September 2017; 123 Pages
One of the major issues with commercializing cell therapy products is 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.
Previously, it was believed that making individual batches of a therapy for a patient would never be a successful commercial model. It was assumed that allogeneic therapies would prevail, because universal cell populations could be prepared in advance to later be prescribed to patients, similar to how drug compounds are administered. Nonetheless, clinical data in support of patient-specific therapy has been compelling, so the pressure is on for the cell therapy industry to manufacture these therapies on a larger scale. Clearly, there are opportunities for the development of both autologous and allogeneic cell therapies, which vary greatly in their manufacturing requirements, routes for patient administration, and cost structures.
While many factors contribute to high pricing associated with cell therapies, a critical factor affecting the pricing of a cell therapy product is the cost of continuously manufacturing the product. To keep a cell therapy product “on market,” manufacturing costs of must be covered and a profit margin achieved. Therefore, great consideration must be given to the variables affecting the costs of goods (COGs) associated with cell therapy manufacturing, as well as strategies for process optimization.
Among cell therapy companies, the pressure for manufacturing process innovation can incentivize them to seek third-party partners who possess technical, manufacturing, and regulatory expertise in cell therapy development and manufacturing, such as cell therapy contract and development manufacturing organizations (CDMOs).
Currently, there are more than 20 cell therapy CDMO’s specializing in manufacturing and clinical trial support for cell therapy companies, including: WuXi PharmaTech (and its subsidiary WuXi AppTec), Lonza Group (acquired PharmaCell, B.V. in May 2017), MasTHerCell (acquired by Orgenesis), Miltenyi Bioprocess (Contract Manufacturing Business of Miltenyi Biotec), Hitachi Chemical Advanced Therapeutics Solutions (acquired PCT), Cell and Gene Therapy Catapult, Nikon CeLL Innovation Co., Ltd. (“NCLi”), KBI Biopharma (acquired Opexa Assets in February 2017), Brammer Bio, Roslin Cell Therapies, apceth Biopharma, and more.
Benefits of partnering with a cell therapy CDMO include scalability, speed to market, access to technical expertise without overhead costs, and cost efficiencies. This report considers the role of cell therapy CDMOs, as well as the importance of in-house manufacturing at cell therapy companies worldwide.
This global strategic report provides detailed analysis of ten leading cell therapy companies worldwide, evaluating the strengths of each company, as well as identifying cell therapy products in development, manufacturing strategies, and partnerships.
These companies include:
- Mesoblast Ltd.
- Asterias Biotherapeutics
- TiGenix NV
- Cynata Therapeutics
- Cytori Therapeutics
- Astellas Pharma (and Subsidiary Ocata Therapeutics)
- Gamida Cell Ltd.
- Pluristem Therapeutics
Additionally, the report identifies cell therapy products that have been reviewed and approved by internationally-recognized regulatory agencies, as well as products brought to market in the U.S. with FDA approval. It also specifies which cell therapies have since been pulled from market, due to factors such as low adoption rates, unsuccessful reimbursement strategies, or high cost of manufacturing.
The report analyzes time frames for cell therapy product development, distribution channels, key trends and technologies impacting cell therapy manufacturing, cost control measures, and challenges and considerations affecting with cell therapy manufacturing.
It provides an overview of the regulatory environment affecting cell therapy manufacturing in key countries worldwide, evaluating the importance of the recent FDA guidelines for Human Cell and Tissue-based Products (HCT/Ps) and the 21st Century Cures Act, as well as accelerated pathways for cell therapy approvals in Japan, including the:
- Act on the Safety of Regenerative Medicine (Law No. 85/2013)
- Pharmaceuticals and Medical Device (PMD) Act (Law No. 84/2013)
Produced for companies involved with cell therapy product development, this global strategic report offers critical insights into strategies for optimizing modern cell therapy manufacturing. Based in Washington, DC, BioInformant is first and only market research firm to specialize in the stem cell industry. Unlike other publishers that hire analysts from foreign countries, BioInformant is an American-based company with more than 10 years of experience with tracking the cell therapy market (2006 to present).
