Targteted Drug Delivery - Technologies and Applications

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This presentation which highlights the various technology innovations and developments in targeted drug delivery as well as maps its applications in different therapeutic segments was presented at the Novel Drug Delivery Systems and Clinical Trial Management 2013 Conference at Shangai, China.

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Targteted Drug Delivery - Technologies and Applications

  1. 1. Targeted Drug Delivery--Technologies and Applications Novel Drug Delivery Systems and Clinical Trial Management China 2013 Aiswariya Chidambaram 28th November 2013
  2. 2. Focus Points Importance of Targeted Drug Delivery Segmentation of Targeted Drug Delivery Technology Capability – Drug Delivery Carriers Technology Value Chain Targeted Drug Delivery – Key Innovations Targeted Drug Delivery – Drivers and Challenges Focus Points Key Technology Developments – Drug Delivery Carriers Life Cycle Analysis of Drug Delivery Systems Demand Side Analysis Potential Applications by Therapeutic Area Patent Distribution and Publishing Trends About Frost & Sullivan 2
  3. 3. Importance of Targeted Drug Delivery Targeted drug delivery is a system of drug delivery which increases the concentration of the drug in specific organs and tissues relative to the others. This helps improve the efficacy of the drug while reducing side effects. Cancer, autoimmune diseases, neurological disorders, pulmonary diseases, cardiovascular diseases and most other conditions that require effective, safe , specific targeting of certain receptors or direct delivery into the organ are attractive targets for targeted drug delivery. Criticality of Targeted Drug Delivery Physiological and Biological Barriers to Drug Delivery Mucous Barrier (lungs, nose, and cancer cells) Maximize precision Blood Brain Barrier (several factors lead to ineffective delivery to brain) Subcellular Targeting (cytosol, ER, nucleus targeting) Minimize toxicity Moderate release Size Exclusion (size barrier of different membranes) Ineffective Ligand Targeting (disease specific ligands on cell surface) Other Barriers (pH, osmotic potential, electric charge) Source: Frost & Sullivan 3
  4. 4. Segmentation of Targeted Drug Delivery • Oncology and neurology are the two most widely researched diseases for targeted drug delivery. • Delivery to the lungs, eyes, and nose are other areas of interest as targeting these organs is relatively difficult. Lipid-based Polymer-based Targeted Drug Delivery Carriers Inorganic Nanoparticles Magnetic Particles Nucleic Acid/ Peptidebased Cell-based • Liposomes nanoliposomes, stimuli responsive liposomes, and conjugated liposomes with other functional attributes are gaining attention from researchers. • Peptides and nucleic acids -Chimeric peptides are formed when a drug that is normally not transported through the BBB is conjugated to a brain drug-targeting vector. • Engineered polymers - PEG, polymeric nanomicelles and other co-polymers; polymers designed to respond to specific biological changes (such as pH, temperature, chemicals and so on) so that the drug load is released only upon stimulation. • Conjugates - antibody conjugated liposomal carriers, multifunctional nanocarriers, and other particles that form conjugates with the drug. Source: Frost & Sullivan D4C1-TI 4
  5. 5. Technology Capability–Lipid-Based Carriers Liposomes are widely used for insoluble drugs and advances in conjugation technologies is enabling them to be used as targeted delivery systems. For example, the biocompatibility and possible diversity with structures and compositions make them suitable for a number of targeted delivery applications. Conventional Liposomes Stimuli Responsive Liposomes Stealth Liposomes Targeted Liposomes Polymer Composite Liposomes Advantages Enables passive/active targeting Easy and rapid internalization Low immunogenicity Drawbacks Rapid degradation (uptake by RES(reticuloendothelial system) Poor scale up/need for extensive modifications Short shelf life Improves solubility/ bioavailability Drug protection/ Biocompatibility Liposomes will find more adoption with the development of nanoscale liposomes embedded with drug depot and polymer depots. Targeted liposomes and environment sensitive liposomes are the ones with maximum potential for tumors and neurodegenerative disorders, and a number of existing chemotherapeutics are being encapsulated in stimuli responsive liposomes. Source: Frost & Sullivan 5
