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市場調査レポート

再生医療の世界市場:2019年

Global Market for Regenerative Medicine (RM), 2019

発行 BIOINFORMANT WORLDWIDE, LLC 商品コード 869663
出版日 ページ情報 英文 390 Pages
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再生医療の世界市場:2019年 Global Market for Regenerative Medicine (RM), 2019
出版日: 2019年06月20日 ページ情報: 英文 390 Pages
概要

再生医療 (RM) 産業におけるベンチャーキャピタル投資は、2010年の2億米ドルから2018年には146億米ドルへと急増しています。

当レポートでは、世界の再生医療 (RM) 産業について調査し、特許、補助金、および化学出版物の割合、タイプ・段階・スポンサー・地域別による治験活動、市場規模の予測、新しい市場機会と脅威、認可済みのRM製品、地域分析、規制動向、および主要企業のプロファイルなどをまとめています。

第1章 レポート概要

第2章 再生医療 (RM) :イントロダクション

  • RM産業の鳥瞰図
  • RM産業:世界の資金
  • 再生医療の市場アクセス
  • 予測されるRM製品発売
  • 上位RM企業20社:パイプライン規模別
  • RM研究機関

第3章 細胞療法 (CT) :概要

  • 細胞療法のタイムライン
  • 細胞療法に使用される幹細胞の種類
  • 神経幹細胞 (NSC)
  • 胚性幹細胞 (ESC)
  • 細胞療法の領域における注目のイベント
  • 細胞療法企業の上位15社

第4章 遺伝子組み換え細胞療法 & 免疫療法

  • 遺伝子組み換え細胞療法の開発企業数
  • CAR-T細胞療法
  • T細胞受容体 (TCR) 療法
  • ナチュラルキラー (NK) 細胞療法
  • TIL (腫瘍浸潤リンパ球) 療法
  • MIL (Marrow-Infiltrating Lymphocytes)
  • ガンマ-デルタT細胞療法
  • 樹状細胞 (DC) 療法

第5章 遺伝子療法

第6章 ヒト組織工学 (TE)

  • TEにおけるスマートバイオマテリアル
  • 人工・自然由来足場
  • 代用細胞
  • TE領域における2つの主な発展

第7章 遺伝子デリバリービークル

  • ウィルスベクター
  • 非ウィルスベクター

第8章 RM:治験情勢

  • RM治験への参加人数
  • 治験:RM部門における治療カテゴリー別
  • 細胞療法の治験
  • 遺伝子組み換え細胞療法の領域における治験
  • 遺伝子療法の治験数
  • TEの治験数

第9章 世界の認可済みRM製品

  • 認可済みRM製品数:地域別
  • 認可済みRM製品:年度別
  • 世界のMA付きRM製品:治療タイプ別
  • オーストラリアにおける認可済みRM製品
  • 中国で認可された遺伝子療法製品
  • 欧州における認可済みRM製品
  • インドにおける認可済みRM製品
  • 日本における認可済みRM製品
  • ニュージーランドにおける認可済みRM製品
  • 韓国における認可済みRM製品
  • 米国における認可済みRM製品、ほか

第10章 細胞療法・遺伝子療法向け細胞の製造

  • 製造コスト
  • 主要な大規模・中規模医薬品企業の社内遺伝子療法製造能力
  • 主要な大規模・中規模医薬品企業の社外遺伝子療法製造
  • 北米における遺伝子療法の製造能力
  • 欧州における遺伝子療法の製造能力

第11章 CMO & CDMO

第12章 市場分析

  • 世界のRM製品市場
  • 世界の細胞療法製品市場
  • 世界の遺伝子療法製品市場
  • 世界のTE製品市場

第13章 企業プロファイル

目次

EXECUTIVE SUMMARY

The Regenerative Medicine (RM) Industry has been rapidly expanding in recent years, making it a high-value, fast-growth market. Key drivers for the market include high rates of clinical trials, accelerated pathways for product approvals, new technologies to support cell and gene therapy manufacturing, and the potential for cell therapies to revolutionize healthcare.

There are now accelerated pathways for advanced therapy medicinal products (ATMPs) in several countries worldwide, including the U.S., Japan, and South Korea. Legislation took effect in Japan in late 2014, in South Korea in 2016, and in the United States in 2017. Additionally, the EU has a program for product acceleration - the Adaptive Pathways. These historic events demonstrate to investors, the public, and funding providers alike that regenerative medicine is a sector that has emerged, no longer one that is evolving in the future.

“Big Pharma” is also demonstrating snowballing interest in regenerative medicine. At its core, this is a strategic way for pharmaceutical companies to diversity their product development pipeline. Much of this interest has been driven by progress with immunotherapy treatments, such as CAR-T cell therapies, most notably, Kymriah and Yescarta. After attaining FDA approvals, these two products achieved historic approvals across varied geographic markets, providing hope to millions of cancer patients worldwide.

The Regenerative Medicine (RM) Industry also saw venture capital investment expand from a mere $200 million in 2010 to a stunning $14.6 billion in 2018. This upsurge in venture capital investment represents growth of 7,300% over an eight-year period. Aggressive construction of cell and gene therapy manufacturing facilities is also being undertaken by biotech companies seeking their own production capabilities, as well as by CDMOs planning to serve third-party clients from across the RM sector. With more than 900+ brave companies composing the marketplace, regenerative medicine advances are truly disrupting healthcare.

The Regenerative Medicine (RM) Industry includes companies that are developing:

  • Stem cell therapies
  • Cellular therapies
  • Gene therapies
  • Tissue engineering
  • Direct cell reprogramming
  • Exosome therapies
  • Cellular scaffolds and matrices
  • 3D bioprinting
  • Biomaterials
  • Combination Products
  • And More

As the first and only market research firm to specialize in the stem cell industry, BioInformant has an exceptional and unique understanding of the Regenerative Medicine (RM) Industry. Founded in 2006, BioInformant has collected over 13 years of historical data on the RM industry, providing it with a robust historical database of data on which to make future market projections. Headquartered in Washington, DC, BioInformant is strategically positioned near industry lawmakers, the National Institutes of Health (N.I.H.), the U.S. FDA, and the Maryland Biotech Corridor, among other critical resources.

In addition to leveraging an experienced team of analysts, this experience provides BioInformant with access to Key Opinion Leaders (KOLs) from across the RM sector, including dozens of executives who were interviewed for this report. To date, BioInformant has interviewed leadership from FUJIFILM, Pluristem Therapeutics, Cynata Therapeutics, RooserBio, Celularity, Hemostemix, Glycostem, Accellta, ORIG3N, Gamida Cell, Nohla Therapeutics, Fortuna Fix, Avery Therapeutics, Stem Cell Medicine Ltd., Minerva Biotechnologies, Celyad, Universal Cells, Rhemastem, and hundreds of others. With research cited by major news outlets that include the Wall Street Journal, Nature Biotechnology, Xconomy, and Vogue Magazine, BioInformant serves Fortune 500 leaders that include GE Healthcare, Pfizer, and Goldman Sachs.

