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This report provides a comprehensive overview of the size of the regenerative medicine market, segmentation of the market (stem cells, tissue engineering and CAR-T therapy), key players and the vast potential of therapies that are in clinical trials.  Kelly Scientific analysis indicates that the global regenerative medicine market was worth $18.9 billion in 2016 and will grow to over $53.7 billion by 2021. Within this market, the stem cell industry will grow significantly. Regenerative medicine's main objective is to heal and replace organs/cells that have been damaged by age, trauma or disease. Congenital defects can also be addressed with regenerative medicine. Therefore, it's market encompasses dermal wounds, cardiovascular disease, specific cancer types and organ replacement. To that end, regenerative medicine is a broader field and manipulates the body's immune system and regeneration potential to achieve its requirement. Financially speaking, investment into this space is dominated by grants, private investors and publicly traded stocks. Looking forward, the regenerative medicine market is promising for a number of robust reasons including: Key Topics Covered: 1.0 Report Synopsis 2.0 Introduction 2.1 Gurdon and Yamanaka Share the Nobel Prize 2.2 Stem Cell Clinical Trials: Initiated in 2010 2.3 Types of Stem Cells 2.4 Adult (Tissue) Stem Cells 2.5 Pluripotent Stem Cells 2.6 Somatic Cell Nuclear Transfer (SCNT) 2.7 Induced pluripotent Stem Cells (iPSC) 2.8 Mesenchymal Cells 2.9 Hematopoietic Stem and Progenitor Cells 2.10 Umbilical Cord Stem Cells 2.11 Heart Stem Cells 2.12 Mammary Stem Cells 2.13 Neural Stem Cells 2.14 Stem Cell Applications in Retinal Repair 2.15 Liver Stem Cells 2.16 Gut Stem Cells 2.16 Pancreatic Stem Cells 2.17 Epidermal Stem Cells 3.0 Stem Cells and Clinical Trials 3.1 Introduction 3.2 Pluripotent Stem Cells 3.3 Limbal Stem Cells 3.4 Neural Stem Cells 3.5 Endothelial Stem or Progenitor Cells 3.6 Placental Stem Cells 3.7 Why Do Stem Cell Clinical Trials Fail? 3.8 What is the Future of Stem Cell Trials? 3.9 Cutting Edge Stem Cell Clinical Trials 3.10 Ocata Therapeutics Current Stem Cell Trials 3.11 CHA Biotech Current Stem Cell Trials 3.12 Pfizer Current Stem Cell Trials 3.13 GSK Current Stem Cell Trials 3.14 Bayer Current Stem Cell Trials 3.15 Mesoblast International Current Stem Cell Trials 3.16 Millennium Pharmaceutical Current Stem Cell Trial 3.17 AstraZeneca Current Stem Cell Trials 3.18 Merck Current Stem Cell Trials 3.19 Chimerix Current Stem Cell Trials 3.20 Eisai Current Stem Cell Trials 3.21 SanBio Current Stem Cell Trials 3.22 Celgene Current Stem Cell Trials 3.23 StemCells Current Stem Cell Trials 3.24 Genzyme (Sanofi) Current Stem Cell Trials 3.25 Teva Current Stem Cell Trials 3.26 MedImmune Current Stem Cell Trials 3.27 Janssen Current Stem Cell Trials 3.28 Seattle Genetics Current Stem Cell Trials 3.29 Baxter Healthcare Current Stem Cell Trials 3.30 InCyte Corp Current Stem Cell Trials 4.0 Stem Cells, Disruptive Technology, Drug Discovery & Toxicity Testing 4.1 Introduction 4.2 Case Study: Genentech and Stem Cell Technology 4.3 3D Sphere Culture Systems 4.4 Stem Cells and High Throughput Screening 4.5 Genetic Instability of Stem Cells 4.6 Comprehensive in Vitro Proarrhythmia Assay (CiPA) & Cardiomyocytes 4.8 Coupling Precise Genome Editing (PGE) and iPSCs 4.9 Stem Cells & Toxicity Testing 4.10 Stem Cell Disease Models 4.11 Defining Human Disease Specific Phenotypes 4.12 Advantages of Stem Cell Derived Cells & Tissues for Drug Screening 5.0 Stem Cell Biomarkers 5.1 Pluripotent Stem Cell Biomarkers 5.2 Mesenchymal Stem Cell Biomarkers 5.3 Neural Stem Cell Biomarkers 5.4 Hematopoietic Stem Cell Biomarkers 6.0 Manufacturing Stem Cell Products 6.1 Manufacturing Strategies For Stem Cell Products 6.2 BioProcess Economics for Stem Cell Products 6.3 Capital Investment 6.4 Cost of Goods 6.5 Bioprocess Economic Drivers & Strategies 6.6 hPSC Expansion & Differentiation using Planar Technology 6.7 hPSC Expansion using 3D Culture 6.8 Microcarrier Systems 6.9 Aggregate Suspension 6.10 Bioreactor Based Differentiation Strategy 6.11 Integrated hPSC Bioprocess Strategy 6.12 GMP Regulations and Stem Cell Products 7.0 Investment & Funding 7.1 What do Investors Want from Cell & Gene Therapy Companies? 