  6. 6. Technology Capability–Polymer-Based Carriers Definition: Polymers have been widely used for sustained release of drugs, and adding functional targeting groups and other moeties has enabled them to be used as targeted delivery vehicles. A number of natural and synthetic polymers (degradable/nondegradable, hydrophilic/hydrophobic) with multifunctionalities are being produced for delivering drugs, while polymer composites with lipids and inorganic nanoparticles are also being developed. Stimuli Responsive Polymers Polymer Composites Multifunctional Polymers/ Co-Polymers Advantages Disadvantages Hypersensitivity (certain polymers) Wide range of drugs can be loaded Artificial Cells Cell mimicking properties (certain polymers) Biocompatibility Issues (synthetic) Easy Functionalization (surface modification) Controlled Release Kinetics Low Cost/Scalable Polymeric templates have been recently used to develop artificial cells, such as platelets and biomimetic vesicles for targeted delivery. Use of more natural polymers and polymer conjugated with other delivery methods will witness increased attention from drug delivery companies and pharmaceutical companies in the next 2 to 3 years. Source: Frost & Sullivan 6
  7. 7. Technology Capability–Inorganic Nanoparticles Definition: Inorganic nanoparticles comprise nanoscale particles made from silica. metals, metal hydroxides, carbon and so on. A number of multifunctional, inorganic nanoparticles are being developed for targeted drug delivery and imaging applications. Hybrid drug carriers combining stimuli sensitive hydrogels and inorganic nanoparticles, and conjugation of biomolecules to nanoparticles are areas of interest. Carbon Based Particles Gold Based (AuNPs) Advantages Optical Properties Silica/Alumina Nanoparticles Quantum Dots Metals/ Oxides/ Sulfides High Stability (over wide temperature and pH range) Drawbacks Toxicity Issues (LongTerm Safety ) Non Biodegradable (accumulation) Highly Tunable Evade RES Clearance Inorganic nanoparticles with multiple functionalities will prompt further research for development of effective cellular delivery systems. A few gold-based colloids and nanoshells are in clinical trials for cancer applications. Source: Frost & Sullivan 7
  8. 8. Technology Capability–Cell-Based Systems Definition: Cells have been found to act as potential drug delivery agents and they can be used to directly encapsulate the drug or used with nanoparticles for better pharmacokinetic properties, biocompatibility and higher drug loading capacity. The figure indicates some of the major types of cell-based delivery platforms in development. Dendritic Cells/ Tumor cells Engineered RBCs Advantages High Drug Loading Capacity Adjuvant Properties Microbial Ghosts and Viral Particles Genetically Engineered Stem Cells Sustained Release Biocompatible (except microbial ghosts) Scalable/Cost Effective Drawbacks Potential Immunogenicity (viral, bacterial particles) for non vaccine delivery Storage and Formulation Issues Maintaining integrity (RBCs, stem cells, macrophages) While viruses and virus-like particles (VLPs) have been widely used for vaccine delivery, the use of engineered bacterial ghosts (BG), engineered RBCs and stem cells is slowly moving to the clinic. Many of these cells and cell derived-particles are conjugated with other delivery strategies to improve the efficacy of treatment. Stem cell-based therapies provide a promising approach to the treatment of several diseases in humans, and extensive research on MSCs for targeted delivery is underway. Source: Frost & Sullivan 8
  9. 9. Technology Capability–Magnetic Particles Definition: These are micro-and nano-scale particles loaded or conjugated with drugs that get activated when exposed to an active magnetic field and release drug cargo at the target site. It is a highly controllable and effective form of drug targeting. In addition to delivery, these particles are apt for safe image guided drug delivery. Advantages Evade RES clearance Image guided delivery with MRI Biological Non-Biological Controlled Drug Release Highly targeted Organic Inorganic Drawbacks •Gradient loss for deep seated tissues •Accumulation of magnetic material at target site •Requirement for specialized manufacturing and QC system Organic magnetic carriers include magnetoliposomes (ferrofluids entrapped in the liposome core) and polymer magnetic particles. Iron oxide particles used directly with the drugs are inorganic magnetic particles. Incorporation of magnetically responsive materials into microspheres makes them susceptible to applied magnetic field, so that they are concentrated to the target site by the application of a magnetic field externally to that site. Ferriliposomes are other magnetic particles that could be used in combination with magnetic resonance imaging (MRI) for targeted drug delivery and also as theranostics. To improve biocompatibility and safety, biological magnetic particles, such as magnetobiosomes and engineered erythrocytes are being designed, and these are highly promising platforms. Source: Frost & Sullivan 9
