with infectious diseases training, infection preventionist, and a close collaboration with the staff in the clinical microbiology laboratory
to prevent antimicrobial overuse, misuse and abuse.
to minimize the developme
CRISPR-Cas9, a revolutionary gene-editing tool, holds immense potential to reshape medicine, agriculture, and our understanding of life. But like any powerful tool, it comes with ethical considerations.
Unveiling CRISPR: This naturally occurring bacterial defense system (crRNA & Cas9 protein) fights viruses. Scientists repurposed it for precise gene editing (correction, deletion, insertion) by targeting specific DNA sequences.
The Promise: CRISPR offers exciting possibilities:
Gene Therapy: Correcting genetic diseases like cystic fibrosis.
Agriculture: Engineering crops resistant to pests and harsh environments.
Research: Studying gene function to unlock new knowledge.
The Peril: Ethical concerns demand attention:
Off-target Effects: Unintended DNA edits can have unforeseen consequences.
Eugenics: Misusing CRISPR for designer babies raises social and ethical questions.
Equity: High costs could limit access to this potentially life-saving technology.
The Path Forward: Responsible development is crucial:
International Collaboration: Clear guidelines are needed for research and human trials.
Public Education: Open discussions ensure informed decisions about CRISPR.
Prioritize Safety and Ethics: Safety and ethical principles must be paramount.
CRISPR offers a powerful tool for a better future, but responsible development and addressing ethical concerns are essential. By prioritizing safety, fostering open dialogue, and ensuring equitable access, we can harness CRISPR's power for the benefit of all. (2998 characters)
Navigating Challenges: Mental Health, Legislation, and the Prison System in B...Guillermo Rivera
This conference will delve into the intricate intersections between mental health, legal frameworks, and the prison system in Bolivia. It aims to provide a comprehensive overview of the current challenges faced by mental health professionals working within the legislative and correctional landscapes. Topics of discussion will include the prevalence and impact of mental health issues among the incarcerated population, the effectiveness of existing mental health policies and legislation, and potential reforms to enhance the mental health support system within prisons.
India Clinical Trials Market: Industry Size and Growth Trends [2030] Analyzed...Kumar Satyam
According to TechSci Research report, "India Clinical Trials Market- By Region, Competition, Forecast & Opportunities, 2030F," the India Clinical Trials Market was valued at USD 2.05 billion in 2024 and is projected to grow at a compound annual growth rate (CAGR) of 8.64% through 2030. The market is driven by a variety of factors, making India an attractive destination for pharmaceutical companies and researchers. India's vast and diverse patient population, cost-effective operational environment, and a large pool of skilled medical professionals contribute significantly to the market's growth. Additionally, increasing government support in streamlining regulations and the growing prevalence of lifestyle diseases further propel the clinical trials market.
Growing Prevalence of Lifestyle Diseases
The rising incidence of lifestyle diseases such as diabetes, cardiovascular diseases, and cancer is a major trend driving the clinical trials market in India. These conditions necessitate the development and testing of new treatment methods, creating a robust demand for clinical trials. The increasing burden of these diseases highlights the need for innovative therapies and underscores the importance of India as a key player in global clinical research.
ICH Guidelines for Pharmacovigilance.pdfNEHA GUPTA
The "ICH Guidelines for Pharmacovigilance" PDF provides a comprehensive overview of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines related to pharmacovigilance. These guidelines aim to ensure that drugs are safe and effective for patients by monitoring and assessing adverse effects, ensuring proper reporting systems, and improving risk management practices. The document is essential for professionals in the pharmaceutical industry, regulatory authorities, and healthcare providers, offering detailed procedures and standards for pharmacovigilance activities to enhance drug safety and protect public health.
2. The life science business of
Merck KGaA, Darmstadt, Germany
operates as MilliporeSigma
in the U.S. and Canada.
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 20182
3. 01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
3
4. 01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
4
5. Introduction
Lipids: What makes them special?