7.2 What Makes a Good Investment? 7.3 What Types of Companies do Not Get Investment? 7.4 Global Funding 7.5 Cell & Gene Therapy Investment Going Forward 7.6 What Cell & Gene Companies are the Most Promising in 2017? 7.7 Insights into Investing in Cell and Gene Therapy Companies 8.0 Regenerative Medicine Market Analysis & Forecast to 2021 8.1 Market Overview 8.2 Global Frequency Analysis 8.3 Economics of Regenerative Medicine 8.4 Market Applications & Opportunities for Regenerative Therapies 8.5 Global Financial Landscape 8.6 Regenerative Medicine Clinical Trial Statistics 8.7 Regenerative Medicine Market Forecast to 2021 8.8 Regenerative Medicine Geographic Analysis and Forecast to 2021 8.9 Regenerative Medicine Geographical Location of Companies 8.10 Regenerative Medicine Technology Breakdown of Companies 8.11 Commercially Available Regenerative Medicine Products 8.12 Major Regenerative Medicine Milestones 9.0 Stem Cell Market Analysis & Forecast to 2021 9.1 Autologous & Allogenic Cell Market Analysis 9.2 Stem Cell Market by Geography 9.3 Stem Cell Market Forecast by Therapeutic Indication 9.4 Stem Cell Reagent Market Trends 10.0 Tissue Engineering Tissue Engineering Market Analysis and Forecast to 2021 10.1 Geographical Analysis and Forecast to 2021 10.2 Geographical Analysis by Company Share 10.3 Tissue Engineering Clinical Indication Analysis & Forecast to 2021 11.0 Biobanking Market Analysis 11.1 Increasing Number of Cord Blood Banks Globally 11.2 Global Biobanking Company Sector Analysis & Breakdown 11.3 Allogenic Versus Autologous Transplant Frequency 11.4 Biobanking Market Analysis & Forecast to 2021 11.5 Major Global Players 12.0 Global Access & Challenges of the Regenerative Medicine Market 12.1 Regenerative Medicine Market in the USA 12.2 Regenerative Medicine in Japan 12.3 Regenerative Medicine in China 12.4 Regenerative Medicine in South Korea 13.0 Cell and CAR T Therapy 13.1 Challenges Relating to Cell therapy and Chimeric Antigen Receptor T Cells in Immunotherapy 13.2 Regulations Pertaining to Immunotherapy, including Adoptive Cell Therapy (CAR-T and TCR) Immunotherapy Regulation in the USA 13.3 Regulations for Cell Therapy & Immunotherapy in Japan 13.4 European Regulation and Cell Therapy & Immunotherapeutics 13.5 Manufacturing of Immunotherapies 13.6 Supply Chain & Logistics 13.7 Pricing & Cost Analysis 14.0 Company Profiles 14.1 Astellas Institute for Regenerative Medicine (Ocata Therapeutics) 14.2 Athersys 14.3 Baxter International (Baxalta, Shire) 14.4 Caladrius Biosciences (NeoStem) 14.5 Cynata Therapeutics 14.6 Cytori Therapeutics 14.7 MEDIPOST 14.8 Mesoblast 14.9 NuVasive 14.10 Osiris Therapeutics 14.11 Plasticell 14.12 Pluristem Therapeutics 14.13 Pfizer 14.14 StemCells Inc 14.15 STEMCELL Technologies 14.16 Takara Bio 14.17 Tigenix 15.0 SWOT Industry Analysis 15.1 What has Strengthened the Industry Thus Far? 15.2 Allogenic and Autologous Stem Cell Industry SWOT Analysis 15.3 What are the Main Driving Forces of this Space? 15.4 Restraints of the Regenerative Medicine Industry as a Whole 15.5 Industry Opportunities Within this Sector 15.6 USA SWOT Analysis 15.7 UK SWOT Analysis 15.8 South Korea SWOT Analysis 15.9 China SWOT Analysis 15.10 Japan SWOT Analysis 15.11 Singapore SWOT Analysis For more information about this report visit http://www.researchandmarkets.com/research/jh2432/global To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-regenerative-medicine-market-analysis--forecast-2017-2021---research-and-markets-300452299.html