Claim this global strategic report to learn:

  • Rates of Regenerative Medicine (RM) Industry Patents, Grants, and Scientific Publications
  • RM Clinical Trial Activity by Type, Stage, Sponsor, and Geography
  • Market Size Determinations with 5-Year Projections
  • Emerging Market Opportunities and Threats
  • Approved RM Products, including Dates of Approval and Market Pricing
  • Geographic Forces Impacting the RM Industry
  • Regulatory Pathways and Accelerated Approval Programs
  • Profiles for Leading Market Competitors
  • And Much More

Table of Contents

1. REPORT OVERVIEW

  • 1.1. Statement of the Report
  • 1.2. Executive Summary

2.0. REGENERATIVE MEDICINE (RM): AN INTRODUCTION

  • 2.1. A Bird's Eye View of the RM Industry
    • 2.1.1. Number of RM Companies
    • 2.1.2. Major Therapeutic Platforms and Enabling Technologies in RM Industry
    • 2.1.3. RM Product Approvals in 2017/2018
    • 2.1.4. Total RM Clinical Trials
    • 2.1.5. Total RM Clinical Trials by Technology Type
    • 2.1.6. Patient Impact of Three U.S.-Approved RM Products, 2018
    • 2.1.7. Select Anticipated RM Industry Clinical Trial Data Readouts: 2019
  • 2.2. RM Industry-Total Global Financings: 2018
    • 2.2.1. Total Financings to RM Industry by Type and Year, 2016-2018
    • 2.2.2. Corporate Partnerships (Upfront Payments) in RM Industry, 2018
    • 2.2.3. Private Placements and Venture Financings in RM Industry, 2018
    • 2.2.4. Public Offerings (IPOs & Follo-Ons) in RM Industry, 2018
    • 2.2.5. Total Merger & Acquisition within RM Industry by Year, 2016-2018
  • 2.3. Market Access for Regenerative Medicines as of January 2019
  • 2.4. Anticipated RM Product Launches
    • 2.4.1. Predicted RM Treated Patient Numbers
  • 2.5. Top 20 RM Companies by Pipeline Size
  • 2.6. RM Research Institutes
    • 2.6.1. RM Research Institutes in the U.S.
    • 2.6.2. RM Research Institutes in Europe
    • 2.6.3. RM Research Institutions in Australia
    • 2.6.4. University Research to Products in the Market

3. CELL THERAPY (CT): AN OVERVIEW

  • 3.1. Cell Therapy Timeline
  • 3.2. Types of Stem Cells Used in Cell Therapy
    • 3.2.1. Hematopoietic Stem Cells (HSCs)
    • 3.2.2. Induced Pluripotent Stem Cells (iPSCs)
    • 3.2.3. Mesenchymal Stem Cells (MSCs)
      • 3.2.3.1. Number of MSCs in Clinical Trials
      • 3.2.3.2. Indications for which MSCs are tested in Clinical Trials
  • 3.3. Neural Stem Cells (NSCs)
    • 3.3.1. Indications Addressed by NSCs in Clinical Trials
  • 3.4. Embryonic Stem Cells (ESCs)
    • 3.4.1. Clinical Trials using Human Embryonic Stem Cells (hESCs)
    • 3.4.2. Number of Clinical Trials using hESC-Derived Cells in 2018
  • 3.5. Notable Events in Cell Therapy Space in 2018
  • 3.6. Top 15 Cell Therapy Companies

4. GENE-MODIFIED CELL THERAPY & IMMUNOTHERAPIES

  • 4.1. Number of Companies Developing Gene-Modified Cell Therapy Products
  • 4.2. CAR-T Cell Therapy
    • 4.2.1. Global Distribution of CAR-T Cell Therapy Companies
    • 4.2.2. Select Events in Cell-Based Immuno-Onclogy Space, 2018
  • 4.3. T-Cell Receptor (TCR) Therapy
  • 4.4. Natural Killer (NK) Cell Therapies
  • 4.5. Tumor Infiltrating Lymphocytes (TILs) Therapy
  • 4.6. Marrow-Infiltrating Lymphocytes (MILs)
  • 4.7. Gamma-Delta T-Cells Therapy
    • 4.7.1. Big Biopharma's Interest in Gamma-Delta T-Cells Therapy
  • 4.8. Dendritic Cell (DC) Therapy
    • 4.8.1. Cancer Types Targeted by Dendritic Cell Therapy Studies
    • 4.8.2. Ongoing Clinical Trials Testing DC Vaccines

5. GENE THERAPY

  • 5.1. Gene Augmentation Therapy
  • 5.2. Gene Inhibition Therapy
  • 5.3. Suicide Gene Therapy
  • 5.4. The Process of DNA Transfer in Gene Therapy
  • 5.5. Ex vivo vs. in vivo Gene Therapies
    • 5.5.1. Ex vivo Gene Therapy
    • 5.5.2. In vivo Gene Therapy
  • 5.6. Autologous vs. Allogeneic Gene Therapies in Development
  • 5.7. Number of Gene Therapy Companies in the World
  • 5.8. Gene Therapy Pipeline Volumes, 1995-2018
  • 5.9. Big Pharma and Cell Therapy/Gene Therapy Development
  • 5.10. Number of Gene Therapy Products Predicted to be Approved in Next Three Years
  • 5.11. Gene Therapy Product Candidates Granted RMAT Designation
  • 5.12. Gene Therapy Companies Active in Neurodegenerative Disorders (ND)
    • 5.12.1. Gene Therapy Developers for Neurodegenerative Diseases
    • 5.12.2. Neurodegenerative Disorders Addressed by Gene Therapy Studies
    • 5.12.3. Anticipated Events in Neurodegenerative Disease Space of Gene Therapy
    • 5.12.4. Important Events in Gene Therapy Space in 2018
  • 5.13. Leading Gene Therapy StartUps

6. TISSUE ENGINEERING

  • 6.1. Smart Biomaterials in TE
    • 6.1.1. Biosynthetic Materials
    • 6.1.2. 3D Printable Inks
  • 6.2. Synthetic and Naturally-Derived Scaffolds
  • 6.3. Tissue Substitutes
    • 6.3.1. Cell-Based Tissues
    • 6.3.2. Collagen
  • 6.4. Two Key Developments in Tissue Engineering Space in 2018

7. GENE DELIVERY VEHICLES

  • 7.1. Viral Vectors
    • 7.1.1. Physical Properties of Commonly used Viral Vectors
    • 7.1.2. Retrovirus Vectors
    • 7.1.3. Adenovirus Vectors
    • 7.1.4. Herpes Simplex Viral Vectors
    • 7.1.5. Lentivirus Vectors (LV)
      • 7.1.5.1. Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
      • 7.1.5.2. Companies Developing Lentivirus Vectors
    • 7.1.6. Adeno-Associated Virus Vectors
      • 7.1.6.1. Types of Diseases in Clinical Trials using AAV
      • 7.1.6.2. Genes Transduced by AAV for Specific Diseases
      • 7.1.6.3. Companies Developing AAV Vector
      • 7.1.6.4. The Predominantly-Used Viral Vectors
  • 7.2. Non-Viral Vectors
    • 7.2.1. Microinjection
    • 7.2.2. Particle Bombardment
    • 7.2.3. Laser Iradiation
    • 7.2.4. Electroporation
    • 7.2.5. Magnetofection
    • 7.2.6. Sonoporation