5 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
hydrophilic head hydrophobic tail
1,3-GDO
(R)-DODMA
DPPC
6. 6 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
Liposome rigidity
Phase transition temperature
Liposome leakage
Liposome shape
Introduction
Lipid-based structures in an aqueous environment
BILAYERED MONOLAYERED
LIQUID CORE
SOLID CORE
7. 7 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
Surface charge, polarity
Liposome shape
Liposome size
Cell affinity
Introduction
Lipid-based structures in an aqueous environment
Non-targeted Targeted
Non-PEGylated
PEGylated
8. Introduction
Liposome manufacture
Lipids in organic solvent
(and hydrophobic drug)
Drying step Hydration
Addition of water
(and hydrophilic drug)
Stirring
Sonication
Extusion
Homogenization
Multilamellar vesicles Unilamellar vesicles
Lipid film / cake
+ Purification
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 20188
Mechanical methods
• Shaking of phospholipid dispersions
• Extrusion through a filter (low/medium
pressure)
• Extrusion through a French press cell
(Microfluidizer technique)
• High-pressure homogenization
• Ultrasonic irritation
• Bubbling of gas
Methods based on organic
solvent replacement by
aqueous media
• Organic solvent removal
• Use of water-inmiscible solvents
• Ethanol injection method
• Ether infusion (solvent vaporization)
• Reverse-phase evaporation
Methods based on detergent
removal
• Gel exclusion chromatography
• Slow dialysis
• Fast dilution
• Other related techniques
Wagner and Vorauer-Uhl. „Liposome technology for industrial Purposes“ Journal of Drug Delivery 2011(2010).
Challenge: scalable + robust + efficient processes
9. Liposomal formulations in the market
9
Product
(Approval Year)
Admin.
route
Active Agent Indication
Doxil® (1995) i.v. Doxorubicin Ovarian, breast cancer, Kaposi’s sarcoma
DaunoXome®
(1996)
i.v. Daunorubicin AIDS-related Kaposi’s sarcoma
Depocyt® (1999) Spinal Cytarabine/Ara-C Neoplastic meningitis
Myocet® (2000) i.v. Doxorubicin Combination therapy with cyclophosphamide in metastatic breast
cance
Mepact® (2004) i.v. Mifamurtide High-grade, resectable, non-metastatic osteosarcoma
Marqibo® (2012) i.v. Vincristine Acute lymphoblastic leukaemia
Onivyde™ (2015) i.v. Irinotecan Combination therapy with fluorouracil and leucovorin in metastatic
adenocarcinoma of the pancreas
Abelcet® (1995) i.v. Amphotericin B Invasive severe fungal infections
Ambisome® (1997) i.v. Amphotericin B Presumed fungal infections
Amphotec® (1996) i.v. Amphotericin B Severe fungal infections
Visudyne® (2000) i.v. Verteporphin Choroidal neovascularisation
DepoDur™ (2004) Epidural Morphine sulfate Pain management
Exparel® (2011) i.v. Bupivacaine Pain management
Epaxal® (1993) i.v. Inactivated hepatitis A virus
(strain RGSB)
Hepatitis A
Inflexal® V (1997) i.v. Inactivated hemaglutinine of
Influenza virus strains A and B
Influenza
Adapted from Bulbake and col. “Liposomal Formulations in Clinical Use: An Updated Review” Pharmaceutics 2017, 9, 12.
10. Liposomal formulations in clinical trials (1)
10
Product Admin.
