This presentation summarizes ACT's business characteristics and regenerative medicine programs. It discusses ACT's clinical trials for dry age-related macular degeneration, Stargardt's macular dystrophy, and myopic macular degeneration. Interim data from these trials show a clean safety profile, evidence of cell engraftment and persistence, and clinically significant visual acuity improvements in some patients. The presentation also outlines ACT's pipeline of ophthalmology programs including retinal pigment epithelial cells, retinal neural progenitors, and corneal programs. ACT has proprietary methods to derive various retinal cell types from pluripotent stem cells with high purity.
A User’s Perspective: ACMG Guidelines for CNVs in VSClinicalGolden Helix
Our last webcast introduced the newest features of VarSeq that will be included in our upcoming release. After a serious developmental effort, we are excited to announce one of these being the integration of ACMG Guidelines for CNVs!
VarSeq is not only the tertiary environment to filter through your imported SNVs and indels from any VCF file, but also includes robust and proven capabilities of CNV detection and clinical variant interpretation. With our upcoming release, users will be able to leverage an automated CNV ACMG classification filter that is paired with the CNV evaluation in the VSClinical interpretation hub. This webcast will expose attendees to literature reinforcing the quality of our CNV caller, as well as showing examples of CNV analysis to demonstrate how this tool can streamline the analysis process. In this webcast, we will cover:
Describing the new CNV guidelines and how Golden Helix tackles their complexity
Assessing CNV impact on Gene and Gene Dosage
Cited literature referencing the accuracy of VarSeq’s CNV calling
Product demonstrations of VSClinical’s CNV interpretation/classification and final report functionalities
Manually traversing the guidelines and classification process is fundamentally complex with multiple criteria considerations, collecting any necessary caveats, and bulk literature review just to name a few. It is our goal to simplify and streamline this process without losing user-control of final results or overlooking any crucial criteria components necessary for final classification. We hope you will join us to see how this is accomplished as we explore the ACMG CNV Guidelines within VSClinical from the user’s perspective!
PhoRank 2.0: Improved Phenotype-Based Gene Ranking in VarSeqGolden Helix
When performing variant analysis on whole exome or large gene panels, clinicians must sort through thousands of variants to determine which variants are most likely to be associated with the patient’s phenotypes. To assist with this process, we have implemented the PhoRank algorithm, which incorporates phenotypic associations to highlight the most relevant genes with potentially damaging variants. PhoRank 1.0 supports researchers leveraging all possible gene-disease associations by traversing multiple gene and pathway ontologies. Recent papers have demonstrated new techniques that have improved ranking performance in a clinical context. We have incorporated these new strategies into PhoRank 2.0: providing better ranking and improved computational performance for most clinically diagnostic and testing scenarios. Join us in this webinar as we cover:
Utility of gene ranking in genetic testing
Scenarios that warrant the use of PhoRank 2.0
New ranking strategies provided by recent papers
Benchmarks of PhoRank 2.0 on published datasets
Golden Helix provides a comprehensive solution for NGS testing labs to perform best practice guidelines such as ACMG and AMP. Our gene ranking methods provide a vital role in scaling tests to large gene panels and exomes. Please join us as we review the testing workflow and how this significant update to our gene ranking algorithm fits into the testing workflow.
VSClinical: First Commercial Product to Integrate the Updated ACMG Guidelines...Golden Helix
As CNV detection has become widely adopted as a component of NGS testing, the demand has grown for an interpreting framework specific to CNVs in the context of rare and inherited disorders. Earlier this year, a ClinGen working group in collaboration with ACMG, published new guidelines for the interpreting and reporting of CNVs detected by NGS. We at Golden Helix have been hard at work expanding VSClinical to incorporate these new scoring and evaluation criteria for the interpretation and clinical reporting of CNVs alongside small variants in our popular guided workflow for following the ACMG guidelines. Please join us in this webinar as we preview the upcoming release of VarSeq with this major development. We will cover the following:
- The new CNV guidelines, the specialized criteria for gains and losses, and how VSClinical simplifies the scoring and interpretation process
- How VS-CNV and VSClinical provides a complete solution for clinical detection and interpretation of CNVs for NGS gene panels and exomes
- The number of CNV specific data sources curated and integrated into the automatic recommendation system
- VSClinical’s integrated Word-based template system for designing custom clinical reports that include quality summary information, coverage at exon and gene level, interpreted variants and CNVs, and patient-level results
We are excited to release this major update to VSClinical, incorporating many small feature enhancements and user requests alongside with the integration of the CNV guidelines. Please join us in this webinar to see how CNVs can be an integrated part of the analysis workflow for your clinical NGS tests.
Building Secure Analysis and Storage Systems with Golden HelixGolden Helix
Genetic testing labs deal with personal data in categories with the highest level of security requirements: personal identity and medical records. Given the liability and risk associated with a breach of this secure information, it is not surprising that many labs and institutes that aggregate genomic data prefer if not require on-premise analysis and storage solutions.