  10. 10. Technology Capability–Nucleic Acid/Peptide Carriers Definition: In addition to peptides and nucleic acids being used as effective drugs, they are also being explored for targeting drugs. Cell penetrating peptides, polypeptide membranes, and DNA Nanorobots are some of the technologies with immense opportunities. Delivery of siRNA and other nucleic acid drugs, which is still a major challenge can be overcome with the use of peptide and aptamer-based targeting. DNA Origami(DNA Nanorobots) Peptide Conjugates Advantages Drawbacks Highly Targeted Mode of administration Biocompatible Aptamer Instability Biodegradable DNA/RNA Aptamers Cell Penetrating Peptides RNA/Peptide dual aptamer systems, polypeptide membranes are being developed for highly targeted delivery and being extensively researched. DNA Origami is a promising technology area which will enable ‘smart delivery’ to become a near-term reality. Overcoming aptamer instability via modifications is improving the clinical value of these systems. Source: Frost & Sullivan 10
  11. 11. Technology Value Chain Clinical Trials Conceptualiza tion of Drug Delivery Carriers Development of Drug Delivery System Scale Up and Large-Scale Manufacture (GMP) Regulatory Approval and Marketing Preformulation and Formulation Source: Frost & Sullivan 11
  12. 12. Key Innovations–Targeted Drug Delivery Multistage Nanocarriers (Leonardo Biosystems, TX, USA)  Multi-stage nanocarrier-based delivery platform  Customized mesoporous silica nanoparticles are used  Functions better than single-stage systems  For delivering siRNA, small molecule and imaging agents to cancer cells. Trojan Horse CNS Targeting Technology for Stroke (ArmaGen Technologies, CA, USA)  The TNF-alpha decoy receptor is engineered as a fusion protein with a BBB molecular Trojan horse (MTH).  The MTH is an engineered monoclonal antibody (MAb) against the BBB specific transferrin receptor (TfR).  Facilitates receptor mediated transport of the drug across the BBB.  Ensures effective and safe delivery of drugs to the brain. Thermally Responsive Liposomes, Lysolipid Thermally Sensitive Liposomes (LTSL) (Celsion Corporation, NJ, USA)  A patented liposomal tumor targeting delivery system  Delivers high concentrations of drug to target site (tumors) on exposure to local hypothermia.  Currently carrying out Phase 3 clinical trials (HEAT Study) for its lead candidate ThermoDox®, a formulation of doxorubicin for targeting HCC (hepatocellular carcinoma).  Expanded applications to develop formulations for other chemotherapeutics, such as docetaxel and carboplatin. Blood Brain Barrier Hyperthermia Drug release into tumor Source: Frost & Sullivan 12
  13. 13. Key Innovations–Targeted Drug Delivery (continued) IVECT Method , (Intezyne, Inc, FL, USA)  A multidisciplinary approach of drug delivery that has the potential to revolutionize cancer treatment.  Developed proprietary polymeric micelles that deliver drugs to tumors using a triggered release mechanism.  It is a highly tunable and cost-effective platform that can incorporate different targeting ligands.  Currently has four cancer chemotherapeutics in its pipeline: two lead programs in final preparation for IND submission and two others in earlier stages of preclinical development. Resealed Erythrocytes for targeted and Controlled Dryg delivery, Erydex (Erydel Spa, Italy)  An erythrocyte based drug delivery system for sustained release of drugs  Encapsulaties autologous red blood cells with glucocorticoid analoguedexamethasone sodium phosphate (Dex 21-P).  Used for treatment of chronic disorders such as cystic fibrosis, Ataxia Telangiectasia.  Ventured in to diagnostics and targeted drug delivery (EryTargeting) aimed at delivery of drug cargo only to targeted macrophages. Resealed RBCs for Targeted Drug Delivery to Tumors , GRASPA (ERYTech Pharma, France)  A tumor targeting therapeutic utilizing erythrocyte encapsulation technology.  Encapsulates a wide array of molecules such as peptides, proteins and small molecules for delivery.  Targets the tumor microenvironment using the encapsulated L-asparginase which depletes the circulating asparagine, a tumor growth factor thereby starving the tumor cells to death.  Currently tested in Acute Lumphoblastic Leukemia, Pancreatic cancer and Acute Myelod Leukemia. Source: Frost & Sullivan 13