BioInformant compiled this global strategic report using primary interviews with more than 50 individuals from across the cell therapy manufacturing industry, including representatives from:
- Gamida Cell Ltd. (Dr. Yael Margolin)
- Pluristem (Yaky Yanay, Yaron Ramati)
- Cynata Therapeutics (Dr. Paul Wotton)
- Accellta (Dr. Itzchak Angel and Eran Cohen)
- Stem Cell Medicine Ltd. (Dr. Frida Grynspan)
- Minerva Biotechnologies (Dr. Cynthia Bamdad)
- RoosterBio (Dr. Jon Rowley)
- Universal Cells (Gregory Block)
- Many More
It incorporates secondary findings from SEC filings, company websites, press releases, investor presentations, government policy documents (FDA / PMDA), and more.
TABLE OF CONTENTS
1. Introduction to Cell Therapy Manufacturing
2. Leading Cell Therapy Companies
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
2.1.2. Mesoblast Ltd.
2.1.3. Asterias Biotherapeutics
2.1.4. TiGenix NV
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
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)
3.13. Strimvelis (GSK)
3.14. TEMCELL (JCR Pharmaceuticals Co. Ltd., Licensee of Mesoblast Ltd.)
4. Pricing Analysis for Cell Therapy Products
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
5.1. Cost of Goods (COGs) Components
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
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
7.1. Securing Reasonable Reimbursement
7.2. Encouraging Adoption
8. Distribution Channels for Cell Therapy Products
8.1. Addressing Distribution Logistics
8.2. Allogeneic vs. Autologous Therapies
9. Market Trend Analysis – Key Trends Impacting the Marketplace
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
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
[Table] Allogeneic vs. Autologous Product Development Among Leading Cell Therapy
12. Cell Therapy Manufacturing Challenges and Considerations
– Mesoblast Ltd.
– Asterias Biotherapeutics
– TiGenix NV
– Cynata Therapeutics
– Cytori Therapeutics
– Astellas Pharma, and Subsidiary Ocata Therapeutics
– Gamida Cell Ltd.
– Pluristem Therapeutics
– Organogenesis, Inc. & Novartis AG
– TiGenix NV
– Advanced Tissue Sciences
– FCB Pharmicell
– Chiesi Farmaceutici
– JCR Pharmaceuticals Co. Ltd. (Licensee of Mesoblast Ltd.)
– MasTHerCell, Acquired by Orgenesis
– PCT, a Caladrius Company (Previously Neostem)
– WuXi PharmaTech, Including Subsidiary WuXi AppTech, Inc.
– Cell and Gene Therapy Catapult
– KBI Biopharma (Acquired Opexa Assets, February 2017)
– Brammer Bio (Formed through Merger of Brammer Biopharmaceuticals and Florida Biologix)
– Roslin Cell Therapies, a Subsidiary of Roslin Cells
– apceth Biopharm
– Cognate Bioservices, Inc.
– MEDINET Co. Ltd.
– PharmaCell, B.V.
– The Clinical Trial Company (TCTC)
– Miltenyi Bioprocess, the Contract Manufacturing Business of Miltenyi Biotec
– Lonza Group, Cell Therapy Manufacturing Unit
– Praxis Pharmaceutical
– Cellular Therapeutics Ltd. (CTL)
– CTI Clinical Trial and Consulting
– Eufets GmbH
– Fraunhofer Gesellschaft
– Cellforcure SASU
– Molmed S.p.A.
Cell Therapies Mentioned
– Apligraf (Organogenesis, Inc. & Novartis AG)
– Carticel (Genzyme)
– Cartistem (MEDIPOST)
– ChrondoCelect (TiGenix NV)
– Cupistem (Anterogen)
– Dermagraft (Advanced Tissue Sciences)
– Epicel (Vericel)
– Hearticellgram-AMI (FCB Pharmicell)
– Holoclar (Chiesi Farmaceutici)
– Osteocel (NuVasive)
– Prochymal (Mesoblast)
– Strimvelis (GSK)
– TEMCELL (JCR Pharmaceuticals Co. Ltd., Licensee of Mesoblast Ltd.)
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