Golden Helix is in a unique position to provide completely on-premise analysis solutions with a history of building analysis software from the ground-up on first principles and a focus on providing integrated, turn-key solutions. This allows for a licensing model based on training and supporting users, not tracking per-sample usage of cloud resources. As the regulatory environment around the world strengthens the privacy rights of individuals and the outcry around data breaches raises the stakes for building a secure system, we have developed a number of best practices for building secure, offline genomic analysis pipelines. Watch as we cover:
- Building a FASTQ to clinical reports pipeline behind a firewall
- On-premise analysis, warehouse and data servers independent of the internet
- Single sign-on based on local credential systems and without internet access
- Storage and network considerations for the analysis of patient-linked data
- Choose when to update and validate new pipelines, data sources and software versions
We hope you enjoy as we review the capabilities and best practices in building the most secure environment for hosting the analytics behind your precision medicine tests.
Exploring New Features and Clinical Reports in the ACMG Guideline WorkflowGolden Helix
For the past year, Golden Helix has been preparing a VarSeq release that includes ACMG Guidelines scoring and classification for not only single nucleotide variants but also copy number variants. In the past few months, our webcasts have introduced these guidelines and explored example CNVs that demonstrated the automated scoring of CNVs according to the new ACMG CNV Guidelines. However, there are many other new features and upgrades that have been incorporated into this VarSeq release. These upgrades have largely been driven by input from VarSeq users! That’s why I am very excited to explore these features together in the upcoming webcast “Exploring New Features and Clinical Reports in the ACMG Guideline Workflow”. In this webcast, we will walk through a workflow with SNVs and CNVs highlighting many of the new features and upgrades that will improve and streamline your ACMG workflow. Notably, we will see how these new features can also be incorporated into clinical reports.
Specifically, we will cover the following features:
New and improved ACMG Classifier algorithms
New and improved assessment catalogs for saving classifications and interpretations
New Project Options interface for creating patient evaluations
Now offering three new word-based clinical report templates
Inclusion of additional sections and features to the clinical reports
Enhanced report customization capability
Before accessing the clinical evidence associated with a specific variation, one must establish that the variant is likely to be a driver mutation, which generates functional changes that enhance tumor cell proliferation. In this webcast, we will discuss VSClinical’s capabilities for determining the oncogenicity of a variant. This will include a deep dive into our oncogenicity scoring system and a discussion of the various criteria used to distinguish driver mutations from benign variations and variants of uncertain significance
What you will learn in this webcast:
How to evaluate the oncogenicity of a variant in VSClinical
What evidence to consider when classifying LoF variants
How to examine the in-silico evidence for missense variants
How to evaluate a variant's rate of occurrence in somatic catalogs
Golden Helix's End-to-End Solution for Clinical LabsGolden Helix
In this webcast, we provide an overview of our complete end-to-end clinical stack. Initially, we walk through our powerful secondary analysis pipeline which allows you to call SNVs and CNVs. We then demonstrate how various types of CNVs are called and discuss metrics that express the confidence associated with each call.
From there, we show you our powerful tertiary analysis capabilities for gene panels, exome, and whole genome data. We show how our users can move seamlessly from the variant interpretation stage to a clinical report. Lastly, we demonstrate how our genetic data warehouse, VSWarehouse, can be used in the clinic. We also demonstrate various use cases and show how a comprehensive assessment catalog can be utilized to ensure consistent analysis across multiple labs.
We hope you enjoy our first presentation on Golden Helix's entire end-to-end solution for clinical labs!
Genome-wide association studies (GWAS) have been providing valuable insight to the genetics of common and complex diseases for many years. In this webcast we will walk through one possible workflow for completing GWAS in Golden Helix SNP & Variation Suite (SVS) with special attention paid to adjusting analysis for population stratification.
Efficient Application of NGS Family-Based AnalysisGolden Helix
Golden Helix is committed to providing next-gen sequencing solutions that are comprehensive and efficient. A significant portion of our user base processes pediatric cases or requires the ability to run family-based analyses that can be applied to all inheritance models in order to identify rare variants in Mendelian disorders and cancer. To increase diagnostic yield and reduce time to clinical results, these models and workflows must be easy to set up and used efficiently. Optimization can involve all the steps, including data import, workflow design (variant filtering), a detailed evaluation of variant impact and clinical relevance via ACMG guidelines and AMP guidelines, as well as providing a full clinical report.
VarSeq is the best tool to accomplish this task. VarSeq, our tertiary analytics platform, is designed to process all sizes of genomic data. It can filter through all variants in order to identify clinically relevant variants. The results are evaluated using automated and standardized guidelines. This webcast will demonstrate workflows tailored to different pedigree scenarios. It will also demonstrate VarSeq features designed to speed up analysis time while maintaining consistency with outcomes.