8. RM: CLINICAL TRIAL LANDSCAPE

  • 8.1. Number of People Participating in RM Clinical Trials
  • 8.2. Clinical Trials by Therapeutic Category in the RM Sector, 2018
  • 8.3. Cell Therapy Clinical Trials
    • 8.3.1. Cell Therapy Clinical Trials by Indication
    • 8.3.2. Mesenchymal Stem Cells in Clinical Trials
    • 8.3.3. Anticipated Clinical Events in Cell Therapy Space
      • 8.3.3.1. Remestemcel-L
      • 8.3.3.2. Tab-Cel
      • 8.3.3.3. MultiStem
  • 8.4. Clinical Trials in Gene-Modified Cell Therapies Space
    • 8.4.1. Most Popular Target Antigens for CAR-T Cell Therapies
    • 8.4.2. Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
    • 8.4.3. Antigens Targeted by CAR-T in Solid Cancers in Clinical Trials
    • 8.4.4. Anticipated Developments in CAR-T Space
  • 8.5. Number of Gene Therapy Clinical Trials
    • 8.5.1. Therapy Clinical Trials by Geography
    • 8.5.2. Diseases Targeted by Gene Therapy
    • 8.5.3. Gene Types Transduced in Clinical Trials
    • 8.5.4. Vectors used in Gene Therapy Clinical Trials
    • 8.5.5. Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
    • 8.5.6. Domination of Oncology in Gene Therapy Drug Development
    • 8.5.7. Dominance of Viral Vectors in Gene Therapy
    • 8.5.8. Gene Therapy Alliances Volume and Value, 2012-2018
    • 8.5.9. Anticipated Clinical Events in Gene Therapy Space, 2019/2020
  • 8.6. Number of Tissue Engineering Clinical Trials
    • 8.6.1. Anticipated Clinical Events in Tissue Engineering Space

9. APPROVED RM PRODUCTS WORLDWIDE

  • 9.1. Number of RM Products Approved by Geography
  • 9.1. Approval of RM Products by Year
  • 9.2. RM Products with MA Worldwide by Therapy Type
    • 9.2.1. Indications Addressed by the Approved RM Products
  • 9.3. Approved RM Products in Australia
    • 9.3.1. Ortho-ACI
  • 9.4. RM Products Approved in Canada
  • 9.5. Gene Therapy Product Approved in China
  • 9.6. Approved RM Products in Europe
    • 9.6.1. Yescarta (Axicabtagene ciloleucel)
    • 9.6.2. Kymriah (Tisagenlecleucel)
    • 9.6.3. Alofisel
    • 9.6.4. Spherox
    • 9.6.5. Zalmoxix
    • 9.6.6. Strimvelis
    • 9.6.7. Imlygic (Talimogene laherparepvec)
    • 9.6.8. Holoclar
    • 9.6.9. RM Products with Withdrawn Approvals in Europe
  • 9.7. Approved RM Products in India
    • 9.7.1. Cartigrow
    • 9.7.2. Ossgrow
    • 9.7.3. Apceden
    • 9.7.4. Stempeucel
  • 9.8. RM Products Approved in Japan
    • 9.8.1. TEMCELL HS Inj.
    • 9.8.2. HeartSheet
    • 9.8.3. JACC
  • 9.9. RM Products Approved in New Zealand
    • 9.9.1. Prochymal
  • 9.10. RM Products Approved in South Korea
    • 9.10.1. KeraHeal-Allo
    • 9.10.2. Neuronata-R
    • 9.10.3. Cupistem
    • 9.10.4. Cartistem
    • 9.10.5. Cellgram-AMI
    • 9.10.6. CureSkin Inj.
    • 9.10.7. Queencell
    • 9.10.8. Kaloderm
    • 9.10.9. RMS Ossron
    • 9.10.10. Immuncell LC
    • 9.10.11. CreaVax-RCC
    • 9.10.12. Kera Heal
    • 9.10.13. Chondron
  • 9.11. Approved RM Products in the U.S.
    • 9.11.1. HPC, Cord Blood
    • 9.11.2. Luxturna (voretigene neparvovec-rzyl)
      • 9.11.2.1. Mechanism of Action
    • 9.11.3. Yescarta (axicabtagene ciloleucel)
      • 9.11.3.1. Mechanism of Action
    • 9.11.4. Kymriah (tisagenlecleucel)
      • 9.11.4.1. Mechanism of Action
      • 9.11.5. MACI
    • 9.11.6. Clevecord
    • 9.11.7. HPC, Cord Blood (Bloodworks)
    • 9.11.8. Imlygic (talimogenelaherparepvec)
    • 9.11.9. HPC, Cord Blood (LifeSouth Community Blood Centers)
    • 9.11.10. Allocord
    • 9.11.11. Ducord
    • 9.11.12. HPC, Cord Blood (Clinimmune Labs)
    • 9.11.13. Gintuit
    • 9.11.14. Hemacord
    • 9.11.15. Laviv (Azficel-T)
    • 9.11.16. Provenge (Sipuleucel-T)
  • 9.12. U.S. RM Products with RMAT Designation
  • 9.13. Pricing of RM Products
  • 9.14. RM Products Expected to be Approved in Late 2019 or 2020

10. MANUFACTURING OF CELLS FOR CELL AND GENE THERAPIES

  • 10.1. Cost of Manufacture
    • 10.1.1. Cost of Manufacture in a Partially-Automated Facility
    • 10.1.2. Cost of Manufacture in a Fully-Automated Facility
    • 10.1.3. Net Present Cost (NPC) of the Three Methods
  • 10.2. In-House Gene Therapy Manufacturing Capabilities of Select Big and Mid Pharma
  • 10.3. External Gene Therapy Manufacturing by Big and Mid Pharma
  • 10.4. Cell and Gene Therapy Manufacturing Capacities in North America
  • 10.5. Cell and Gene Therapy Manufacturing Capacities in Europe

11. CDMOs & CDMOs

  • 11.1. Appropriate Phase for Outsourcing
  • 11.2. Important Points Condirered while Fixing a CMO/CDMO
  • 11.3. In-House vs. Outsourcing
  • 11.4. Number of Cell Gene Therapy CMOs/CDMOs in Europe
  • 11.5. Projected Growth of CMOs and CDMOs
  • 11.6. Projected Growth of Cell Manufacturing Capacity

12. MARKET ANALYSIS

  • 12.1. Global Market for RM Products
    • 12.1.1. Global RM Products Market by Geography
  • 12.2. Global Market for Cell Therapy Products
  • 12.3. Global Market for Gene Therapy Products
    • 12.3.1. Competitive Landscape in Gene-Modified Cell Therapy Products
    • 12.3.2. Global Market for Viral Vector and Plasmid DNA Manufacturing
  • 12.4. Global Market for Tissue-Engineered Products