News Article | April 18, 2017
Site: marketersmedia.com

Tissue Engineering is a process involving in-vitro development of tissues or organs. It is done to replace or support the function of defective or injured body part. Tissue engineering involves the application of biology and engineering for innovation of tissue substitutes that can maintain, restore and improve the function of ruptured human tissue. Products developed by this procedure are efficient and durable. Tissue engineering is gaining its popularity in various areas such as burn treatment or wound care, neurology products, orthopedics, urological products and others. On the basis of type of material used, tissue engineering and regeneration market can be segmented into synthetic, genetically modified and biological materials. For more information or any query mail at sales@wiseguyreports.com This report focuses on the Tissue Engineering in Global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application. Market Segment by Manufacturers, this report covers Allergan Integra Lifesciences C. R. Bard Zimmer Biomet Organogenesis Osiris Therapeutics Cryolife ACell Biocomposites DSM Episkin J-TEC Athersys Biotime B. Braun International Stem Cell Bio Tissue Technologies Market Segment by Regions, regional analysis covers North America (USA, Canada and Mexico) Europe (Germany, France, UK, Russia and Italy) Asia-Pacific (China, Japan, Korea, India and Southeast Asia) South America (Brazil, Argentina, Columbia etc.) Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa) Market Segment by Applications, can be divided into Orthopedics, Musculoskeletal & Spine Neurology Cardiology & Vascular Skin & Integumentary Others There are 15 Chapters to deeply display the global Tissue Engineering market. Chapter 2, to analyze the top manufacturers of Tissue Engineering, with sales, revenue, and price of Tissue Engineering, in 2016 and 2017; Chapter 3, to display the competitive situation among the top manufacturers, with sales, revenue and market share in 2016 and 2017; 2 Manufacturers Profiles 2.1 Allergan 2.1.1 Business Overview 2.1.2 Tissue Engineering Type and Applications 2.1.2.1 Type 1 2.1.2.2 Type 2 2.1.3 Allergan Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.2 Integra Lifesciences 2.2.1 Business Overview 2.2.2 Tissue Engineering Type and Applications 2.2.2.1 Type 1 2.2.2.2 Type 2 2.2.3 Integra Lifesciences Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.3 C. R. Bard 2.3.1 Business Overview 2.3.2 Tissue Engineering Type and Applications 2.3.2.1 Type 1 2.3.2.2 Type 2 2.3.3 C. R. Bard Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.4 Zimmer Biomet 2.4.1 Business Overview 2.4.2 Tissue Engineering Type and Applications 2.4.2.1 Type 1 2.4.2.2 Type 2 2.4.3 Zimmer Biomet Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.5 Organogenesis 2.5.1 Business Overview 2.5.2 Tissue Engineering Type and Applications 2.5.2.1 Type 1 2.5.2.2 Type 2 2.5.3 Organogenesis Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.6 Osiris Therapeutics 2.6.1 Business Overview 2.6.2 Tissue Engineering Type and Applications 2.6.2.1 Type 1 2.6.2.2 Type 2 2.6.3 Osiris Therapeutics Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.7 Cryolife 2.7.1 Business Overview 2.7.2 Tissue Engineering Type and Applications 2.7.2.1 Type 1 2.7.2.2 Type 2 2.7.3 Cryolife Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.8 ACell 2.8.1 Business Overview 2.8.2 Tissue Engineering Type and Applications 2.8.2.1 Type 1 2.8.2.2 Type 2 2.8.3 ACell Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.9 