Regenerative Therapies for Neurological Disorders

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  • Regenerative Therapies for Neurological Disorders
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  • Company Overview
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  • Company Background Quick Facts Clinical-stage regenerative medicine company Founded in 2001 Headquartered in Mountain View, Calif. Funding to Date: $53 million 35 patents issued, 41 patents pending Mission Statement SanBio develops regenerative therapies addressing unmet medical needs and creating value for stakeholders.
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  • Financial Support Clinical Trial & Development Innovative Business Infrastructure Innovative Science & Technology Japan/U.S. Hybrid Innovation
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  • Neurological Disorders: Market Size Neurological Disorder Sector is Growing Rapidly, Faster Than Other Markets DiseasePrevalence in U.S. (2010) U.S. Product Sales (2010) Stroke6.8MM Parkinsons Disease1.6MM Multiple Sclerosis400,000 Spinal Cord Injury300,000N/A We are researching the product sales and will add
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  • Potential Market Value Valuation Summary
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  • Competitive Overview Neural Deficit Recovery (NDR) patented drug discovery platform Bone marrow-derived cells Industrial scale expansion Therapeutic potential
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  • Management Team Keita Mori, MBA Co-CEO, Chairman, Co-Founder Toru Kawanishi Co-CEO, Co-Founder Damien Bates MD, PhD, FRACS, MBA Chief Medical Officer Michael P. McGrogan, Ph.D. Senior Vice President, Production Development Placeholder: Headshot
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  • Senior Advisors Donald KennedyFormer President of Stanford University, Commissioner of the United States Food and Drug Administration, and Editor-in-Chief of Science Mario RosatiPartner at Wilson Sonsini Goodrich & Rosati. Managing partner of WS Investments, an investment partnership composed of the partners and associates of the firm Charles GarvinPrincipal, Palisades Associates, a merchant banking firm. A founding principal of The Beta Group, a Silicon Valley business development organization Gary SnableFounder, Ex CEO, Layton Bioscience George MartinFormer Scientific Director, National Institute of Aging (NIH); SVP Fibrogen Arnold CaplanProfessor, Case Western Reserve Martha BohnProfessor, Northwestern University, Past Member of RAC Cesario BorlonganProfessor, University of Southern Florida Krys BankiewiczProfessor, UCSF Yoichi NabeshimaFormer Professor, Kyoto University; Director, IBRI Scientific Advisory Board Advisors
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  • Partnerships Clinical Trial Partners Corporate Partners Innovation Awards
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  • Approach to Brain Regeneration Donor bone marrow stem cells are harvested Allogeneic Cell Transplant Regenerative effect on recipient cells Brain & body function restored
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  • NDR Technology Platform Bone Marrow SB623 SB618 SB308 Products (Cells-in-a-bottle) Production...... Neural Deficit Recovery NDR Technology
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  • Product Pipeline
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  • Bone Marrow Derived Cells Stroke, TBI, SCI, Retinal Disease, PD, AD, etc. Multiple Sclerosis, Peripheral Nerve, SCI, etc. Muscle Dystrophy, etc. SB623SB618SB308 Bone Marrow-Derived Neuroregenerative Cells Enhanced Marrow Stromal Cells (eMSC) Bone Marrow-Derived Muscle Regenerative Cells P-1/2NonclinicalResearch Different image Bone Marrow-Derived Regenerative Cell Medicines Damien Can you recommend an image for SB618?