13. COMPANY PROFILES

  • 13.1. 4D Molecular Therapeutics, LLC
    • 13.1.1. Key Capabilities
  • 13.2. Abeona Therapeutics, Inc.
  • 13.3. Adaptimmune Therapeutics plc
  • 13.4. Admedus Ltd.
    • 13.4.1. CardioCel
    • 13.4.2. CardioCel 3D
    • 13.4.3. VascuCel
  • 13.5. Advanced Bioscience Laboratories, Inc.
    • 13.5.1. Biomanufacturing Services
      • 13.5.1.1. Manufacturing of Virus-Based Products
      • 13.5.1.2. Manufacturing of Protein-Based Products
    • 13.5.2. Aseptic Filling Services
    • 13.5.3. Immunology and Biomarker Testing
    • 13.5.4. Contract Research Assays
    • 13.5.5. Clinical Immunology Solutions
    • 13.5.6. Preclinical Solutions
    • 13.5.7. In vivo and ex vivo Preclinical Studies
    • 13.5.8. Product Development Solutions
    • 13.5.9. Government Partnering
    • 13.5.10. Translational Product Development
  • 13.6. Adverum Biotechnologies, Inc.
  • 13.7. Akouos, Inc.
  • 13.8. Aldevron, LLC
    • 13.8.1. Services
  • 13.9. Allogene Therapeutics, Inc.
  • 13.10. AlloSource, Inc.
  • 13.11. Amarna Therapeutics B.V.
    • 13.11.1. Simian Virus 40 (SVac) Platform
  • 13.12. Ambys Medicines, Inc.
  • 13.13. American Gene Technologies International, Inc.
  • 13.14. Amgen, Inc.
    • 13.14.1. IMLYGIC (Talimogene laherparepvec)
  • 13.15. Amicus Therapeutics, Inc.
  • 13.16. Anterogen Co., Ltd.
    • 13.16.1. Cupistem Injection
    • 13.16.2. Queencell
  • 13.17. APAC Biotech Pvt., Ltd.
    • 13.17.1. APCEDEN
  • 13.18. apceth Biopharma GmbH
  • 13.19. AskBio/Asclepios Biopharmaceutical, Inc.
  • 13.20. Asterias Biotherapeutics, Inc.
  • 13.21. Atara Biotherapeutics, Inc.
  • 13.22. Athersys, Inc.
    • 13.22.1. MultiStem
  • 13.23. ATVIO Biotech Ltd.
  • 13.24. Audentes Therapeutics, Inc.
    • 13.24.1. AT132 for X-Linked Myotubular Myopathy
    • 13.24.2. AT342 for Crigler-Najjar Syndrome
    • 13.24.3. AT982 for Pompe Disease
    • 13.24.4. AT307 for CASQ2-CPVT
  • 13.25. AveXis, Inc.
    • 13.25.1. AVXS-101
  • 13.26. Avita Medical Ltd.
    • 13.26.1. RECELL System
    • 13.26.2. ReGenerCell
    • 13.26.3. ReNovaCell
  • 13.27. AVROBIO, INC.
    • 13.27.1. AVR-RD-01 for Fabry Disease
    • 13.27.2. AVR-RD-02 for Gaucher Disease
    • 13.27.3. AVR-RD-03 for Pompe Disease
    • 13.27.4. AVR-RD-04 for Cystinosis
  • 13.28. Batavia Bioscience B.V.
    • 13.28.1. Research Services
    • 13.28.2. Development Services
    • 13.28.3. Characterization Services
    • 13.28.4. Manufacturing Services
    • 13.28.5. Consultancy Services
  • 13.29. Bellicum Pharmaceuticals, Inc.
  • 13.30. Benitec Biopharma Limited
  • 13.31. Betalin Therapeutics
    • 13.31.1. Engineered Micro-Pancreas (EMP)
  • 13.32. BioCardia, Inc.
  • 13.33. Biomay AG
    • 13.33.1. Biomay's Pipeline
  • 13.34. BioReliance Corporation
  • 13.35. Biosolution Co., Ltd.
  • 13.36. Biostage, Inc.
    • 13.36.1. Cellspan Esophageal Implants
    • 13.36.2. Cellspan Bronchial Implants
    • 13.36.3. Cellspan Tracheal Implants
  • 13.37. Bioverativ, Inc.
  • 13.38. Biovian Oy
  • 13.39. bluebird bio, Inc.
    • 13.39.1. Partnering
    • 13.39.2. Current Partners
  • 13.40. BlueRock Therapeutics
    • 13.40.1. BlueRock's Pipeline
  • 13.41. Bone Therapeutics SA
    • 13.41.1. ALLOB
    • 13.41.2. JTA-004
  • 13.42. Brainstorm Cell Therapeutics, Inc.
    • 13.42.1. NurOwn
  • 13.43. Brammer Bio, LLC
    • 13.43.1. Services
    • 13.43.2. Process Development
    • 13.43.3. Gene Therapy Manufacturing
    • 13.43.4. Viral Vectors
  • 13.44. Caladrius Biosciences, Inc.
  • 13.45. Carina Biotech
    • 13.45.1. CNA1003
  • 13.46. CARsgen Therapeutics, Ltd.
  • 13.47. Casebia Therapeutics
  • 13.48. Cell Cure Neurociences Ltd.
    • 13.48.1. OpRegen
  • 13.49. Cellerant Therapeutics, Inc.
  • 13.50. Cell Medica Limited
  • 13.51. Cell Therapy Catapult, Ltd.
    • 13.51.1. Capabilities
  • 13.52. Cellular Biomedicine Group, Inc.
  • 13.53. Cellular Dynamics International, Inc.
  • 13.54. Celonic AG
  • 13.55. Celyad SA
  • 13.56. Cevec Pharmaceuticals GmbH
    • 13.56.1. Services
  • 13.57. Farmaceutici S.p.A.
    • 13.57.1. Holoclar
  • 13.58. Cobra Biologics Ltd.
    • 13.58.1. DNA Platform Process, Development and Scale Up
  • 13.58.2. Viral Vector Platform Process
  • 13.59. co-don AG
    • 13.59.1. ACT3D
  • 13.60. CombiGene AB
    • 13.60.1. Epilepsy Project
  • 13.61. Corestem, Inc.
    • 13.61.1. NEURONATA-R inj.
  • 13.62. CRISPR Therapeutics AG
  • 13.63. CureVac
  • 13.64. Cynata Therapeutics, Ltd.
  • 13.65. CytomX Therapeutics, Inc.
  • 13.66. Cytori Therapeutics, Inc.
  • 13.67. Delphi Genetics S.A.
    • 13.67.1. Services
  • 13.68. Dendreon Pharmaceuticals, Inc.
    • 13.68.1. PROVENGE
  • 13.69. DNAlite Therapeutics, Inc.
  • 13.70. Editas Medicine, Inc.
  • 13.71. Elixirgen Therapeutics, LLC
  • 13.72. Enzyvant, Inc.
    • 13.72.1. RVT-802
  • 13.73. Eukarys
  • 13.74. Eureka Therapeutics, Inc.
    • 13.74.1. ARTEMIS
    • 13.74.2. E-ALPHA
  • 13.75. Fate Therapeautics, Inc.
  • 13.76. Fibrocell Technologies, Inc.
    • 13.76.1. Fibrocell's Pipeline
  • 13.77. FinVector Vision Therapies Oy
    • 13.77.1. Services
  • 13.78. Freeline Therapeutics Ltd.
  • 13.79. FUJIFILM Diosynth Biotechnologies, Inc.
    • 13.79.1. Services
  • 13.80. Gamida Cell Ltd.
  • 13.81. GC Cell
  • 13.82. Gemini Therapeutics, Inc.
  • 13.83. GEG Tech
    • 13.83.1. Lenti-ONE Vectors
  • 13.84. Genethon
  • 13.85. Generation Bio Co.
  • 13.86. Genzyme Corporation
    • 13.86.1. Epicel
  • 13.87. Glycostem Therapeutics BV
    • 13.87.1. Technology
    • 13.87.2. oNKord
  • 13.88. Hemostemix, Inc.
  • 13.89. Histogenics Corporation
  • 13.96. Homology Medicines, Inc.
    • 13.96.1. Gene Editing Platform
  • 13.97. Horama SAS
  • 13.98. Humacyte, Inc.
  • 13.99. Immune Design Corporation
  • 13.100. Immusoft Corporation
    • 13.100.1. ISP Technology
  • 13.101. Innovative Cellular Therapeutics Co., Ltd.
  • 13.102. Intellia Therapeutics, Inc.
  • 13.103. Iovance Biotherapeutics, Inc.
  • 13.104. JCR Pharmaceuticals Co., Ltd.
    • 13.104.1. TEMCELL HS Inj.
  • 13.105. J-TEC
  • 13.106. JW CreaGene Co., Ltd.
    • 13.106.1. Products
  • 13.107. Kadimastem Ltd.
    • 13.107.1. AstroRx
    • 13.107.2. Incapsulin
  • 13.108. Kaneka Eurogentec S.A.
  • 13.109. KBI Biopharma, Inc.
  • 13.110. Kiadis Pharma N.V.
    • 13.110.1. ATIR101
  • 13.111. Kite Pharma, Inc.
    • 13.111.1. Yescarta
  • 13.112. Kolon Lifescience, Inc.
    • 13.112.1. Invossa-K inj.
  • 13.113. Krystal Biotech, Inc.
  • 13.114. LakePharma
    • 13.114.1. Viral Vector Services
  • 13.115. Lion TCR Pte. Ltd.
  • 13.116. Lonza Group Ltd.