route
Active Agent Indication
Arikace Aerosol Amikacin Lung infections
Stimuvax s.c. Tecemotide Non-small cell lung cancer
T4N5 liposomal
lotion
Topical T4 endonuclease V Xeroderma pigmentosum
Liprostin i.v. Prostaglandin E-1 (PGE-1) Restenosis after angioplasty
ThermoDox i.v. Doxorubicin Hepatocellular carcinoma and also recurring chest wall breast cancer
Lipoplatin i.v. Cisplatin Non-small cell lung cancer
Aroplatin i.v. Platinum analogue Metastatic colorectal cancer
Liposomal
annamycin
i.v. Semi-synthetic doxorubicin
analogue annamycin
Relapsed or refractory acute
myeloid leukaemia
SPI-077 i.v. Cisplatin Lung, head and neck cancer
OSI-211 i.v. Lurtotecan Ovarian, head and neck cancer
S-CKD602 i.v. Potent topoisomerase I inhibitor Cancer
LE-SN38 i.v. Irinotecan’s active metabolite Advanced colorectal cancer
LEP-ETU i.v. Paclitaxel Cancer
Endotag-I i.v. Paclitaxel Breast and pancreatic cancers
Atragen i.v. All-trans retinoic acid Hormone-resistant prostate cancer, renal cell carcinoma and acute
myelogenous leukaemia
Adapted from Bulbake and col. “Liposomal Formulations in Clinical Use: An Updated Review” Pharmaceutics 2017, 9, 12.
11. Liposomal formulations in clinical trials (2)
11
Product Admin.
route
Active Agent Indication
LEM-ETU i.v. Mitoxantrone Various cancers
Liposomal
Grb-2
i.v. Antisense oligodeoxynucleotide growth factor receptor
boundprotein 2 (Grb-2)
Hematologic malignancies
INX-0125 i.v. Vinorelbine tartrate Advanced solid tumours
INX-0076 i.v. Topotecan Advanced solid tumours
TKM-080301 Hepatic intra-
arterial Admin.
PLK1 siRNA Neuroendocrine tumours
Atu027 i.v. PKN3 siRNA Pancreatic cancer
2B3-101 i.v. Doxorubicin Solid tumours
MTL-CEBPA i.v. CEBPA siRNA Liver cancer
ATI-1123 i.v. Docetaxel Solid tumours
LiPlaCis i.v. Cisplatin Advanced solid tumours
MCC-465 i.v. Doxorubicin Metastatic stomach cancer
SGT-53 i.v. p53 gene Various solid tumours
Alocrest i.v. Vinorelbine Breast and lung cancers
Adapted from Bulbake and col. “Liposomal Formulations in Clinical Use: An Updated Review” Pharmaceutics 2017, 9, 12.
12. Agenda
01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
12
01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
13. NDA/BLASUBMITTED
Lipid development
Pharmaceutical product development
Graph adapted from Pharmaceutical Research and Manufacturers of America, Biopharmaceuticals
in Perspective, Facts and Figures 2012, (Washington, DC: PhRMA, March 2012).
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201813
14. 14 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
Synthetic lipids for drug development
What is needed from them?
Convenient and reproducible behavior in formulation: particle characteristics, stability and release profile
High purity will influence:
• lipid stability
• lipid bilayer structure in formulation
• formulation stability
• formulation release profile
1
15. Lipids purity
How can it be optimized?
15 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
LIPID PURITY
16. Lipids purity
How can it be optimized?
16 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
LIPID PURITY
Quality of
starting
materials
High and consistent quality raw materials
• Low level of by-products
• Defined stereochemistry (cis/trans)
• Low bioburden and endotoxin levels
• Plant-derived raw materials with
BSE/TSE and non-GMO certificates
• Use class II and III solvents
17. Lipids purity
How can it be optimized?
17 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
LIPID PURITY
Manufacturing
process
Quality of
starting
materials
The final GMP process needs to be scalable and
reproducible regarding yield and product quality
• Avoid reaction conditions which could lead
to isomerization
• Consider scalability from the very beginning
• Economy of scale, batch size definition
• Reduce number of steps, define GMP steps
18. Lipids purity
How can it be optimized?
18 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
LIPID PURITY
Purification
process
Purification steps must be scalable as well
• Use crystallization if possible
• Employ liquid/liquid extraction methods
• Avoid chromatography
• Convert chromatography into
a filtration over silica gel
Manufacturing
process
Quality of
starting
materials
19. 19 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
Synthetic lipids for drug development
What is needed from them?
Convenient and reproducible behavior in formulation: particle characteristics, stability and release profile
Consistent quality: In every step of the development
• Avoiding variability in the formulation development process
• Avoiding bridging toxicity studies
Saving resources
1
2
High purity.