In this webcast, we will cover the following topics:
Explore various family-based project designs (includes trio and duo & extended families)
Discuss various inheritance models (compound het, de novo, dominant inherited, etc)
Briefly examine the ACMG guidelines relevant to inheritance and review an example trio clinical report
Demonstrate how VSPipeline can automate the creation of projects
Creating & Managing Reusable Gene Lists with VSClinicalGolden Helix
Golden Helix supports performing repeatable clinical workflows designed to meet the needs of your lab's clinical genetic tests. A critical component of any genetic test is the reporting of clinically significant genes and the ability to limit interpretations to a predefined set of test-specific genes. This webinar will cover the upcoming “Managed Gene List” feature of VarSeq and VSClinical which enables the defining and re-use of gene lists outside of individual product templates.
The community has stepped up to provide disease-specific gene lists that are validated and well-researched for specific genetic disorders. In this webinar, we will discuss how these gene lists can be incorporated into your VSClinical workflows and we will demonstrate how these gene lists can be modified to meet the specific needs of your lab.
Things you will learn:
How to manage gene lists in a single location and use them for filtering, annotation, and reporting
How to use the recently released ACMG Secondary Findings v3.0 list for reporting incidental findings
How to leverage the well-researched PanelApp knowledgebase to construct your gene lists based on different levels of evidence for a specific disease
How to define gene lists based on the specific phenotypes and disorders you are targeting with your tests
A User’s Perspective: ACMG Guidelines for CNVs in VSClinicalGolden Helix
Our last webcast introduced the newest features of VarSeq that will be included in our upcoming release. After a serious developmental effort, we are excited to announce one of these being the integration of ACMG Guidelines for CNVs!
VarSeq is not only the tertiary environment to filter through your imported SNVs and indels from any VCF file, but also includes robust and proven capabilities of CNV detection and clinical variant interpretation. With our upcoming release, users will be able to leverage an automated CNV ACMG classification filter that is paired with the CNV evaluation in the VSClinical interpretation hub. This webcast will expose attendees to literature reinforcing the quality of our CNV caller, as well as showing examples of CNV analysis to demonstrate how this tool can streamline the analysis process. In this webcast, we will cover:
Describing the new CNV guidelines and how Golden Helix tackles their complexity
Assessing CNV impact on Gene and Gene Dosage
Cited literature referencing the accuracy of VarSeq’s CNV calling
Product demonstrations of VSClinical’s CNV interpretation/classification and final report functionalities
Manually traversing the guidelines and classification process is fundamentally complex with multiple criteria considerations, collecting any necessary caveats, and bulk literature review just to name a few. It is our goal to simplify and streamline this process without losing user-control of final results or overlooking any crucial criteria components necessary for final classification. We hope you will join us to see how this is accomplished as we explore the ACMG CNV Guidelines within VSClinical from the user’s perspective!
PhoRank 2.0: Improved Phenotype-Based Gene Ranking in VarSeqGolden Helix
When performing variant analysis on whole exome or large gene panels, clinicians must sort through thousands of variants to determine which variants are most likely to be associated with the patient’s phenotypes. To assist with this process, we have implemented the PhoRank algorithm, which incorporates phenotypic associations to highlight the most relevant genes with potentially damaging variants. PhoRank 1.0 supports researchers leveraging all possible gene-disease associations by traversing multiple gene and pathway ontologies. Recent papers have demonstrated new techniques that have improved ranking performance in a clinical context. We have incorporated these new strategies into PhoRank 2.0: providing better ranking and improved computational performance for most clinically diagnostic and testing scenarios. Join us in this webinar as we cover:
Utility of gene ranking in genetic testing
Scenarios that warrant the use of PhoRank 2.0
New ranking strategies provided by recent papers
Benchmarks of PhoRank 2.0 on published datasets
Golden Helix provides a comprehensive solution for NGS testing labs to perform best practice guidelines such as ACMG and AMP. Our gene ranking methods provide a vital role in scaling tests to large gene panels and exomes. Please join us as we review the testing workflow and how this significant update to our gene ranking algorithm fits into the testing workflow.
VSClinical: First Commercial Product to Integrate the Updated ACMG Guidelines...Golden Helix
As CNV detection has become widely adopted as a component of NGS testing, the demand has grown for an interpreting framework specific to CNVs in the context of rare and inherited disorders. Earlier this year, a ClinGen working group in collaboration with ACMG, published new guidelines for the interpreting and reporting of CNVs detected by NGS. We at Golden Helix have been hard at work expanding VSClinical to incorporate these new scoring and evaluation criteria for the interpretation and clinical reporting of CNVs alongside small variants in our popular guided workflow for following the ACMG guidelines. Please join us in this webinar as we preview the upcoming release of VarSeq with this major development. We will cover the following:
- The new CNV guidelines, the specialized criteria for gains and losses, and how VSClinical simplifies the scoring and interpretation process
- How VS-CNV and VSClinical provides a complete solution for clinical detection and interpretation of CNVs for NGS gene panels and exomes
- The number of CNV specific data sources curated and integrated into the automatic recommendation system
- VSClinical’s integrated Word-based template system for designing custom clinical reports that include quality summary information, coverage at exon and gene level, interpreted variants and CNVs, and patient-level results
We are excited to release this major update to VSClinical, incorporating many small feature enhancements and user requests alongside with the integration of the CNV guidelines. Please join us in this webinar to see how CNVs can be an integrated part of the analysis workflow for your clinical NGS tests.