    • 13.116.1. Custom Development and Manufacturing
  • 13.117. Lysogene S.A.
    • 13.117.1. MPS IIIA
    • 13.117.2. GM1 Gangliosidosis
  • 13.118. Magenta Therapeutics, Inc.
    • 13.118.1. Magenta's Programs
      • 13.118.1.1. Transplant Conditioning
      • 13.118.1.2. Stem Cell Expansion
      • 13.118.1.3. Stem Cell Harvesting
      • 13.118.1.4. Post Transplant Complications
  • 13.119. MassBiologics
  • 13.120. MaxCyte, Inc.
    • 13.120.1. Products
      • 13.120.1.1. MaxCyte STX
      • 13.120.1.2. MaxCyte VLX
      • 13.120.1.3. MaxCyte GT
  • 13.121. Medigene AG
  • 13.122. MEDIPOST CO., Ltd.
    • 13.122.1. CARTISTEM
    • 13.123.2. PNEUMOSTEM
    • 13.123.3. NEUROSTEM
  • 13.124. MeiraGTx Holdings plc
    • 13.124.1. Meira's Pipeline
    • 13.124.2. AAV-CNGB3
    • 13.124.3. AAV-RPE65
    • 13.124.4. AAV-RPGR
    • 13.124.5. AAV-AQP1
    • 13.124.6. AAV-GAD
    • 13.124.7. AAV-UPF1
  • 13.125. Mesoblast Limited
  • 13.126. Miromatrix Medical, Inc.
    • 13.126.1. MIRODERM
    • 13.126.2. MICROMESH
  • 13.127. MolMed S.p.A
    • 13.127.1. MolMed's Clinical Trial Programs
    • 13.127.2. MolMed's GMP Solutions
  • 13.128. multimmune GmbH
    • 13.128.1. Platform Technology
  • 13.129. Mustang Bio, Inc.
    • 13.129.1. MB-101
    • 13.129.2. MB-102
    • 13.129.3. MB-103
    • 13.129.4. MB-104
    • 13.129.5. MB-105
    • 13.129.6. MB-106
    • 13.129.7. MB-107
  • 13.130. Myonexus Therapeutics, Inc.
  • 13.131. NantKwest, Inc.
  • 13.132. NexImmune, Inc.
  • 13.133. Nightstar Therapeutics plc
  • 13.134. Neuralstem, Inc.
  • 13.135. Nohla Therapeutics, Inc.
  • 13.136. Novartis AG
    • 13.136.1. Kymriah
  • 13.137. Novasep Holding SAS
    • 13.137.1. Biopharmaceutical Manufacturing Solutions
  • 13.138. Noveome Biotherapeutics, Inc.
  • 13.139. NuVasive, Inc.
    • 13.139.1. Osteocel
    • 13.139.2. AttraX Scaffold
  • 13.140. Orchard Therapeutics plc
    • 13.140.1. Strimvelis
    • 13.140.2. OTL-101
    • 13.140.3. OTL-102
    • 13.140.4. OTL-103
    • 13.140.5. OTL-200
    • 13.140.6. OTL-201
    • 13.140.7. OTL-202
    • 13.140.8. OTL-300
  • 13.141. Orgenesis, Inc.
    • 13.141.1. MaSTHerCell
  • 13.142. Orthocell Limited
  • 13.143. Osiris Therapeutics, Inc.
    • 13.143.1. Grafix
    • 13.143.2. Grafix PL PRIME
    • 13.143.3. Cartiform
    • 13.143.4. BIO4
  • 13.144. Orthofix Medical, Inc.
    • 13.144.1. Trinity ELITE
    • 13.144.2. Trinity EVOLUTION
    • 13.144.3. VersaShield
  • 13.145. Oxford BioMedica plc
    • 13.145.1. Oxford BioMedica's Partnering Activities
  • 13.146. Oxford Genetics Ltd.
    • 13.146.1. Scalable AAV Custom Solutions
  • 13.147. Paragon Bioservices, Inc.
    • 13.147.1. Services
  • 13.148. Pharmicell Co., Ltd.
    • 13.148.1. Cellgram AMI
  • 13.149. Pluristem Therapeutics, Inc.
  • 13.150. Posedia Therapeutics, Inc.
  • 13.151. Precision BioSciences, Inc.
    • 13.151.1. ARCUS Genome Editing Platform
  • 13.152. Prevail Therapeutics, Inc.
    • 13.152.1. Prevail's Focus
  • 13.153. PROMETHERA Biosciences S.A.
  • 13.154. PTC Therapeutics, Inc.
  • 13.155. Quethera Ltd.
    • 13.155.1. Technology
  • 13.156. Recipharm AB
  • 13.157. Regenerative Medical System (RMS)
    • 13.157.1. Chondron
    • 13.157.2. Ossron
  • 13.158. REGENXBIO, Inc.
    • 13.158.1. NAV Technology
    • 13.158.2. Therapeutic Programs
    • 13.158.3. Product Candidates
      • 13.158.3.1. RGX-314
      • 13.158.3.2. RGX-501
      • 13.158.3.3. RGX-111
      • 13.158.3.4. RGX-121
      • 13.158.3.5. RGX-181
    • 13.158.4. Partnerships and Licensing
  • 13.159. ReNeuron Group plc
  • 13.160. Renova Therapeutics, Inc.
  • 13.161. Richter-Helm BioLogics GmbH & Co. KG.
    • 13.161.1. Process Development
    • 13.161.2. Analytics
    • 13.161.3. Production
  • 13.162. Rocket Pharmaceuticals, Inc.
  • 13.163. RoosterBio, Inc.
    • 13.163.1. MSCs in different Formats
    • 13.163.2. Bioprocess Media
    • 13.163.3. Plug & Play Systems
    • 13.163.4. CliniControl Products
  • 13.164. RoslinCT
    • 13.164.1. Services
  • 13.165. Rubius Therapeutics, Inc.
  • 13.166. Sangamo Therapeutics, Inc.
  • 13.167. Sarepta Therapeutics, Inc.
  • 13.168. SCM Lifesciences Co., Ltd.
  • 13.169. Semma Therapeutics Inc.
    • 13.169.1. SCbeta Cells
  • 13.170. Sentien Biotechnologies, Inc.
    • 13.170.1. SBI-101
  • 13.171. Sibiono GeneTech Co., Ltd.
    • 13.171.1. Gendicine
  • 13.172. Sirion Biotech GmbH
    • 13.172.1. Viral Vectors
    • 13.172.2. Technologies
  • 13.173. Solid Biosciences, Inc.
    • 13.173.1. SGT-001 (Microdystrophin Gene Transfer)
  • 13.174. Spark Therapeutics, Inc.
    • 13.174.1. Luxturna (voretigene neparvovec)
    • 13.174.2. Spark Therapeutics' Pipeline
  • 13.175. SQZ Biotechnologies
    • 13.175.1. SQZ Platform
  • 13.176. Stempeutics Research Pvt., Ltd.
    • 13.176.1. Stempeucel
    • 13.176.2. Stempeucare
    • 13.176.3. Stempeutron
  • 13.177. Synpromics Ltd.
    • 13.177.1. Technology
    • 13.177.2. PromPT
  • 13.178. Takara Bio, Inc.
    • 13.178.1. Services and Support
  • 13.179. Terumo Corporation
    • 13.179.1. HeartSheet
  • 13.180. The Cell and Gene Therapy Catapult
    • 13.180.1. Catapult's Industrialization Team
    • 13.180.2. Catapult's Manufacturing Center at Stevenage
    • 13.180.3. Catapult's Regulatory Team
  • 13.181. Translate Bio, Inc.
  • 13.182. Triumvira Immunologics USA, Inc.
  • 13.183. TxCell S.A.
    • 13.183.1. Technology
    • 13.183.2. CAR-Treg Transplantation (TX200)
    • 13.183.3. CAR-Treg Dermatology
    • 13.183.4. CAR-Treg Multiple Sclerosis
  • 13.184. Ultragenyx Pharmaceutical, Inc.
    • 13.184.1. DTX301
    • 13.184.2. DTX401
  • 13.185. uniQure N.V.
  • 13.186. Vericel Corporation
    • 13.186.1. MACI
    • 13.186.2. Epicel
  • 13.187. Vertex Pharmaceuticals, Inc.
  • 13.188. VGXI, Inc.
  • 13.189. ViaCyte, Inc.
    • 13.189.1. Product Candidates
      • 13.189.1.1. PEC-Direct
      • 13.189.1.2. PEC-Encap
  • 13.190. Vibalogics GmbH
  • 13.191. Vybion, Inc.
    • 13.191.1. INT41
  • 13.192. Vigene Biosciences
  • 13.193. Virovek, Inc.
    • 13.193.1. Custom-Made AAVs
  • 13.194. Vivebiotech SL
    • 13.194.1. Lentisoma Technology
    • 13.194.2. Services
  • 13.195. Vivet Therapeutics
    • 13.195.1. Vivet's Approach
  • 13.196. Voyager Therapeutics, Inc.
  • 13.197. Waisman Biomanufacturing
    • 13.197.1. Services and Products
  • 13.198. WindMIL Therapeutics, Inc.
    • 13.198.1. Unmodified MILs
    • 13.198.2. MIL CARs
    • 13.198.3. genMILs
  • 13.199. Wuxi App Tec, Inc.
    • 13.199.1. Services
  • 13.200. Xalud Therapeutics, Inc.
    • 13.200.1. XT-101