  14. 14. Targeted Drug Delivery Systems–Drivers and Challenges • • • • • • • • • • • • Need for effective delivery of biologics Patenting opportunities for targeted therapies Quicker time to market/ease of approval Discovery of disease biomarkers Development of spatially/temporally controlled systems Advances in Nanotechnology Improve patient compliance Competition from medical devices for targeting High costs of several types of systems Potential long-term effects of nano particles Lack of multipronged/ combinatorial targeting approaches Poor funding scenario Source: Frost & Sullivan 14
  15. 15. Technology Development— Lipid Based Carriers 2 1 Below listed are some of the areas being developed by universities and research institutions • Dual response sensitive liposomes Company 5 0 Technology Development • Zwitterionic oligopeptide liposomes targeting mitochondria • Targeted liposomes for intracellular delivery 4 4.0 • Self assembling nanoemulsions • Liposomes fused on inorganic nanoparticles 3 Key 0-2  Less than 10 company developments 2 - 3.5  Between 10 and 20 company developments 3.5 - 5  More than 20 company developments Key Developments Silence Therapeutics (UK) Silence's siRNA delivery platform is based on the proprietary lipid moeities that embed siRNA into lipid-bi-layer particles. The siRNA is combined with Silence's developed lipid moieties containing cationic lipids, co-lipids and PEGylated lipids to form nanoscale structures. Celsion Corporation(NJ, USA) ThermoDox, the patented heat sensitive liposomal formulation of doxorubicin is in clinical trials for liver cancer and also being tested for a number of oncology indications. LiPlasome Pharma ApS (Denmark) The tumor targeting drug loaded lipid nanocarriers are designed to be susceptible to degradation by phospholipase A2 (PLA2), which is high in the cancer environment. The prodrug lipids are degraded by PLA2 and get converted to active drugs such as anticancer lysolipids and/or fatty acid drug derivatives. The degraded entities also enhance the permeability of the drugs across cancer cell membranes to deliver high doses of drug to the target site. Source: Frost & Sullivan 15
  16. 16. Technology Development—Polymer Based Carriers Below listed are some of the areas being developed by universities and research institutions • • • • • • Artificial Cells, Synthetic Platelets (Scripps Research Institute and Sanford-Burnham Institute) Dual Stimuli Responsive „Smart‟ Capsules (University of Melbourne) Protein Polymer Drug Conjugates Polymer drug depot in liposomal nanoparticles Multifunctional polymeric vesicles Stealth particles coated polymers, Amphiphilic Biodegradable Dendrimer-Like Star Polymers Company 3 2 1 4 4.5 5 0 Technology Development Key Developments PolyTherics Limited (UK) Flexible polymer platform for targeted, sustained delivery of biopharmaceuticals. GlycoPol TM is a targeting glycopolymer developed by attachment of saccharides a poly(methacrylate) backbone. Drugs can be attached to the backbone for targeted delivery. Intezyne, Inc. (FL, USA) IVECT Copolymer Micelles that are based on the IVECT Method offer a lost cost, modular and highly targeted delivery platform. It is highly tunable and can be used to deliver a number of drugs for varied indications. The lead candidate IT-141, has demonstrated significant activity against a diverse number of cancer cell lines. Another formulation IT-143, is the encapsulated daunorubicin, is being assessed for treatment of lung cancer, osteosarcoma, and ovarian cancer. Arrowhead Research Corporation (CA, USA) The company has a portfolio of in house developed and acquired targeted delivery platforms, of which DPCs(Dynamic polyconjugates) and RONDEL are based on polymer nanoparticles. Both are being explored for delivery of siRNA therapeutics. Source: Frost & Sullivan 16