Biocomposites 2.9.1 Business Overview 2.9.2 Tissue Engineering Type and Applications 2.9.2.1 Type 1 2.9.2.2 Type 2 2.9.3 Biocomposites Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.10 DSM 2.10.1 Business Overview 2.10.2 Tissue Engineering Type and Applications 2.10.2.1 Type 1 2.10.2.2 Type 2 2.10.3 DSM Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.11 Episkin 2.11.1 Business Overview 2.11.2 Tissue Engineering Type and Applications 2.11.2.1 Type 1 2.11.2.2 Type 2 2.11.3 Episkin Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.12 J-TEC 2.12.1 Business Overview 2.12.2 Tissue Engineering Type and Applications 2.12.2.1 Type 1 2.12.2.2 Type 2 2.12.3 J-TEC Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.13 Athersys 2.13.1 Business Overview 2.13.2 Tissue Engineering Type and Applications 2.13.2.1 Type 1 2.13.2.2 Type 2 2.13.3 Athersys Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.14 Biotime 2.14.1 Business Overview 2.14.2 Tissue Engineering Type and Applications 2.14.2.1 Type 1 2.14.2.2 Type 2 2.14.3 Biotime Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.15 B. Braun 2.15.1 Business Overview 2.15.2 Tissue Engineering Type and Applications 2.15.2.1 Type 1 2.15.2.2 Type 2 2.15.3 B. Braun Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.16 International Stem Cell 2.16.1 Business Overview 2.16.2 Tissue Engineering Type and Applications 2.16.2.1 Type 1 2.16.2.2 Type 2 2.16.3 International Stem Cell Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 2.17 Bio Tissue Technologies 2.17.1 Business Overview 2.17.2 Tissue Engineering Type and Applications 2.17.2.1 Type 1 2.17.2.2 Type 2 2.17.3 Bio Tissue Technologies Tissue Engineering Sales, Price, Revenue, Gross Margin and Market Share (2016-2017) 3 Global Tissue Engineering Market Competition, by Manufacturer 3.1 Global Tissue Engineering Sales and Market Share by Manufacturer 3.2 Global Tissue Engineering Revenue and Market Share by Manufacturer 3.3 Market Concentration Rate 3.3.1 Top 3 Tissue Engineering Manufacturer Market Share 3.3.2 Top 6 Tissue Engineering Manufacturer Market Share 3.4 Market Competition Trend For more information or any query mail at sales@wiseguyreports.com ABOUT US: Wise Guy Reports is part of the Wise Guy Consultants Pvt. Ltd. and offers premium progressive statistical surveying, market research reports, analysis & forecast data for industries and governments around the globe. Wise Guy Reports features an exhaustive list of market research reports from hundreds of publishers worldwide. We boast a database spanning virtually every market category and an even more comprehensive collection of rmaket research reports under these categories and sub-categories. For more information, please visit https://www.wiseguyreports.com


Patent
Athersys and Oregon Health And Science University | Date: 2014-01-15

Isolated cells are described that are not embryonic stem cells, not embryonic germ cells, and not germ cells. The cells can differentiate into at least one cell type of each of at least two of the endodermal, ectodermal, and mesodermal lineages. The cells do not provoke a harmful immune response. The cells can modulate immune responses. As an example, the cells can suppress an immune response in a host engendered by allogeneic cells, tissues, and organs. Methods are described for using the cells, by themselves or adjunctively, to treat subjects. For instance, the cells can be used adjunctively for immunosuppression in transplant therapy. Methods for obtaining the cells and compositions for using them also are described.