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  • Indications Overview IndicationSB623SB618SB308 Stroke Recovery Parkinsons Disease Spinal Cord Injury Multiple Sclerosis Muscular Dystrophy Trauma
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  • SB623
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  • Therapy Overview Developed as treatment for chronic neurological deficits stable stroke, advanced Parkinsons disease, traumatic brain injury Reverses neural damage when injected into neural tissue Single allogeneic donor cell can be used to treat thousands of patients. Shown to restore function to damaged neurons associated with stroke, spinal cord injury and Parkinson's disease SB623 Cells
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  • Cells are expanded, formulated and cryopreserved in vials stored in vapor phase of liquid nitrogen Final product is a sterile frozen suspension containing at least 10 million viable cells per mL in a cryovial Shipped to clinical sites using a Dry Nitrogen Cryo shipper with adsorbed liquid nitrogen Cells will grow with a doubling time of 3 to 4 days after thaw Scalable Production Methods Reduced to Practice Manufacturing Process & Product Preparation
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  • Multiple Modes of Action SB623 Modes of Action Trophic Factors Extracellular Matrix Anti-inflammation Stimulating Migration Angiogenesis Damien Please note that we will have our creative team rework this slide to make it look more polished
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  • Product Characteristics Bulk Substance Specifications
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  • Product Characteristics Drug Product Specifications AttributeSpecification Purity and Impurities Purity 80% Positive CD29, CD90, CD105 Impurities 5% Positive CD31, CD34, CD45 Quality Cell Viability 70% viable Total Viable Yield 8x10 6 viable cells/mL Plating 50% Cell Growth RateDoubles in 1 to 5 days Safety Colony Growth in Soft AgarNo Colonies SterilityNo Growth BacterialNo Growth FungalNo Growth Endotoxin 5 EU/mL
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  • Loss of NICD with Continued Passages Disappearance of The Plasmid During Expansion (prior to phase 2) Plasmid pN-2 was measured using qPCR and expressed as copies per diploid genome. Passage number is represented as P1, P2, etc. The negative control is buffer alone. Lanes 1, 2, 5 and 6 are unactivated/unstabilized negative controls. Lanes 3 and 4 show activated and stabilized samples and indicate the location of the endogenous NICD. Lane 7 shows high levels of NICD protein 3 days post transfection. Lane 8 shows no detectable NICD in the final product.
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  • Transient Transfection with NICD Causes Epigenetic Changes CpG Methylation Status of 5 Different Genes for 3 Donors
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  • Homogeneity and Identity of SB623 Cells Cells are Positive for MSC Markers and Negative for Hematopoietic Markers
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  • Proteomic Analysis of the Extracellular Matrix Characterization of the ECM Produced by SB623
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  • Extracellular Matrix Produced by Bone Marrow Stromal Cells ECM Promotes Neural Growth
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  • Extracellular Matrix Produced by Bone Marrow Stromal Cells Supports Growth of Different Neural Cell Types
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  • Comparison of The Neuropoietic Activity Derived ECM Supports Nestin Growth; Mediated by TGM2
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  • Human Mesenchymal Stromal Cells and Their Derivative Multiple Growth Factors Released Placeholder: We are simplifying the table per your notes and will add in
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  • Human Mesenchymal Stromal Cells and Their Derivative Reduces Cell Death In Vitro Model Ischemia
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  • Quantitative Microplate Assay for Studying MSCs Promote Neural Cell Growth and Differentiation
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  • Comparison of The Neuropoietic Activity SB623 Promotes More Neural Cell Growth and Differentiation Than MSCs
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  • Comparison of The Neuropoietic Activity SB623 Action Mediated by FGF2 and BMP4
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  • Comparing The Angiogenic Potency SB623 and MSCs Secrete Proangiogenic Factors
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  • Comparing The Angiogenic Potency SB623 Promotes More Branching in Aortic Ring Assay
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  • Comparing The Immunosuppressive Potency Decreases Cell Mediated Immune Response
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  • Effect on Peripheral and Nervous System Innate Immune Cells SB623 Cells Reduce The Percentage of Pro-Inflammatory Peripheral Blood Monocytes
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  • Comparing The Immunosuppressive Potency SB623 Induces Regulatory T Lymphocytes
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  • Comparing The Immunosuppressive Potency SB623 Inhibits Differentation of Monocytes to Dendritic Cells
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  • Stem Cell Recruitment of Newly Formed Host Cells SB623 Supports Robust Migration and Proliferation of Endogenous Stem Cells in Traumatic Brain Injury Model
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  • Effect of SB623 on Neural Progenitor Cell Migration Dose Response of SB623 Conditioned Medium on NPC Migration
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  • Safety Cells Are Not Tumorigenic Absence of Growth on Brain Slices SB623 Cells Do Not Exhibit Signs of Tumorigenic Transformation
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  • Safety Transient Persistence of SB623 Cells In Vivo and No Migration Away From Site of Implantation Rapid Disappearance of SB623 Cells In Vivo
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  • Safety Differentiation Potential of MSCs and SB623 SB623 Cells Have Limited Differentiation Potential
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  • Clinical Trials and Data
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  • SB308 Muscle Degeneration Development Stages Damien Please note that we will have our creative team rework this slide to make it look more polished
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  • ResearchNon-clin.IND Approval P1P2P3 Traumatic Brain Injury Ret