20. 20 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
Synthetic lipids for drug development
What is needed from them?
Convenient and reproducible behavior in formulation: particle characteristics, stability and release profile
Consistent quality.
1
2
3
Good material characteristics: solubility, cristallinity, stability, flowability
influence on drug product GMP manufacturing process
High purity.
21. by courtesy of Büchi Labortechnik AG, Flawil, Switzerland
Lipids characteristics
How can they be optimized for an easier formulation process?
21 Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
The material’s surface can be enhanced by:
• Crystallization
• Lyophilization
• Spray drying
• Milling
• Solubility improvements
• Higher purity
• Enhanced stability
• Easier handling
characteristics
Enabling an easier
formulation
process
GMP relevantAmorphous vs. cristalline material
22. Lipid development
Process development behind lipid-based formulations
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201822
The right choice of raw material will reduce the risk during product development
PROCESS
RESEARCH
• Feasability
• Finding a cost-
effective route
• Initial amounts
manufacture
From months up
to 1 year
PROCESS
OPTIMIZATION
• Economics of process,
yield
• Adjust process to major
safety aspects
• Reliable analytical
methods
• Determination of critical
raw materials
• Initial product stability
studies
1-2 Years
PROCESS
MATURATION
• Process Validation
• Fix raw material
suppliers
• Quality risk analysis
• Final development
reports
• Stability studies for
intermediates
1-2 Years
24. Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201824
Regulatory aspects
Successful approval of a lipid-based drug product: which guidelines
should be taken into account?
“[…] The quality of lipid components, including
modified lipids (e.g., polyethylene glycol (PEG)
modified lipids), can affect the quality and
performance of the liposome drug product.
In cases of novel lipid components, the level of
detail provided in the submission should be
comparable to that for a drug substance.[…] see
ICH Q11 Development and Manufacture of Drug
Substances […]”
“[…] The quality and purity of the lipid
starting materials is essential for the later
quality of the drug product, therefore the
appropriate characterization and specification
of the lipid starting material is considered as
vital. […]
The level of information to be provided
with the relevant submission depends on
complexity of the excipients. […]”
“[…] Because the quality of liposome components
such as lipids can affect the quality of whole
liposome drug products, the quality of liposome
components should be appropriately controlled […]
In liposome drug products, lipid components […] and
molecules for liposome modification […] contribute to
an improvement in the in vivo stability,
pharmacokinetics, and intracellular behavior of the
active substance. Therefore, liposome components,
especially ligands (targeting moiety) and antibodies
that have a significant impact on the function of
the drug product, should be evaluated and
controlled to a greater extent than general
excipients to ensure their intended
properties.[…]”
25. Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201825
Regulatory aspects
Successful approval of a lipid-based drug product: which guidelines
should be taken into account?
FDA (April 2018)
Liposome Drug
Products: Chemistry,
Manufacturing, and
Controls; Human
Pharmacokinetics and
Bioavailability; and
Labeling
Documentation.
Guidance for Industry.
EMA (2013)
Reflection paper on the
data requirements for
intravenous liposomal
products developed with
reference to an innovator
liposomal product.
NIHS (2016)
Guideline for the
Development of
Liposome Drug
Products.
Site
implementation
ICH Q7 (API GMP) and other ICH Guidelines for Drug
Product and Drug Substance
26. Regulatory aspects, our approach.
Quality-related deliverables for synthetic lipids
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201826
27. Agenda
01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
27
28. Troubleshooting throughout the years
Are all those painpoints such a game changer?
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201828
Convenient and reproducible behavior in formulation: particle characteristics, stability and release
profile
Consistent quality.
1
2
High purity.
29. Purity & Consistent quality
Lipids developed with the application in mind: 1,3-GDO
• Very high isomeric purity
leading to defined and
controlled behaviour
Commercially available sample
Our high purity 1,3-GDO
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201829
Our high purity 1,3 & 1,2-GDO
• HPLC purity > 99% 1,3-GDO
• Highly defined
isomeric mixture (1,3 & 1,2)
30. Troubleshooting throughout the years
Are all those painpoints such a game changer?