  17. 17. Technology Development— Inorganic Nanoparticles 2 The adoption of inorganic nanoparticles is on the rise with the development of gold and silica nanoshells, nanowires, nanorods, and many more. They are emerging as an important and useful class of targeted entities that can be functionalized for specific needs. These inorganic nanoparticles can also be used for simultaneous imaging and delivery applications. Iron oxide, silica, gold, fullerenes, and carbon are the most widely researched materials. 3 1 4 3.5 5 0 Technology Development Below listed are some of the areas being developed by universities and research institutions • • • • • Mesoporous nanoparticles conjugated with peptides, antibodies, and other entities Stimuli sensitive nanoparticles Surfactant functionalized nanoparticles Aptamer gated nanoparticles Multistage nanoparticles Company Leonardo Biosystems (TX, USA) CytImmune Sciences Inc. Key Developments Multistage mesoporous silica nanoparticle based platform for spatiotemporally controlled drug release is attracting a lot of interest from investors and pharma companies. The system is undergoing optimization and enhancements. The technology is based on pegylated colloidal gold nanoparticles that can be used directly as drugs via tumor targeting molecules or can be used as carriers for cancer drugs. Source: Frost & Sullivan 17
  18. 18. Technology Development— Cell Based Carriers 2 The adoption of cell-based drug carriers is relatively low despite research being carried out for more than a decade. Modified RBCs form the majority of therapeutic carriers, while stem-cell based drug targeting is showing promising results in clinical trials. Microbial cells have been used for targeting vaccines and drugs for several years, and modified forms of these cells are now being developed for more effective and safe targeting. 3 1 4 2.5 0 5 Technology Development Below listed are some of the areas being developed by universities and research institutions • • • • • • Resealed erythrocytes/ Engineered erythrocytes with viral fusion proteins (for example, Erythro-magneto-HA virosomes) Engineered mesenchymal/ neural stem cells (for example, Silica nanorattle-drug anchored mesenchymal stem cells Virosomes/Bacterial ghosts for DNA vaccine and subunit vaccines iPSCs reprogrammed for organelle specific targeting Magnetotactic bacteria Prodrugs/Drugs bound to RBCs in circulation for long-term thrombosis treatment Company Key Developments EryDel SpA (Italy), EryTech Pharma (France) Erythrocyte loaded drugs for multiple disease areas currently in clinical development. These are being used for controlled delivery as well as targeting. Pevion Biotech AG (Germany) Proprietary virosome-based platform for targeted drug delivery and adjuvant activity for subunit vaccines. A number of vaccines are in advanced phases of clinical trials using their VLP technology, and has also been outlicensed to several pharma majors. Engene IC (Australia) EDV (Engene Delivery Vehicle) technology basically consists of inert bacterial cell derived nanoscale minicells conjugated with bispecific antibodies for highly targeted intracellular delivery of cancer drugs/siRNA. Immune stimulating properties enhance performance and the drugs using this platform are currently in clinical trials. Source: Frost & Sullivan 18
  19. 19. Technology Development—Magnetic Particles Below listed are some of the areas being developed by universities and research institutions • Bacterial magnetosomes from magnetotactic bacteria for drug targeting that can be functionally superior to artificial magnetic particles • Ocular magnetic drug targeting • Magnetic resealed erythrocytes • Enzyme and temperature responsive magnetic nanoparticles • Multifunctional nanoparticles for delivery and imaging (for example, multilayered nanorattles) Company 2 3 1 4 1.5 0 5 Technology Development Key Developments Vascular Magnetics, Inc, (PA, USA) Biodegradable, magnetic drug-loaded particles in combination with a magnetic targeting catheter and a magnetic field generating device that guides the particles to narrowed arteries in PAD (Peripheral Artery Disease). Nanobiomagnetics Inc. (SW R and D) (OK,USA) Magnetic vectored drug delivery using magnetically responsive therapeutic constructs. nanoTherics (UK) Magnetic transfection method MagnetofectionTM using DNA, siRNA coupled with magnetic nanoparticles to form a complex. Upon exposure to oscillating magnetic arrays, cells show uptake of the complex via rapid endocytosis. MagForce AG ( Berlin, Germany) Iron oxide nanoparticles with aminosilane coating that safely delivers drugs to tumors upon activation by their proprietary NanoActivator™ magnetic field. Source: Frost & Sullivan 19