Building Secure Analysis and Storage Systems with Golden HelixGolden Helix
Genetic testing labs deal with personal data in categories with the highest level of security requirements: personal identity and medical records. Given the liability and risk associated with a breach of this secure information, it is not surprising that many labs and institutes that aggregate genomic data prefer if not require on-premise analysis and storage solutions.
Golden Helix is in a unique position to provide completely on-premise analysis solutions with a history of building analysis software from the ground-up on first principles and a focus on providing integrated, turn-key solutions. This allows for a licensing model based on training and supporting users, not tracking per-sample usage of cloud resources. As the regulatory environment around the world strengthens the privacy rights of individuals and the outcry around data breaches raises the stakes for building a secure system, we have developed a number of best practices for building secure, offline genomic analysis pipelines. Watch as we cover:
- Building a FASTQ to clinical reports pipeline behind a firewall
- On-premise analysis, warehouse and data servers independent of the internet
- Single sign-on based on local credential systems and without internet access
- Storage and network considerations for the analysis of patient-linked data
- Choose when to update and validate new pipelines, data sources and software versions
We hope you enjoy as we review the capabilities and best practices in building the most secure environment for hosting the analytics behind your precision medicine tests.
Exploring New Features and Clinical Reports in the ACMG Guideline WorkflowGolden Helix
For the past year, Golden Helix has been preparing a VarSeq release that includes ACMG Guidelines scoring and classification for not only single nucleotide variants but also copy number variants. In the past few months, our webcasts have introduced these guidelines and explored example CNVs that demonstrated the automated scoring of CNVs according to the new ACMG CNV Guidelines. However, there are many other new features and upgrades that have been incorporated into this VarSeq release. These upgrades have largely been driven by input from VarSeq users! That’s why I am very excited to explore these features together in the upcoming webcast “Exploring New Features and Clinical Reports in the ACMG Guideline Workflow”. In this webcast, we will walk through a workflow with SNVs and CNVs highlighting many of the new features and upgrades that will improve and streamline your ACMG workflow. Notably, we will see how these new features can also be incorporated into clinical reports.
Specifically, we will cover the following features:
New and improved ACMG Classifier algorithms
New and improved assessment catalogs for saving classifications and interpretations
New Project Options interface for creating patient evaluations
Now offering three new word-based clinical report templates
Inclusion of additional sections and features to the clinical reports
Enhanced report customization capability
Before accessing the clinical evidence associated with a specific variation, one must establish that the variant is likely to be a driver mutation, which generates functional changes that enhance tumor cell proliferation. In this webcast, we will discuss VSClinical’s capabilities for determining the oncogenicity of a variant. This will include a deep dive into our oncogenicity scoring system and a discussion of the various criteria used to distinguish driver mutations from benign variations and variants of uncertain significance
What you will learn in this webcast:
How to evaluate the oncogenicity of a variant in VSClinical
What evidence to consider when classifying LoF variants
How to examine the in-silico evidence for missense variants
How to evaluate a variant's rate of occurrence in somatic catalogs
Golden Helix's End-to-End Solution for Clinical LabsGolden Helix
In this webcast, we provide an overview of our complete end-to-end clinical stack. Initially, we walk through our powerful secondary analysis pipeline which allows you to call SNVs and CNVs. We then demonstrate how various types of CNVs are called and discuss metrics that express the confidence associated with each call.
From there, we show you our powerful tertiary analysis capabilities for gene panels, exome, and whole genome data. We show how our users can move seamlessly from the variant interpretation stage to a clinical report. Lastly, we demonstrate how our genetic data warehouse, VSWarehouse, can be used in the clinic. We also demonstrate various use cases and show how a comprehensive assessment catalog can be utilized to ensure consistent analysis across multiple labs.
We hope you enjoy our first presentation on Golden Helix's entire end-to-end solution for clinical labs!
Genome-wide association studies (GWAS) have been providing valuable insight to the genetics of common and complex diseases for many years. In this webcast we will walk through one possible workflow for completing GWAS in Golden Helix SNP & Variation Suite (SVS) with special attention paid to adjusting analysis for population stratification.
Efficient Application of NGS Family-Based AnalysisGolden Helix
Golden Helix is committed to providing next-gen sequencing solutions that are comprehensive and efficient. A significant portion of our user base processes pediatric cases or requires the ability to run family-based analyses that can be applied to all inheritance models in order to identify rare variants in Mendelian disorders and cancer. To increase diagnostic yield and reduce time to clinical results, these models and workflows must be easy to set up and used efficiently. Optimization can involve all the steps, including data import, workflow design (variant filtering), a detailed evaluation of variant impact and clinical relevance via ACMG guidelines and AMP guidelines, as well as providing a full clinical report.