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201830
Convenient and reproducible behavior in formulation: particle characteristics, stability and release profile.
Consistent quality.
1
2
3
Good material characteristics: solubility, cristallinity, stability, flowability
influence on drug product GMP manufacturing process
High purity.
31. Cristallinity & Stability
Lipids developed with the application in mind: DOPC
Benefits
• Crystalline material
• Enhanced stability: more
than 7 years at 25 °C / 60% rH
• Fast dissoluton rate
• Free flowing powder: easy
weighing & portioning
• Patented:
WO 2009/146779Crystalline DOPC
DMF type II
available
Amorphous DOPC
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201831
32. Lipids developed with the application in mind: (R,S)-DOTAP Cl
Cristallinity & Solubility…and definition
Crystalline DOTAP
Amorphous, wax-like DOTAP
DMF type II available
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201832
33. Solubility & Flowability
Lipids developed with the application in mind: DOPE
Outstanding solubility, easier handling in comparison to conventional, amorphous material
of wax-like consistency.
• Free flowing powder
• Fast and complete dissolution
• DOPE appeared as lumps, gel & foam
• limited solubility even after lyophilization
Powder DOPEWax-like DOPE
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201833
34. Agenda
01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
34
35. Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201835
Customized lipids
Enhanced liposome surface characteristics
PARTICLE SURFACE MODIFICATION
… through conjugated lipids with multiple functionalities
• Lipidic nanocarriers have demonstrated their suitability for the delivery of
different APIs.
• Certain needs remain still unmet
• STEALTH particles
• TARGETED particles
• Avoid accumulation in undesired locations, off-target side effects, toxicity
• Improve efficacy, reduce dose, improve economy of treatment
36. In-solution lipidation of peptides
Functional groups in the side chain lead to by-products
Incompatibility of solubility result in low yields
Expensive product
Hardly scalable process
Not suitable for GMP production
R RR R R R R RR R R R
SPPS
Resin
+ //
Customized Lipids: Our technology
Solid phase synthesis
Start with a lipidated amino acid
Uses large excess of reagents
Fast work-up simply by washing the resin
Can be automated
Cheaper products
Scalable process
Suitable for GMP production
many functionalties are present in
the side chains of the targeting peptide
aqueous solubility aprotic solubility
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201836
37. Agenda
01 Lipids: role in liposomal formulation
02 Synthetic lipids: process development
03 Regulatory aspects
04 Troubleshooting: examples
05 Enhanced surface characteristics
06 Overview of facilities and capabilities
37
38. 38
Where is our team located?
A global setup
Develop. & Launch Site
Schaffhausen
Global setup:
Additional European site: Arklow (Ireland)
US sites: St. Louis, Madison/Verona, Sheboygan (Wisconsin)
Risk mitigation (multiple production sites)
Production Site
Altdorf
Buchs
Contract Manufacturing
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 2018
We seek the needed
balance:
flexibility of
small teams
dedicated to Drug
Delivery
compounds and
the
global reach
with multiple
alternatives
39. A GMP manufacturing process that is scalable and reproducible in quality and yield facilitates
formulation and is a prerequisite for a consistent quality of the final product.
We specialize in cGMP manufacturing that follows the concepts of ICH Q7
Scale-up processes, from lab to kilo-lab to a production scale of up to 4,000 liters
The quality of lipids has a major impact on the performance of the liposomal formulation.
We offer a consistently high product quality from grams to tons.
High regulatory requirements that are close to the ones for APIs.
We support you with regulatory expertise and counsel from a dedicated team through all phases of
clinical development and commercialization
Covering all aspects of quality assurance and documentation
Successful drug development with synthetic lipids
Summary
Our high purity lipids and our expertise in cGMP manufacture and regulatory matters
facilitate your formulation and support you from development to commercialization.
Successful drug development with synthetic lipids: Critical aspects and strategies | May 24, 201839