  20. 20. Technology Development— Aptamers/Peptides/Nucleic Acids 2 3 Below listed are some of the areas being developed by universities and research institutions 1 • Peptide Dendrimer Conjugates • Engineered Oleosin–Self assembled to form Biomimetic Vesicles (University of Pennsylania) • Peptide and Aptamer Functionalized Nanoparticles; Aptamer nanoparticle Bioconjugates (PANOPTES project --novel peptide-based nanomaterials for ocular delivery) • Cell penetrating peptides (HIV TAT peptide, human calcitonin (hCT) hormone) • Carbohydrate mimetic peptides (UC, Berkeley) • Cell specific aptamers, peptide conjugated environment sensitive particles Company 4 2.5 5 0 Technology Development Key Developments Savara Inc. (TX, USA) Condensed nanoparticles of cell penetrating peptides and RNA therapeutics for targeted intracellular delivery. This is known as Nanonucleic technology used primarily for RNAi therapy. Artificial Cell Technologies, Inc (CT, USA) Layer by layer assembly of polypeptide artificial biofilm nanoparticles for developing synthetic vaccines. The synthetic nanoparticles incorporate immunogenic epitopes and give rise to “Artificial Virus” like structures that can be used for vaccine delivery. Aptagen, LLC (PA, USA) Aptabodies™ developed by the company are proprietary, functionalized aptamers that can be used for drug delivery or diagnostics. The company is a good collaborative partner for pharma/biotech companies for delivery of peptide therapeutics. Source: Frost & Sullivan 20
  21. 21. Market Position Life Cycle Analysis of Drug Delivery Systems Conventional Liposomes Stimuli sensitive liposomes Non Biodegradable Polymers Stimuli responsive polymers Conjugates/Hybrid Carriers/ Composite particles Engineered peptides/Aptamers Magnetic nanoparticles Functionalized Polymersomes Multistage/Multifunctional nanoparticles Inorganic nanorods, nanoshells, nanowires Biological Magnetic carriers Stem cell carriers Artificial cells/ Biomimetic particles DNA Nanorobots Development Growth Time Maturity Decline Source: Frost & Sullivan 21
  22. 22. Demand Side Analysis Applications Versatility ‘Smart’ Delivery Tunability End-User Requirements Market Exclusivity Cost Effectiveness and Scalability Biocompatibilit y Source: Frost & Sullivan 22
  23. 23. Potential Applications - Ocular InSite Vision Inc. Integral BioSystems Novagali Pharma Quark Pharmaceuticals Ocular Therapeutix • Challenges with regard to topical administration include poor access to the posterior portion of the eye, need for frequent doses, and poor controlled delivery. • Ophthalmic inserts developed for sustained release suffer from limitations, such as high costs and difficulty with insertion. • Liposomes and polymers - most widely used vehicles for ophthalmologic delivery. Targeting ligands that can cross the Blood Retinal Barrier(BRB) explored for a number of orphan retinal diseases. • Identification of ocular transporters and development of transporter targeted drugs modified with targeting ligands • Other commonly deployed delivery systems - functionalized nanoparticles, in situ forming gels, and colloidal dosage forms Source: Frost & Sullivan 23
  24. 24. Potential Applications - CNS Serina Therapeutics Inc. to-BBB ArmaGen Technologies VECT-HORUS Angiochem Inc. Lauren Sciences LLC Advectus Life Sciences Inc. • Systemically administered drugs have limited access across the BBB, while local administration is painful and challenging. • Most innovative drug developments for neurological disorders are biologicals, such as cell based therapy, RNAi or gene therapy. • Drug delivery systems that can cross the BBB (blood brain barriers) primarily include nanoparticles with targeting ligands and CNS targeting vectors that can bind to receptors on the BBB and cross the barrier in order to improve efficacy and increase safe therapeutic window. • Most of the research is in the basic research or pre-clinical stage • A number of partnerships and collaborations have been witnessed in the last 1 to 2 years. (For example, Genzyme Pharmaceuticals and Pharmidex Pharmaceutical Services Ltd) Source: Frost & Sullivan 24
  25. 25. Potential Applications - Oncology Celsion Corp BIND Biosciences LiPlasome Pharma Alchemia Savara Pharmaceuticals Silence Therapeutics Intezyne EnGene IC ERYTECH Pharma CytImmune Sciences Inc. Cerulean Pharma Inc. MagForce AG • Oncology is the most well penetrated area - more than 80% of targeted drug delivery activity is focused on safe and effective delivery of chemotherapeutics to cancerous cells. • Strategies based on differences in tumor cell metabolism, tumor microenvironment, and expression of receptors on tumor cell surfaces are being developed. • Most of them in clinical development are conjugates of polymers, liposomes, and inorganic nanoparticles, while magnetic targeting and imaging is also being explored by a few Participants. • Cell-based delivery systems that can trigger the immune system against cancer cells and multifunctional, multiple ligand targeting strategies will take predominance. Source: Frost & Sullivan 25