VarSeq is the best tool to accomplish this task. VarSeq, our tertiary analytics platform, is designed to process all sizes of genomic data. It can filter through all variants in order to identify clinically relevant variants. The results are evaluated using automated and standardized guidelines. This webcast will demonstrate workflows tailored to different pedigree scenarios. It will also demonstrate VarSeq features designed to speed up analysis time while maintaining consistency with outcomes.
In this webcast, we will cover the following topics:
Explore various family-based project designs (includes trio and duo & extended families)
Discuss various inheritance models (compound het, de novo, dominant inherited, etc)
Briefly examine the ACMG guidelines relevant to inheritance and review an example trio clinical report
Demonstrate how VSPipeline can automate the creation of projects
Creating & Managing Reusable Gene Lists with VSClinicalGolden Helix
Golden Helix supports performing repeatable clinical workflows designed to meet the needs of your lab's clinical genetic tests. A critical component of any genetic test is the reporting of clinically significant genes and the ability to limit interpretations to a predefined set of test-specific genes. This webinar will cover the upcoming “Managed Gene List” feature of VarSeq and VSClinical which enables the defining and re-use of gene lists outside of individual product templates.
The community has stepped up to provide disease-specific gene lists that are validated and well-researched for specific genetic disorders. In this webinar, we will discuss how these gene lists can be incorporated into your VSClinical workflows and we will demonstrate how these gene lists can be modified to meet the specific needs of your lab.
Things you will learn:
How to manage gene lists in a single location and use them for filtering, annotation, and reporting
How to use the recently released ACMG Secondary Findings v3.0 list for reporting incidental findings
How to leverage the well-researched PanelApp knowledgebase to construct your gene lists based on different levels of evidence for a specific disease
How to define gene lists based on the specific phenotypes and disorders you are targeting with your tests
2014 MFDS Global Biopharmaceutical Forum: "Clinical and Preclinical Researches in Human Pluripotent Stem Cells"
Human ES Cell Product – RPE Program for Blindness
(CHA & ACT-Ocata)
2014 MFDS Meeting - 2014. 7. 9~10.
Hyung Min Chung
Konkuk University, College of Medicine, Seoul, Korea
What are stem cells? This presentation provides an overview of multiple different stem cells including embryonic stem cells, mesenchymal stem cells, cancer stem cells, induced pluripotent stem cells, hematopoietic stem cells and neural stem cells.
$ATHX @athersys Corporate Overview - Investor Presentation (dated 08-10-18 / 32 slides) - http://www.athersys.com/static-files/df957fcc-48c5-46aa-8ae4-c254e18e05ee As found by "imz72", with growing, insightful shareholder's comments at reddit - https://redd.it/98b693 #MultiStem #Stroke Company Website -http://www.athersys.com/ As posted at Twitter - https://twitter.com/twenty2John/status/1030883310082318337
9/11/2017 Revised Bocce Courts 4 HB (Huntington Beach). This report, given in book form, to each of the seven members of the HB City Council (including, Mayor), and two copies to David Dominguez - Facilities, Development & Concessions Manager (HB).
Special Note: All links should be CLICKABLE (Tap link twice), EXCEPT links on first (3) pages. Links, on first (3) pages of this report are not accessible.
Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies
Published Online: 15 October 2014
Including Comment By, Anthony Atala
Q2 2015 ARM - Alliance for Regenerative Medicine
Quarterly Data Report: Q2 2015 provides an in-depth look at regenerative medicine and advanced therapies sector trends and metrics compiled from more than 580 leading therapeutic companies worldwide.
Part 2 Of 2 Comment/Reply on ACT/Ocata Lancet Report (Oct. 2014)
Reply by, *Steven D Schwartz, Eddy Anglade, Robert Lanza, on behalf of the Ocata
Macular Disease Investigator Group
schwartz@jsei.ucla.edu
Jules Stein Eye Institute Retina Division, and David
Geffen School of Medicine, University of California,
Los Angeles, CA 90095, USA (SDS); and Ocata
Therapeutics Inc, Marlborough, MA, USA (EA, RL)
Source Material: http://www.thelancet.com/pdfs/journals/lancet/PIIS0140-6736(15)61203-X.pdf
March 23, 2015 Sheraton Silver Spring Hotel - Silver Spring, Maryland USA
Bioassays 2015: Scientific Approaches & Regulatory Strategies
Bioassay Development for Human Stem Cell-derived Retinal Pigment Epithelium: Progress and Challenges
Irina Klimanskaya, Ocata Therapeutics, Inc., Marlborough, MA USA
BIOASSAYS 2015: SCIENTIFIC PROGRAM
March 23 - 24, 2015 at the Sheraton Hotel in Silver Spring, Maryland.
Irina Klimanskaya, Ocata Therapeutics, Inc., Marlborough, MA USA - Monday March 23, 2015 10:05 – 10:30 Bioassay Development for Human Stem Cell-derived Retinal Pigment Epithelium: Progress and Challenges
Acute scrotum is a general term referring to an emergency condition affecting the contents or the wall of the scrotum.