Grant
Agency: Department of Health and Human Services | Branch: | Program: STTR | Phase: Phase I | Award Amount: 224.72K | Year: 2014

DESCRIPTION (provided by applicant): This is a resubmission of a Phase I-Phase II Fast-Track application for the clinical use of MultiStem in patients with acute respiratory distress syndrome (ARDS). ARDS is defined as acute onset hypoxemia, bilateral radiographic pulmonary infiltrates and lack of atrial pressure hypertension. A novel and exiting possibility is the use of cells as part of the therapy in lung injury. We and other groups have demonstrated that exogenous infusion of isolated mesenchymal stem cells (B-MSC) prevents inflammation and aberrant repair after lung injury. These and other observations suggest that B-MSC is a potentially safe and effective therapeutic intervention in lung injury. Progress toward B-MSC as a cell therapy for ARDS in humans requires completion of preclinical studies and validation in animal models. We propose to evaluate the therapeutic effect of a GMP-produced human adherent bone marrow derived stem cell (MultiStem) in a sheep model of endotoxin-induced moderate-sever


Grant
Agency: GTR | Branch: Innovate UK | Program: | Phase: Collaborative Research & Development | Award Amount: 2.00M | Year: 2015

MUST-ARDS (MultiStem® for the Treatment of Acute Respiratory Distress Syndrome) is a phase 1/2 clinical trial designed to investigate a novel adult cell therapy for ARDS. MultiStem is an allogeneic, adult, multipotent progenitor stem cell that is currently being investigated in phase 2 clinical trials to treat ischemic stroke and ulcerative colitis. Athersys, Ltd., a SME and clinical stage biotechnology company, has been developing the MultiStem cell therapy platform for multiple disease indications. MultiStem cells exhibit a drug-like profile in that they act primarily through the production of multiple factors that regulate the immune system, protect damaged or injured tissue, promote tissue repair and healing and enhance the formation of new blood vessels in regions of ischemic injury. Athersys and the Cell Therapy Catapult through MUST-ARDS determine if MultiStem can reduce mortality, shorten hospital stay and improve patient outcomes for those suffering from ARDS.


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase II | Award Amount: 1.71M | Year: 2015

DESCRIPTION provided by applicant Cardiovascular disease CVD represents a significant unmet medical need with over million patients in the United States suffering from acute myocardial infarction AMI each year This epidemic of heart attacks also represents a risk factor for further cardiovascular disease with over million individuals suffering from chronic heart failure CHF and andgt new patients diagnosed with CHF annually in the United States CHF is associated with high morbidity and mortality as well as high cost of care In fact taken together CVD is the leading cause of death in the United States and is responsible for an estimated $ billion dollars annually in direct and indirect costs according the American Heart As the population ages and the incidence of diabetes and obesity increase the incidence and associated cost of care for CVD Is expected to rise as well By the number of people in US age and over will increase by Despite the high cost of care and prevalence the medical options to treat both AMI and CHF are limited Based on the current clinical data it would appear highly likely that cell therapy will play a role in the prevention ad treatment of cardiac dysfunction in the ensuing years In this study we proposed to develop in the Phase I portion of this proposal an adult bone marrow derived cell therapy product MultiStem that can formulated in a cryovial for rapid thaw and delivery to treat acute ischemic injury For the Phase II portion we plan to investigate the effects of cell therapy in a specific nd novel population of patients with cardiovascular disease those patients with heart attacks that present as non ST elevated myocardial infarcts NSTEMI to determine if we can reduce the morbidity and mortality and provide functional benefit to the heart Importantly the NSTEMI population is growing in prevalence unlike the clinical population of first time ST elevation AMI which has been steadily declining over the past decade Successful completion of these studies will provide the data required for a Phase III registration trial that will be required to market MultiStem for the treatment of acute cardiovascular injury PUBLIC HEALTH RELEVANCE The development of cell therapy for the treatment of acute cardiovascular injury will provide a novel therapeutic for one the leading causes of death in the world


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: STTR | Phase: Phase II | Award Amount: 450.04K | Year: 2016