INDEX OF FIGURES

  • FIGURE 2.1: Number of RM Companies by Geography, 2018
  • FIGURE 2.2: Total RM Clinical Trials by Phase, 2017-2018
  • FIGURE 2.3: Total RM Clinical Trials by Technology Type
  • FIGURE 2.4: RM Industry-Total Global Financings: 2018
  • FIGURE 2.5: Total Financings to RM Industry by Type and Year, 2016-2018
  • FIGURE 2.6: Total Merger & Acquisition within RM Industry by Year, 2016-2018
  • FIGURE 2.7: Predicted CGT Product Launches, 2018-2030
  • FIGURE 2.8: Predicted Treated Patient Numbers, 2018-2030
  • FIGURE 3.1: Number of Clinical Trials using MSCs by Phase of Development
  • FIGURE 3.2: Percent (%) Share of Indications using MSCs in Clinical Trials
  • FIGURE 3.3: Types of Diseases Targeted by Neural Stem Cells in Clinical Trials
  • FIGURE 4.1: Geographical Distribution of CAR-T Cell Therapy Companies
  • FIGURE 4.2: Cancer Types Targeted by Dendritic Cell Therapy Studies
  • FIGURE 5.1: Diagrammatic Representation of Gene Augmentation Therapy
  • FIGURE 5.2: Schematic Representation of Gene Inhibition Therapy
  • FIGURE 5.3: Diagrammatic Representation of Therapy by Suicide Genes
  • FIGURE 5.4: Illustration Showing the Viral Vector Transferring the Gene into Patient's Cell
  • FIGURE 5.5: Autologous vs. Allogeneic Gene Therapies in Development
  • FIGURE 5.6: Number of Gene and Gene-Based Medicine Companies by Geography
  • FIGURE 5.7: Gene Therapy Pipeline Growth by Year
  • FIGURE 5.8: Minor Role Played by Big Pharma in Cell/Gene Therapy Development
  • FIGURE 5.9: Number of Gene Therapy Products to Gain FDA Approval by 2022
  • FIGURE 5.10: Gene Therapy Companies Active in Neurodegenerative Diseases by Geography
  • FIGURE 7.1: Utilization of Lentivirus Vectors by Disease Type
  • FIGURE 7.2: Percent Shares of Companies in Lentiviral Vector Market
  • FIGURE 7.3: Types of Diseases in Clinical Trials using AAV
  • FIGURE 7.4: Percent Shares of Companies in AAV Vector Market
  • FIGURE 8.1: Number of Clinical Trials in RM Sector, 2017-2018
  • FIGURE 8.2: Number of Patient Participation in RM Clinical Trials, 2018
  • FIGURE 8.3: Number of Cell Therapy Clinical Trials, 2017-2018
  • FIGURE 8.4: Cell Therapy Clinical Trials by Indication, 2018
  • FIGURE 8.5: Clinical Trials using MSCs by Indication
  • FIGURE 8.6: Number of Gene-Modified Cell Therapy Clinical Trials, 2017-2018
  • FIGURE 8.7: The Popular Target Antigens for CAR-T in Clinical Trials
  • FIGURE 8.8: Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
  • FIGURE 8.9: Antigens Targeted by CAR-T in Solid Cancers in Clinical Trials
  • FIGURE 8.10: Number of Gene Therapy Clinical Trials, 2017-2018
  • FIGURE 8.11: Gene Therapy Clinical Trials by Geography
  • FIGURE 8.12: Diseases Targeted by Gene Therapy, 2018
  • FIGURE 8.13: Gene Types Transduced in Clinical Trials, 2018
  • FIGURE 8.14: Vectors used in Gene Therapy Clinical Trials, 2018
  • FIGURE 8.15: Gene Therapy for Neurodegenerative Indications by Type, 2018
  • FIGURE 8.16: Domination of Oncology in Gene Therapy Drug Development
  • FIGURE 8.17: Domination of Viral Vectors in Gene Therapy Drug Development
  • FIGURE 8.18: Gene Therapy Alliances Volume and Value, 2012-2018
  • FIGURE 8.19: Number of Clinical Trials in TE Space, 2017-2018
  • FIGURE 9.1: Number of RM Products with MA by Region/Country
  • FIGURE 9.2: Type of RM Products with MA Worldwide by Product Type, 2018
  • FIGURE 9.3: RM Products with MA Worldwide by Therapy Type, 2018
  • FIGURE 9.4: Number of RM Products with MA Worldwide by Indication, 2018
  • FIGURE 10.1: Expenditure Categories in Cell Products Manufacturing
  • FIGURE 10.2: The Impact of Head Count on Manufascturing Cost
  • FIGURE 10.3: Cost of Production in a Partially Automated Facility
  • FIGURE 10.4: Cost of Production in a Fully Automated Facility
  • FIGURE 10.5: NPC of Manual, Partially Automated and Fully Automated Processes
  • FIGURE 11.1: The Appropriate Phase for Outsourcing a Cell or Gene Therapy Project
  • FIGURE 11.2: Important Points Considered while Fixing a CDMO
  • FIGURE 11.3: Share of In-House and CDMO Manufacture of Cell and Gene Therapies
  • FIGURE 11.4: Number of Cell Gene Therapy CMOs/CDMOs in Europe
  • FIGURE 11.5: Projected Growth of Outsourcing Market, 2018-2025
  • FIGURE 11.6: Projected Growth of Cell Manufacturing Capacity, 2018-2025
  • FIGURE 12.1: Global Market for Regenerative Medicinal Products, 2018-2025
  • FIGURE 12.2: Global Market for RM Products by Geography, 2018-2025
  • FIGURE 12.3: Global Market for Approved Cell Therapy Products, 2018-2025
  • FIGURE 12.4: Global Market for Gene Therapy Products, 2018-2025
  • FIGURE 12.5: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
  • FIGURE 12.6: Global Market for Tissue-Engineered Products, 2018-2025