  26. 26. Potential Applications – Infectious Diseases Artificial Cell Technologies Inc. EryDel Xenetic Biosciences Arrowhead Research Corporation Pevion Biotech AG • High degree of noncompliance to therapy in a number of infectious diseases - led to development of targeted drugs that can specifically target the pathogen and associated pathways. • TB, HIV, and malaria are some of the highly researched areas. • Virosomes and bacterial ghosts have been widely used for vaccine delivery. • By developing different nanoparticle formulations with cell penetrating peptide conjugates, glycomimetic peptides, and other moieties, intracellular delivery to pathogens is becoming possible. Source: Frost & Sullivan 26
  27. 27. Potential Applications – Cardiovascular Vascular Magnetics Inc. Spheringenics Inc. BIND Biosciences • Eluting stents and cardiovascular implants – invasive drug delivery methods – strong need for non-invasive drug delivery systems. • Targeted immunoliposomes and biodegradable targeted polymeric particles are being developed for cardiac delivery and imaging. • Cell-selective targeted drug delivery - key research area for cardiovascular applications. • In atherosclerosis and other ischemic conditions, it is necessary to develop systems that can release drugs on sensing difference in shear stress or tissue injury. Source: Frost & Sullivan 27
  28. 28. Potential Applications – Pulmonary MannKind Corporation Pulmatrix BioParticle Technologies Liquidia Technologies Insmed Inc. • Most appropriate route for the treatment of asthma, cystic fibrosis and other pulmonary diseases such as tuberculosis, COPD, and lung cancer; explored for systemic diseases like diabetes. • Useful for developing inhalable vaccines, which hold huge market potential as a needle-free vaccination strategy for a number of respiratory infections. • Peptide and protein drugs are ideal for pulmonary delivery as they undergo degradation in the gastrointestinal (GI) tract). • Liquidia‟s PRINT (Particle Replication In Non-Wetting Templates) and MannKind‟s Technosphere have demonstrated success for pulmonary delivery. • Liposomes are the most extensively studied system for controlled drug delivery to the lungs. • Emerging areas of interest are magnetic aerosol droplets, bioresponsive nanoparticles, and co-polymers with better release profiles. Source: Frost & Sullivan 28
  29. 29. Potential Applications–Mapping Disease prevalence and criticality of delivery challenges Funding Research Initiatives Company Developments Cardiovascular Medium Medium Medium Low Ocular Medium Medium Low Low CNS High Medium Medium Medium Oncology High High High High Medium Low Low Low High High Medium Medium Infectious Diseases (Targeting Pathogens) Pulmonary Criteria Key - Funding Low  Less than $100 million federal and private funding Medium  Between $100 - $200 million federal and private funding High  More than $200 million federal and private funding Key – Research Initiatives Low  Less than 50 published research projects by academia Medium  Between 50 and 100 published research projects by academia High  More than 100 published research projects by academia Key – Company Developments Low  Less than 10 companies working on TDD Medium  Between 10 and 30 companies working on TDD 3.5 - 5  More than 30 companies working on TDD Source: Frost & Sullivan 29
  30. 30. Assignee-wise Patent Distribution Total Number of Patents/applications – 1080 (100 unique patent families). Source: Frost & Sullivan 30
  31. 31. Top Countries for Publishing Patent Applications US = United States EP = European Union WO = WIPO AU = Australia CA = Canada JP = Japan CN = China AT = Austria DE = Germany ES = Spain Most of the innovations in targeted drug delivery is happening in the US and European Union, which is clearly indicated by the number of patent applications published from these regions. Source: Frost & Sullivan 31
  32. 32. Patent Publishing Trends This chart shows the patenting trends in the last 4 years and the patenting trend has been quite stable. On an average 250 patents were published every year. Source: Frost & Sullivan 32
  33. 33. Questions
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