DESCRIPTION provided by applicant This is a resubmission of a Phase I Phase II Fast Track application for the clinical use of MultiStem in patients with acute respiratory distress syndrome ARDS ARDS is defined as acute onset hypoxemia bilateral radiographic pulmonary infiltrates and lack of atrial pressure hypertension A novel and exiting possibility is the use of cells as part of the therapy in lung injury We and other groups have demonstrated that exogenous infusion of isolated mesenchymal stem cells B MSC prevents inflammation and aberrant repair after lung injury These and other observations suggest that B MSC is a potentially safe and effective therapeutic intervention in lung injury Progress toward B MSC as a cell therapy for ARDS in humans requires completion of preclinical studies and validation in animal models We propose to evaluate the therapeutic effect of a GMP produced human adherent bone marrow derived stem cell MultiStem in a sheep model of endotoxin induced moderate severe ARDS To our knowledge there are no published references on the use of this animal model to evaluate the effect of cell therapies for ARDS making these pre clinical studies unique and highly novel This proposal is the result of a close collaboration between the McGowan Institute of Regenerative Medicine the Division of Pulmonary Allergy and Critical Care and the Division of Cardiothoracic Transplantation at the University of Pittsburgh with Athersys Inc a biotechnology company specialized in the generation of an allogeneic GMP grade bone marrow derived adherent stem cells termed MultiStem We propose that a partnership between academia and industry will accelerate and validate the use of human GMP produced MultiStem in patients with ARDS Our groups have the infrastructure and expertise required to assure successful completion of this project Our goal is to complete the necessary pre clinical studies required to obtain an Investigational New Drug IND Based on a pre IND meeting with the FDA their suggestions have been incorporated in the present proposal {Determination of the biological consequences of intrabronchial or intravenous delivery of MultiStem on the sheep model of LPS induced ARDS} and Specific Aim Phase II to demonstrate the safety of MultiStem in patients with ARDS in a Phase I clinical trial PUBLIC HEALTH RELEVANCE Acute Respiratory Distress Syndrome ARDS is a very common clinical entity and a major cause of morbidity and mortality in the critical care setting with limited therapeutic options The development of cell therapy for the treatment of ARDS will provide a novel therapeutic for this significant disease


Grant
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 300.00K | Year: 2012

DESCRIPTION (provided by applicant): Previously performed and published studies have demonstrated that MultiStem(R), Athersys' patented adult adherent stem cell product, modulates the inflammatory component of secondary brain injury in rodent models of traumatic brain injury (TBI). Further, there are supporting published data illustrating the efficacy of MultiStem in other CNS injury models including stroke, hypoxic-ischemic injury, and spinal cord injury among others. Athersys, Inc., and the University ofTexas Medical School at Houston have a collaborative research agreement in place for the development of proof-of-concept studies in vivo and in vitro for the treatment of TBI and stroke. The ultimate goal of this relationship is to translate these initialpositive findings into clinical trials and novel therapeutic approaches for neurological injury. The specific objective of this SBIR Fast-Track Research Proposal is to define and successfully execute pivotal pre-clinical safety and efficacy studies required for a successful Investigational New Drug submission to the FDA for an optimized cellular therapy regimen for treatment of TBI and its related outcomes. This application proposes an initial GLP toxicity study in Phase 1, followed by sequential studies to address clinically relevant translational issues in progenitor cell therapy for neurological injury/disease. The specific aims are: Phase 1: Define the safety profile of MultiStem delivered intravenously after TBI with both short and long-term GLP toxicity/pathology-necropsy evaluation. The rationale for the proposed groups is that safety must be defined in naive and injured animals, since injury affects biodistribution secondary to chemo-attractant signals from injured tissues. NO GO decision will be based principally on the development of ectopic tissue in any organ (not just cell presence), or significant exacerbation of inflammation/organ function. Phase 2a: The goal of Phase 2a is the completion of comprehensive toxicity studies in TBI, with doses shown to be efficacious in our previous proof-of-concept efficacy testing in rodents. Comprehensive toxicity and anatomic pathology studies will need to be completed at higher doses/multiple doses based on previous proof- of-concept studies. GO/NO GO decisions will be made by assessing the dose toxicity profiles (compared to Controls) relative to previous proof-of-concept efficacy data. Phase 2b: The goal of the Phase 2b portion of the proposal is to establish the optimal dosing scheme based on primary and secondary outcomes measures, after clearing safety studies in Phase 1 and Phase 2a. Translational issues of catheter delivery systems and osmolarity of the cell infusion environment will be evaluated in terms of affecting cell survival and potency. Phase 2c:The primary goals of this sub-phase are (1) IND submission for both adult and pediatric protocols using intravenous MultiStem for severe TBI, and (2) addressing/revising the submissions in response to any critiques, and (3) approval and local IRB submission to allow initiation of the clinical trials.


