細胞療法：医薬品製造受託機関 (CMO) にとっての市場機会

細胞療法は、以前より輸血や生体移植といった形で医療現場で活用されてきましたが、バイオテクノロジーや医薬品開発といった分野では近年になってようやく本格的な利用が始まりました。それでも、難治性血液がん向けのCAR-T細胞療法など、一部の分野では実用化が着実に進んでいます。一方、治験の成果が現れて、それが製品認証につながるまで長い時間を要することから、次世代型の技術の開発も今から同時並行で進める必要もあります。

当レポートでは、世界の細胞療法市場における現在・将来の市場機会について分析し、技術の概要や市場の基本構造、技術開発・治験の現状と今後の方向性、市場規模の動向見通し、分野別の詳細動向、業界関係者 - CMO (医薬品製造受託機関)、バイオテクノロジー／製薬企業ほか - にとっての将来的な期待収益や投資機会などを調査しております。

目次

• エグゼクティブ・サマリー
• イントロダクション
• 細胞療法：実用化の体験
• 細胞療法のパイプライン製品
• 次世代型の技術
• 当局の認証見通し
• 細胞療法のサプライチェーン
• 需要モデル
• 結論
• 分析手法：注記
• 付録

List of Tables

• Table 1 : FDA-Approved, Marketed Cell Therapies 7
• Table 2: Cell Therapy Approvals 15
• Table 3: Cell Therapy CDMOs' Biological Services 17
• Table 5: Cell Therapy CDMOs' Packaging Capabilities 19
• Table 6: Cell Therapy CDMO Sites and Associated Cell Source and Types 21
• Table 7: Method of Transfection Used at Cell Therapy CDMO Sites 23
• Table 8: Model for Cell Therapy Clinical Requirements 24
• Table 9: Model for Cell Therapy Commercial Requirements 25
• Table 10: Model for Yearly Cell Therapy Commercial Requirements 31

List of Figures

• Figure 1: Kymriah Global Sales Forecast 2018-2024 ($B) 8
• Figure 2: Yescarta Global Sales Forecast 2018-2024 ($B) 9
• Figure 3: Pipeline Cell Therapy Candidates 10
• Figure 4: Phase I Trial Starts by Sponsor Type 11
• Figure 5: Phase II Planned Trial Starts by Sponsor Type 12
• Figure 6: Phase III Planned Trial Starts by Sponsor Type 12

Cell therapy is defined as the administration of living whole cells to the patient for the treatment of a disease. The origin of the cells can be from the same individual (autologous source) or from a donor (allogeneic source). While cell therapies have a long history as blood transfusion and surgical procedures, their use as bio/pharmaceutical therapies is a recent development. Despite their newness, CAR-T cell therapies have already shown promise in the treatment of refractory hematological cancers, where there were limited treatment options previously.

Cell therapies represent an exciting and complex new treatment type, with the potential to treat an enormous range of indications, in some cases offering the possibility of a disease cure with a single treatment. Of the current clinical pipeline, it is anticipate that approximately 80 therapies will be approved by the FDA.

Cell therapy manufacturing is in its infancy, using time-, space-, and labor-intensive techniques that are appropriate for current volume needs, mostly driven by clinical trials. However, these methods will no longer be practicable for the scaled-up commercial cell therapy requirements of the near future. Given the necessary time-lag for building capacity, the industry must immediately begin to address next-generation technologies, driven by automation.

Portions of the CDMO industry are investing in greater capacity to support the future cell therapy pipeline. And small- and medium-sized cell therapy developers, which represent a sizeable portion of the pipeline, will need to rely on contract manufacturing, at least at first, as initial outlay for these complex technologies will be very high.

The report "PharmSource - Cell Therapy Market Opportunity for CMOs - 2018 Edition", will explore whether the bio/pharma industry is adequately prepared for future cell therapy developments and whether pharma companies and CDMOs are making appropriate investments in the capacity and technology to support the coming opportunity. In particular, this Trend Report seeks to achieve the following objectives -

• To describe and evaluate the cell therapy market opportunity;
• To describe and address the adequacy of the network of CDMOs that manufacture for the cell therapy market, both clinical and commercial;
• To assess the outlook for buyers and sellers of contract manufacturing services for cell therapies, including the supply/demand balance.

Companies Mentioned: Cellectis, China PLA General Hospital, GSK, 3P Biopharmaceuticals, Abzena, Anemocyte SRL, apcethBiopharma GmbH, Areta International, Army Military Medical University (formerly Third Military Medical University), Astellas, Atvio Biotech Ltd, Austrianova Singapore Pte. Ltd., Autolus, Bio Elpida, Bioinovasro, Brammer Bio, CCRM, Celgene, Cell and Gene Therapy Catapult, CellforCure, Cellin Technologies LLC, Cellular Therapeutics Ltd., Celularity, City of Hope, Cognate Bioservices, Cook General BioTechnology, LLC, Dendreon Pharmaceuticals LLC, Esperite, Fraunhofer Group, GE Healthcare, Gencure, Gilead Sciences Inc., Histocell SL, Hitachi Chemical Co Ltd, Hitachi Chemical Co., Ltd., HolostemTerapieAvanzate, Incell Corporation LLC, International Stem Cell Corporation, Japan Tissue Engineering, Johnson & Johnson, KBI Biopharma, Kite, Kite Pharma Inc., Lonza Group, Lonza Netherlands, MaSThercell, Medinet, MolMedSpA, New York Blood Center, Inc, Newcastle University, NHS Blood and Transplant, Nikon Corporation, Novartis International AG, Novartis Pharmaceuticals Corp., Organogenesis Inc., Oxford Biomedica, Pfizer, Provia Laboratories LLC, Roslin Cell Therapies, Sanpower Group Co Ltd, Sanquin Blood Supply Foundation, Shenzhen Geno-Immune Medical Institute, Stem Cell and Cancer Institute (SCI), Takara Bio, University College London, University of Edinburgh, University of Miami Health System, University of Minnesota, University of Pittsburgh, University of Utah Health, Vericel Corp., WuXiAppTec, Yposkesi.

Scope

This report seeks to achieve the following objectives -

• To describe and evaluate the cell therapy market opportunity;
• To describe and address the adequacy of the network of CDMOs that manufacture for the cell therapy market, both clinical and commercial;
• To assess the outlook for buyers and sellers of contract manufacturing services for cell therapies, including the supply/demand balance.

Reasons to buy

This report is required reading for -

• CMO executives who must have deep understanding of the Cell Therapy marketplace to make strategic planning and investment decisions.
• Sourcing and procurement executives who must understand crucial components of the supply base in order to make decisions about supplier selection and management.
• Pharmaceutical and Biotech companies involved in the development of Cell Therapies or Cell Therapy technologies.
• Private equity investors that need a deeper understanding of the market to identify and value potential investment targets.

Table of Contents

• 1. Executive Summary 4
• 2. Introduction 5
• 3. Commercial Cell Therapy Experience 7
• 4. Cell Therapy Pipeline 10
• 5. Next-Generation Technologies 13
• 6. Approvals Outlook 15
• 7. Cell Therapy Supply Chain 16
• 8. Demand Models 23
• 9. What It Means 25
• 10. Notes on Methodology 28
• 11. Appendix 32