INDEX OF TABLES

  • TABLE 2.1: Number of RM Companies by Geography, 2018
  • TABLE 2.2: Major Therapeutic Platforms and Enabling Technologies in RM Industry
  • TABLE 2.3: RM Product Approvals in 2017/2018
  • TABLE 2.4: Total RM Clinical Trials by Phase, 2017/2018
  • TABLE 2.5: Total RM Clinical Trials by Technology Type
  • TABLE 2.6: Patient Impact of Three RM Products, 2018
  • TABLE 2.7: Select Anticipated RM Industry Clinical Trial Data Readouts: 2019
  • TABLE 2.7: (CONTINUED)
  • TABLE 2.7: (CONTINUED)
  • TABLE 2.8: RM Industry-Total Global Financings, 2018
  • TABLE 2.9: Total Financings to RM Industry by Type and Year, 2016-2018
  • TABLE 2.10: Corporate Partnerships (Upfront Payments) in RM Industry, 2018
  • TABLE 2.11: Private Placements and Venture Financings in RM Industry, 2018
  • TABLE 2.12: Public Offerings (IPOs & Follow-Ons) in RM Industry, 2018
  • TABLE 2.13: Total Merger & Acquisitions Within RM Industry by Year, 2015-2018
  • TABLE 2.14: Market Access for Regenerative Medicines as of January 2019
  • TABLE 2.15: Top 20 RM Companies by Pipeline Size
  • TABLE 2.16: RM Research Institutes in North America
  • TABLE 2.17: RM Research Institutes in Europe
  • TABLE 2.18: RM Research Institutes in Australia
  • TABLE 2.19: From University Research to Products in the Market
  • TABLE 3.1: Timeline of Notable Events in Cell Therapy Sector
  • TABLE 3.2: Common Indications for HSCT
  • TABLE 3.3: Leading iPS Cell Therapy Companies
  • TABLE 3.4: MSC-Based Products with Marketing Approval (MA)
  • TABLE 3.4: (CONTINUED)
  • TABLE 3.5: Select List of Companies Active in Clinical Trials using Neural Stem Cells
  • TABLE 3.6: Select Clinical Trials that Use hESCs
  • TABLE 3.7: The 29 Clinical Trials using hESCs, 2018
  • TABLE 3.8: Select Events in Cell Therapy Sector, 2018
  • TABLE 3.9: Top 15 Cell and Gene Therapy Biotechs to Watch
  • TABLE 4.1: Number of Companies Developing Gene-Modified Cell Therapy Products
  • TABLE 4.2: Geographical Distribution of CAR-T Cell Therapy Companies
  • TABLE 4.3: Select Events in Cell-Based Immuno-Onclogy Space, 2018
  • TABLE 4.4: Examples of Clinical Trials Involving T-Cell Receptors (TCRs)
  • TABLE 4.5: Select NK Cell Threrapies in Clinical and Preclinical Devrelopment for Cancer
  • TABLE 4.6: Examples of Clinical Trials using TILs
  • TABLE 4.7: Known Gamma-Delta T-Cell Therapy Companies
  • TABLE 4.8: Select Ongoing Clinical Trials Testing DC Vaccines
  • TABLE 5.1: Approved Gene Therapy Products (ex vivo and in vivo)
  • TABLE 5.2: Number of Gene and Gene-Based Medicine Companies by Geography
  • TABLE 5.3: Gene Therapy Product Candidates Granted RMAT Designation, 2018
  • TABLE 5.4: Gene Therapy Companies Active in ND by Region, 2018
  • TABLE 5.5: Gene Therapy Developers for Neurodegenerative Disorders, 2018
  • TABLE 5.6: Neurodegenerative Disorders Addressed by Gene Therapy Studies
  • TABLE 5.7: Select Anticipated Events in ND Space of Gene Therapy, 2018
  • TABLE 5.8: Select Events in Gene Therapy and Genome Editing Space, 2017/2018
  • TABLE 5.9: Leading Gene Therapy Startups
  • TABLE 6.1: Approved TE Products
  • TABLE 6.1: (CONTINUED)
  • TABLE 6.2: Select Events in Tissue Engineering Sector, 2018
  • TABLE 7.1: Attributes of Commonly used Viral Vectors
  • TABLE 7.2: Retroviruses: A Snapshot
  • TABLE 7.3: Adenoviruses: A Snapshot
  • TABLE 7.4: Herpes Simplex Viruses (HSV): A Snapshot
  • TABLE 7.5: Lentiviruses: A Snapshot
  • TABLE 7.6: Adeno-Associated Viruses: A Snapshot
  • TABLE 7.7: Genes Transduced by AAV for Specific Diseases
  • TABLE 7.8: Dominating Viral Vectors in Gene Therapy Drug Development
  • TABLE 7.9: Microinjection Method of Gene Delivery
  • TABLE 7.10: Particle Bombardment Method of Gene Delivery
  • TABLE 7.11: Laser Irradiation Method of Gene Delivery
  • TABLE 7.12: Electroporation Method of Gene Delivery
  • TABLE 7.13: Magnetofection Method of Gene Delivery
  • TABLE 7.14: Sonoporation Method of Gene Delivery
  • TABLE 8.1: Number of Clinical Trials in RM Sector, 2017-2018
  • TABLE 8.2: Number of Patient Participation in RM Clinical Trials, 2018
  • TABLE 8.3: Clinical Trials by Therapeutic Category in the RM Sector, 2018
  • TABLE 8.4: Number of Cell Therapy Clinical Trials, 2017-2018
  • TABLE 8.5: Number of Clinical Trials using MSCs
  • TABLE 8.6: Select Anticipated Clinical Data and Events in Cell Therapy Sector
  • TABLE 8.6: (CONTINUED)
  • TABLE 8.6: (CONTINUED)
  • TABLE 8.6: (CONTINUED)
  • TABLE 8.6: (CONTINUED)
  • TABLE 8.6: (CONTINUED)
  • TABLE 8.7: Number of Gene-Modified Cell Therapy Clinical Trials, 2017-2018
  • TABLE 8.8: Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
  • TABLE 8.9: Select Anticipated Clinical Data and Events in CAR-T Cell Therapy Sector
  • TABLE 8.9: (CONTINUED)
  • TABLE 8.10: Number of Gene Therapy Clinical Trials, 2017-2018
  • TABLE 8.11: Gene Therapy Clinical Trials by Geography
  • TABLE 8.12: Diseases Targeted by Gene Therapy, 2018
  • TABLE 8.13: Gene Types Transduced in Clinical Trials, 2018