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase I | Award Amount: 212.80K | Year: 2015

DESCRIPTION provided by applicant One out of every fifty people living in the United States suffers from some form of paralysis a total of almost million Americans This proposal outlines the translational development of a cellular therapeutic already in clinical trials with a novel regenerative peptide to be used in combination for treatment of spinal cord injury SCI and associated dysfunction Preclinical data from Athersys has demonstrated that acute intravenous administration of bone marrow derived multipotent adult progenitor cells MAPC provides immediate and durable neuroprotection after SCI Benefit to locomotor recovery was seen beginning at one week post injury as well as significant sparing of white matter tracts Dr Silver and his colleagues have identified the glial scar as a significant impediment to long distance regeneration specifically inhibitory molecules known as chondroitin sulfate proteoglycans CSPGs After discovering the receptor and signaling pathways associated with this pathway Dr Silverandapos s laboratory has developed a novel peptide Intracellular Sigma Peptide ISP which modulates the key receptor in this pathway In a preclinical study long term subcutaneous treatment with this peptide initiated in the acute phase following spinal cord injury allowed for delayed recovery of locomotion and bladder behaviors following SCI Interestingly while ISP treatment resulted in recovery it had no neuroprotective effects In the month time frame of this Phase I project we propose to combine the use of MAPC with ISP treatment following a translationally relevant rodent model of contusive SCI with the therapeutic aims of i promoting enhanced neuroprotection and ii enhancing sprouting regeneration by altering the response of regenerating axons to inhibitory extracellular matrix We will evaluate the synergistic effects of treatment by analyzing improvements in motor hind limb recovery return of coordinated control of the lower urinary tract and quantification of neuronal sprouting regeneration Dr Silver and collaborators have been investigating the ability of ISP peptide to induce sympathetic neural regeneration and alleviate arrhythmia following myocardial ischemia reperfusion injury This data suggests that ISP could also be utilized in other disorders in which CSPGs play a critical inhibitory role such as traumatic brain injury multiple sclerosis and stroke The fact that MAPC has already proven efficacious in preclinical models of acute myocardial infarction multiple sclerosis traumatic brain injury and stroke dramatically expands the impact that this combinatorial therapy could have in the field of regenerative medicine If MAPC and ISP administration can be shown to be effective in ameliorating SCI associated deficits and dysfunction this therapy could relieve some of the economic burden on the direct and indirect costs associated with SCI related care and more importantly provide meaningful improvement to SCI patients Both treatments are non invasive and systemic making them highly attractive therapeutic options for clinical use PUBLIC HEALTH RELEVANCE This proposal outlines the translational development of a cellular therapeutic already in clinical trials with a novel regenerative peptide to be used in synergy for treatment of contusive spinal cord injury SCI and associated dysfunction We propose to test the combined use of two non invasive and systemic therapeutics in a rodent model with the therapeutic aims of i promoting enhanced neuroprotection and ii enhancing regeneration If successful this combinatorial therapy could relieve a significant portion of the economic burden on the direct and indirect costs associated with SCI related care and more importantly provide meaningful improvement to SCI patients


The present invention generally relates to a series of compounds, to pharmaceutical compositions containing the compounds, and to use of the compounds and compositions as therapeutic agents. More specifically, compounds of the present invention are tricyclic indeno-pyrrole compounds. These compounds are serotonin receptor (5-HT) ligands and are useful for treating diseases, disorders, and conditions wherein modulation of the activity of serotonin receptors (5-HT) is desired (e.g. anxiety, depression and obesity).

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