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The Market for cell Therapy Manufacturing ... Bone Therapeutics Gosselies , Belgium Cell Therapy BrainStorm Cell Therapeutics Kiryat Aryeh, Israel Autologous Stem Cell Therapies for

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  • THE MARKET FOR CELL THERAPY

    MANUFACTURING

    Strategies for Cell Therapy Product Pricing, Cost

    Control, Reimbursement, Distribution, & More

    BioInformant

    Worldwide, LLC

  • Page 1 of 122

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  • Page 2 of 122

    TABLE OF CONTENTS

    1. Introduction to Cell Therapy Manufacturing……………………………………….p.4 2. Leading Cell Therapy Companies……………………………………………………p.6

    2.1. List of Cell Therapy Companies Worldwide

    [Table] List of Cell Therapy Companies Worldwide by Location and Type

    2.2. Leading Cell Therapy Companies

    2.1.1. ReNeuron 2.1.2. Mesoblast Ltd. 2.1.3. Asterias Biotherapeutics 2.1.4. TiGenix NV 2.1.5. Cellectis 2.1.6. Cynata Therapeutics 2.1.7. Cytori Therapeutics 2.1.8. Astellas Pharma, and Subsidiary Ocata Therapeutics 2.1.9. Gamida Cell Ltd. 2.1.10. Pluristem Therapeutics

    3. Approved Cell Therapy Products……………………………………………………p.41 3.1. Apligraf (Organogenesis, Inc. & Novartis AG) 3.2. Carticel (Genzyme) 3.3. Cartistem (MEDIPOST) 3.4. ChrondoCelect (TiGenix NV) 3.5. Cupistem (Anterogen) 3.6. Dermagraft (Advanced Tissue Sciences) 3.7. Epicel (Vericel) 3.8. Hearticellgram-AMI (FCB Pharmicell) 3.9. Holoclar (Chiesi Farmaceutici) 3.10. Osteocel (NuVasive) 3.11. Prochymal (Mesoblast) 3.12. Provenge 3.13. Strimvelis (GSK) 3.14. TEMCELL (JCR Pharmaceuticals Co. Ltd., Licensee of Mesoblast Ltd.)

    4. Pricing Analysis for Cell Therapy Products…………………………………………p.60 4.1. Pricing of Approved Cell Therapy Products

    [Table] Pricing of Approved Cell Therapies

    [Table] Pricing Scale for Approved Cell Therapies, by Type

    4.2. Reasons for High Cell Therapy Product Costs 4.2.1. High Cost of Manufacturing 4.2.2. Need to Recoup Developmental Costs 4.2.3. Cash Flow and Quantity of Cash Reserves 4.2.4. Need for Return on Investment (ROI) 4.2.5. High Cost of Delivery 4.2.6. Lack of Comparative Studies as Evidence for Reimbursement Scheme 4.2.7. Lack of Competition 4.2.8. Potential for High Utility Patient Outcomes (Cures) 4.2.9. Small Market Size

    5. Cost-Control for Cell Therapy Products...…………………………………………..p.69 5.1. Cost of Goods (COGs) Components

  • Page 3 of 122

    5.2. Automation as a Cost Control Measure [Table] Cost Per Million of Clinical-Grade MSCs Expanded by Different Methods

    5.3. Effect of Process Development and Process Change on COGs 5.4. Managing Cash Flow 5.5. Role of Cell Therapy CDMOs

    6. Time Frames for Cell Therapy Product Development…….………………………..p.80 6.1. Pre-Market Challenges 6.2. Persevering Through Lengthy Developmental Timelines 6.3. Navigating the Regulatory Environment 6.4. Timeline from Phase I to Commercialization

    7. Reimbursement of Cell Therapy Products…………………………………..………p.85 7.1. Securing Reasonable Reimbursement 7.2. Encouraging Adoption

    8. Distribution Channels for Cell Therapy Products…..……………………………...p.90 8.1. Addressing Distribution Logistics 8.2. Allogeneic vs. Autologous Therapies

    9. Market Trend Analysis - Key Trends Impacting the Marketplace……………..…p.93 9.1. Regulatory Issues

    9.1.1. FDA Guidelines for HCT/Ps 9.1.2. 21st Century Cures Act)

    9.2. Global Trends 9.2.1. Accelerated Approval Pathway in Japan 9.2.2. Prolific Partnering Between Cell Therapy Companies and Japanese

    Pharmaceutical Companies

    9.2.3. Cell Therapy Products (and Pricing) Resulting from the Japanese Regulatory Framework

    10. Technologies Impacting the Cell Therapy Manufacturing Market…..…………p.109 10.1. Closed-System Manufacturing 10.2. Automation of Cell Therapy Manufacturing Processes 10.3. Automation of Data Management 10.4. Bioreactor Technologies

    11. Market Potential for Autologous vs. Allogeneic Manufacturing………………….p.113

    [Table] Allogeneic vs. Autologous Product Development Among Leading Cell Therapy 12. Cell Therapy Manufacturing Challenges and Considerations…………………….p.117 13. Conclusions…………………………………………….………………………………p.120

  • Page 4 of 122

    1. Introduction to Cell Therapy Manufacturing

    Cell therapy is the use of cells and tissues to regenerate, repair, and enhance human health.

    Traditionally, the modern medical system has relied on a pharmaceutical based approach. This

    lead to the rise of the U.S. Food and Drug Administration (FDA) which was founded in 1906,

    making it more than one-hundred years old. Unfortunately, our approach to human health has not

    experienced a dramatic shift over the past 100+ years, and as a society, we still tend to rely on a

    drug-based approach.

    Although variations exist, drugs generally work by binding to receptors on the cell surface or by

    exerting an enzymatic effect to regulate the rate of an internal chemical reaction.

    Pharmaceuticals can frequently modulate the human system with great effect, but rarely do they

    have the capacity to regenerate or restore entire tissues or systems within the human body.

    In contrast, cell therapy works

    because cells are powerful factories

    that can exert therapeutic effects

    through a wide range of strategies,

    including honing to sites of injury,

    exerting paracrine effects, and in

    some cases, differentiating into new

    types of cells and tissues. This

    versatility makes cell therapy

    extremely powerful and gives it the potential to reverse previously untreatable diseases. Needless

    to say, it is an exciting time in history.

    For this reason, cell therapy – or more likely, cell therapy in combination with pharmaceuticals –

    will be the future of human health.

  • Page 5 of 122

    One of the major issues with moving cell therapy

    products from “bench to bedside” has been

    manufacturing bottlenecks. The heterogeneous nature

    of cell therapy products has introduced manufacturing

    complexity and regulatory concerns, as well as scale-

    up complexities that are not present within traditional

    pharmaceutical manufacturing.

    Furthermore, much of cell therapy manufacturing now involves patient-specific cell therapies,

    where cells for an individual patient are processed one batch at a time. Ten years ago, it was a

    common belief that making individual batches of a therapy for each patient would never provide

    an economical business model. Rather, allogeneic therapies would better align with the

    pharmaceutical model, because universal cell populations could be prepared in advance to later

    be prescribed to patients in need, much like drug compounds have been administered for

    decades.

    Nonetheless, clinical data in support of patient-specific therapy has been compelling, so the

    pressure is on cell therapy industry to manufacture these therapies on a larger scale. It is now

    understood that both autologous and allogeneic cell therapies will contribute important

    therapeutic solutions. There are opp

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