There are a number of conditions that present acutely, predominantly with pain and/or swelling
A careful and detailed history and examination, and in some cases, investigations allow differentiation between these diagnoses. A prompt diagnosis is essential as the patient may require urgent surgical intervention
Testicular torsion refers to twisting of the spermatic cord, causing ischaemia of the testicle.
Testicular torsion results from inadequate fixation of the testis to the tunica vaginalis producing ischemia from reduced arterial inflow and venous outflow obstruction.
The prevalence of testicular torsion in adult patients hospitalized with acute scrotal pain is approximately 25 to 50 percent
Report Back from SGO 2024: What’s the Latest in Cervical Cancer?bkling
Are you curious about what’s new in cervical cancer research or unsure what the findings mean? Join Dr. Emily Ko, a gynecologic oncologist at Penn Medicine, to learn about the latest updates from the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer. Dr. Ko will discuss what the research presented at the conference means for you and answer your questions about the new developments.
Ethanol (CH3CH2OH), or beverage alcohol, is a two-carbon alcohol
that is rapidly distributed in the body and brain. Ethanol alters many
neurochemical systems and has rewarding and addictive properties. It
is the oldest recreational drug and likely contributes to more morbidity,
mortality, and public health costs than all illicit drugs combined. The
5th edition of the Diagnostic and Statistical Manual of Mental Disorders
(DSM-5) integrates alcohol abuse and alcohol dependence into a single
disorder called alcohol use disorder (AUD), with mild, moderate,
and severe subclassifications (American Psychiatric Association, 2013).
In the DSM-5, all types of substance abuse and dependence have been
combined into a single substance use disorder (SUD) on a continuum
from mild to severe. A diagnosis of AUD requires that at least two of
the 11 DSM-5 behaviors be present within a 12-month period (mild
AUD: 2–3 criteria; moderate AUD: 4–5 criteria; severe AUD: 6–11 criteria).
The four main behavioral effects of AUD are impaired control over
drinking, negative social consequences, risky use, and altered physiological
effects (tolerance, withdrawal). This chapter presents an overview
of the prevalence and harmful consequences of AUD in the U.S.,
the systemic nature of the disease, neurocircuitry and stages of AUD,
comorbidities, fetal alcohol spectrum disorders, genetic risk factors, and
pharmacotherapies for AUD.
NVBDCP.pptx Nation vector borne disease control programSapna Thakur
NVBDCP was launched in 2003-2004 . Vector-Borne Disease: Disease that results from an infection transmitted to humans and other animals by blood-feeding arthropods, such as mosquitoes, ticks, and fleas. Examples of vector-borne diseases include Dengue fever, West Nile Virus, Lyme disease, and malaria.
Knee anatomy and clinical tests 2024.pdfvimalpl1234
This includes all relevant anatomy and clinical tests compiled from standard textbooks, Campbell,netter etc..It is comprehensive and best suited for orthopaedicians and orthopaedic residents.
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We specializes in exporting high quality Research chemical, medical intermediate, Pharmaceutical chemicals and so on. Products are exported to USA, Canada, France, Korea, Japan,Russia, Southeast Asia and other countries.
Prix Galien International 2024 Forum ProgramLevi Shapiro
June 20, 2024, Prix Galien International and Jerusalem Ethics Forum in ROME. Detailed agenda including panels:
- ADVANCES IN CARDIOLOGY: A NEW PARADIGM IS COMING
- WOMEN’S HEALTH: FERTILITY PRESERVATION
- WHAT’S NEW IN THE TREATMENT OF INFECTIOUS,
ONCOLOGICAL AND INFLAMMATORY SKIN DISEASES?
- ARTIFICIAL INTELLIGENCE AND ETHICS
- GENE THERAPY
- BEYOND BORDERS: GLOBAL INITIATIVES FOR DEMOCRATIZING LIFE SCIENCE TECHNOLOGIES AND PROMOTING ACCESS TO HEALTHCARE
- ETHICAL CHALLENGES IN LIFE SCIENCES
- Prix Galien International Awards Ceremony
2. 2
This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell
Technology Inc.”, or “the Company”) salient business characteristics.
The information herein contains “forward-looking statements” as defined under the federal
securities laws. Actual results could vary materially. Factors that could cause actual results
to vary materially are described in our filings with the Securities and Exchange Commission.
You should pay particular attention to the “risk factors” contained in documents we file from
time to time with the Securities and Exchange Commission. The risks identified therein, as
well as others not identified by the Company, could cause the Company’s actual results to
differ materially from those expressed in any forward-looking statements. Ropes Gray
Cautionary Statement Concerning Forward-Looking Statements
6. 6
Life Support to Photoreceptors
Provides nutrients and growth factors
• photoreceptors see no blood
Recycles Vitamin A
• maintains photoreceptor excitability
Detoxifies photoreceptor layer
Maintains Bruch’s Membrane
• natural antiangiogenic barrier
• immune privilege of retina
Absorbs stray light / protects from UV
RPE Layer has
multiple
critical roles
in the
health and
function
of photoreceptors and
the retina as a whole.
7. 7
Life Support to Photoreceptors
Failure of RPE cells
results in many
degenerative diseases
Stargardt’s disease (SMD)
Myopic Macular Dystrophy (MMD)
Age-related macular degeneration (AMD)
8. Age-Related Macular Degeneration will Soon Take on Aspects of an Epidemic
Data from http://www.nei.nih.gov/eyedata/ and
U.S. Census Bureau Publication “65+ in the United States”, P23-209
Exponential rise in
prevalence and
incidence rates with age.
Prevalence rates of late
AMD quadruple per
decade
40%
30%
20%
10%
50-5940-49 60-69 70-79 80+
Age
Intermediate AMD
Late AMD
%Prevalence(U.S.)
8
The projected number of people worldwide with age-
related macular degeneration in 2020 is 196 million,
increasing to 288 million in 2040 - Wong et al. Lancet January 2014
9. Dissociated RPE Cells can be injected into the subretinal
space and will rebuild functional RPE monolayer where
native RPE is compromised or lost.
• Simple Handling
• Optimized for large scale and centralized manufacturing
• Small Dose Size translates into ACT’s ability to manufacture
50,000-100,000 per year in existing facilities.
Therapeutic Thesis
9
Product Cold Chain
is Easily Scaled for
Global Sales
• RPE layer is surgically accessible -
- least invasive means to locally deliver cells
• Adult RPE translocation studies included
dissociated cell formats
• hESC-derived RPE supported by both In Vitro
and In Vivo Studies at ACT and OHSU
10. Cell Therapy for RPE, Achievable by a Small Company
10
Small dosage size
• less than 200K cells
Relatively Immune-privileged site
• minimal immunosuppression
Ease of administration
• no separate device approval
Unique measuring and observation environment
• measurable anatomical and functional endpoints
11. January 2013: FDA approved additional 4 subject “better vision”
cohorts in each trial.
For Cohort 2a – can enroll subjects with vision as
good as 20/100.
Cohort 1
50K Cells
Cohort 2
100K Cells
Cohort 3
150K Cells
Cohort 4
200K Cells
Cohort 2a
100K Cells
First Treatments informed a more aggressive strategy to treat “better vision” cohort, could
lead to broader label and/or earlier approval
11
4 Approved Trials – 3 U.S. and 1 U.K.
Initial Trial Design is Ascending Dosage Safety Study
12 Subjects – Four Cohorts of 3 subjects each
Inclusion Criteria
BCVA: 20/800 or worse
Inclusion Criteria
BCVA: 20/400 or worse
Inclusion Criteria
BCVA: 20/100 or worse
Inclusion Criteria
BCVA: 20/400 or worse
Inclusion Criteria
BCVA: 20/400 or worse
12. IND
Approved
50% Patient
Enrollment
100% Patient
Enrollment
U.S. – Dry AMD
U.S. – SMD
U.K. – SMD
U.S. – MMD
12/16 patients treated
12/16 patients treated
10/12 patients treated
Enrolling – 12 patients total
Current Status of P1 Trials enables Planning for P2
34 Patients Treated to Date
12
Early Hypothesis Developed for PII,
Vetted with Investigators, Dialoging with MHRA and FDA
13. Jules Stein
(UCLA)
Mass
Eye & Ear
Infirmary
Wills Eye
Institute
Bascom
Palmer Eye
Institute
Moorfields
Eye
Hospital
Edinburgh
Royal
Infirmary
Clinical Trials being led by World Leaders in Ophthalmology
13
World Renowned Leadership to help us navigate the Clinical
Path and Ultimately Support Market Launch
14. Delivery of Cells Builds on Common Surgery
14
Procedure:
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation
• Subretinal hESC-derived RPE cells injection
• Bleb Confirmation
• Day Surgery/Sedation only
Vitrectomy is the 3rd Most Frequently
Performed Ophthalmic Surgery; 1.1m
globally, 300k in US, per year
15. Elapsed Time Since Transplant – as May 2014
15
US
Dry AMD
US
SMD
UK
SMD
Interim Data Analysis
Interim Data Analysis
2 years 3 years1 year
* US AMD & SMD Cohort 2a
subjects range from 1-12 months
post-transplant
Cohort
2a
16. Overview of Interim Data
16
Manuscript detailing interim data under review at impactful journal
Co-authored by participating surgeons and company
o Clean safety profile
o Evidence of engraftment and persistence of transplanted RPE cells
o Persisting Improvement of Visual Acuity for many patients
A number of patients with clinically significant improvements in BCVA
AMD-207 improved 20/400 to 20/40
• Improvement has persisted through follow-up visits (> 1 year)
SMD-001 improved from HM to 20/500
• Improvement (with further gain) has persisted through follow-up visits (>2 year)
Based on advice from all 5 clinical trial sites and participating surgeons, as
well as input from regulators, we are advancing to phase 2 studies
› Active dialog with MHRA and FDA
› Anticipate P2 commencement in 2H2014
Exemplars of
Patients With
Improved
Visual Acuity
17. Expanding RPE Program to Address an Additional Leading Cause of Blindness
17
Myopia creates a higher risk of permanent vision loss due
to Myopic Macular Degeneration (MMD)
• Severe near-sightedness causes elongation of the eyeball --
which can cause fissures in RPE layer.
As the cause of legal blindness, MMD is ranked:
• 7th in the United Sates
• 4th in Hong Kong
• 2nd in mainland China and Japan
Jules Stein Eye (UCLA) and ACT to Initiate Phase I/II Study
18. Second Generation RPE Cell Therapy Products
By engineering the master stem cell bank used to manufacture RPE
cells, the transplanted RPE cells can express
• Anti-angiogeneic agents
Reduce occurrence of choroidal neovascularization (wet AMD).
• Complement factor D, Factor C5 and/or Factor C3 Inhibitors
Activation of alternative complement pathway implicated in disease
progression for certain patients
• Anti b-amyloid agents
Drusen deposits resemble amyloid deposits.
• Anti-Inflammatory agents
IL-1, IL-2, IL-3, and TNF-α antagonists
Recombinant Lipocortin – a potent anti-inflammatory protein
18
19. Intellectual Property – RPE Program
Dominant Patent Positions in Major Markets
for Platform Technology and Treatments
• Broad Coverage for Manufacturing RPE Cells
• Broad protection of pharmaceutical preparations of RPE cells
Covers RPE cell suspensions for injection
Covers scaffolded RPE layers for implantation
Covers polarized monolayers of cells
• Covers RPE Cells derived from any pluripotent stem cell source
- From hESC to iPS cells to pluripotent stem cells yet to be invented
19
First Mover and First-in-Clinic Efforts
Translate into Broad First-to-File Patents
Every practical
formulation format for
use in human patients
22. 22
Pluripotent
Stem Cell
Eye Field
Stem Cell
RPE
Photoreceptor
Ganglion
Proprietary Methods for Deriving Various Cell Types of the Retina
Purity of Resulting
Cell Preparations
Approaches 100%
23. 23
Cell lines
Percentage of PAX6
+ neural stem cells
Percentage of Nestin
+ neural stem cells
ES lines (N=3) 92%-98% 95%-99%
ES lines, blastomere technology
(N=2)
94%-99% 95%-99%
iPS lines, Episomal Vector (N=4) 90%-99.6% 96%-100%
iPS lines, mRNA (N=2) 92%-98% 96%-100%
• Evaluated in multiple hESC and iPS cells lines
• Highly synchronized culture
Highly Reproducible with Various Pluripotent Stem Cell Sources
24. 24
Neurosensory Retina – Photoreceptor Replacement
We have demonstrated that
subretinally injected hESC-
and iPSC-derived photoreceptor progenitors can
migrate to correct anatomical site in retina, differentiate,
and functionally rescue vision.
Carrying out disease model POC studies for demonstrating utility in
treatment of:
• Late stage macular degeneration
• Retinitis Pigmentosa (RP)
• Night Blindness
Unique photoreceptor progenitor
(“PhRP-x”) maintains plasticity to form
both rods and cones
25. 25
When delivered systemically
• Scotopic (shown) and Phototopic ERG
preservation of rod and cone function
• ERG recovery is cell-dependent (CSA
removal reverses ERG recovery)
• Also observed preservation of ONL
thickness relative to control.
Only the PhRP-x progenitors have
paracrine neuroprotective activity
– earlier stage high purity retinal
progenitors do not.
Neurosensory Retina – Neuroprotection
A differentiated functional phenotype
PhRP-x are photoreceptor progenitors which:
• form both rods and cones
• functionally integrate in ONL
• secrete a neuroprotective agent(s)
• have phagocytic activity for OS
26. Edward Myles – Interim President, CFO and EVP of Corp
Development
Dr. Matthew Vincent, Ph.D. – Dir., Business Development
Dr. Robert Lanza, MD – Chief Scientific Officer
Dr. Irina Klimanskaya, Ph.D. – Dir., Stem Cell Biology
Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research
Eddy Anglade, M.D. – EVP, Clinical Development
Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing
Proven business leaders who can
develop and implement corporate
strategy and monetize assets to
maximize shareholder value
World-renowned scientific thought
leaders pushing the cutting edge of
science to develop important
therapies
Deep experience in clinical development
programs for ophthalmology drug products from
early through late-and post-marketing stages
GMP manufacturing to ensure the highest quality
products are delivered to our patients
An Experienced and Dedicated Management Team
26
27. Michael Heffernan, Chairman CEO – Collegium Pharmaceuticals
Robert S. Langer, Sc.D. Institute Professor, MIT
Zohar Loshitzer
CEO – Presbia, Inc., & Principal in
Orchard Capital
Greg Perry EVP & CFO – Eleven Biotherapeutics
Alan C. Shapiro
Finance Professor and Chairman of the
Department of Finance and Business
Economics (retired) – University of
Southern California
A World-Class Board of Directors
27