  • TABLE 8.14: Vectors used in Gene Therapy Clinical Trials, 2018
  • TABLE 8.15: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type, 2018
  • TABLE 8.16: Domination of Oncology in Gene Therapy Drug Development
  • TABLE 8.17: Gene Therapy Alliances Volume and Value, 2012-2018
  • TABLE 8.18: Select Anticipated Clinical Data and Events in Gene Therapy Sector
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.18: (CONTINUED)
  • TABLE 8.19: Number of Clinical Trials in TE Space, 2017-2018
  • TABLE 8.20: Select Anticipated Clinical Data and Events in Tissue Engineering Space
  • TABLE 9.1: RM Products with MA by Region/Country
  • TABLE 9.2: RM Products with MA Worldwide by Year
  • TABLE 9.3: RM Products with MA Worldwide by Product Type, 2018
  • TABLE 9.4: RM Products with MA Worldwide by Therapy Type, 2018
  • TABLE 9.5: Number of RM Products with MA Worldwide by Indication, 2018
  • TABLE 9.6: Cell Therapy Product with MA in Australia by TGA
  • TABLE 9.7: Cell Therapy and Gene Therapy Products with MA in Canada by Health Canada
  • TABLE 9.8: Gene Therapy Product with MA in China by CFDA
  • TABLE 9.9: RM Products with MA in Europe by EMA
  • TABLE 9.9: (CONTINUED)
  • TABLE 9.10: RM Products with MA Withdrawn in the E.U. by EMA
  • TABLE 9.11: Cell Therapy Products with MA in India by DCGI
  • TABLE 9.12: Cell and Tissue Engineered Products with MA in Japan by PMDA
  • TABLE 9.13: Cell Therapy Products with MA in New Zealand by MEDSAFE
  • TABLE 9.14: RM Products with MA in South Korea by MFDS
  • TABLE 9.14: (CONTINUED)
  • TABLE 9.14: (CONTINUED)
  • TABLE 9.15: RM Products with MA in the U.S. by FDA
  • TABLE 9.15: (CONTINUED)
  • TABLE 9.15: (CONTINUED)
  • TABLE 9.15: (CONTINUED)
  • TABLE 9.16: RM Products with RMAT Designation by FDA
  • TABLE 9.16: (CONTINUED)
  • TABLE 9.16: (CONTINUED)
  • TABLE 9.16: (CONTINUED)
  • TABLE 9.17: Pricing of RM Products
  • TABLE 9.18: The Five Forthcoming RM Products
  • TABLE 9.18: (CONTINUED)
  • TABLE 10.1: Expenditure Categories in Cell Products Manufacturing
  • TABLE 10.2: The Impact of Head Count on Labor Cost
  • TABLE 10.3: Cost of Production in a Partially Automated Facility
  • TABLE 10.4: Cost of Production in a Fully Automated Facility
  • TABLE 10.5: In-House Gene Therapy Manufacturing Capabilities of Select Big and Mid Pharma
  • TABLE 10.6: External Gene Therapy Manufacturing by Big and Mid Pharma
  • TABLE 10.7: Cell and Gene Therapy Manufacturing Capacities in North America
  • TABLE 10.8: Cell and Gene Therapy Manufacturing Capacities in Europe
  • TABLE 10.8: (CONTINUED)
  • TABLE 10.8: (CONTINUED)
  • TABLE 11.1: Select CDMOs in Cell and Gene Therapy Sector
  • TABLE 11.2: The Appropriate Phase for Outsourcing a Cell or Gene Therapy Project
  • TABLE 11.3: Important Points Considered While Fixing a CDMO
  • TABLE 11.4: Share of In-House and CDMO Manufacture of Cell and Gene Therapies
  • TABLE 12.1: Global Market for RM Products by Therapy Type, 2018-2025
  • TABLE 12.2: Global Market for RM Products by Geography, 2018-2025
  • TABLE 13.3: Global Market for Approved Cell Therapy Products, 2018-2025
  • TABLE 12.4: Global Market for Gene Therapy Products, 2018-2025
  • TABLE 12.5: Competitive Landscape in Gene-Modified Cell Therapy Products
  • TABLE 12.6: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
  • TABLE 12.7: Global Market for Tissue-Engineered Products, 2018-2025
  • TABLE 13.1: 4D Molecular Therapeutics' Product Pipeline
  • TABLE 13.2: Abeona Therapeutics' Product Pipeline
  • TABLE 13.3: Adaptimmune's Pipeline Programs
  • TABLE 13.4: Allogene Therapeutics' Pipeline of Allogeneic T-Cell Product Candidates
  • TABLE 13.5: Amicus' Gene Therapy Pipeline
  • TABLE 13.6: apceth's Pipeline Overview
  • TABLE 13.7: AskBio's Therapeutic Pipeline
  • TABLE 13.8: Asterias' Pipeline Programs
  • TABLE 13.9: Atara's Pipeline of T-Cell Immunotherapy and CAR-T Product Candidates
  • TABLE 13.10: Athersys' Clinical Programs using MultiStem
  • TABLE 13.11: Audentes' Product Candidates
  • TABLE 13.12: AVROBIO's Gene Therapy Programs
  • TABLE 13.13: Bellicum Pharmaceuticals' Product Candidates
  • TABLE 13.14: Benitec's Product Pipeline using Gene Silencing Technologies
  • TABLE 13.15: BioCardia's Advanced Pipeline
  • TABLE 13.16: Biomay's Pipeline of Products
  • TABLE 13.17: Biosolution's Products and Product Candidates
  • TABLE 13.18: Bioverativ's Product Pipeline
  • TABLE 13.19: bluebird bio's Product Pipeline
  • TABLE 13.20: Brainstorm's Clinical Programs
  • TABLE 13.21: Caladrius' Proprietary Technology Platforms and Pipeline Programs
  • TABLE 13.22: CARsgen Therapeutics' Product Candidates
  • TABLE 13.23: Casebia's Pipeline at-a-glance
  • TABLE 13.24: Cellerant's Program Pipeline for Blood Disorders
  • TABLE 13.25: Cell Medica's Product Pipeline
  • TABLE 13.26: CBG's Immuno-Oncology Stem Cell Therapy Pipeline
  • TABLE 13.27: Celyad's Programs' Status at a Glance
  • TABLE 13.28: Corestem's Key Clinical Pharmaceutical Development
  • TABLE 13.29: CRISPR Therapeutics' Clinical Programs
  • TABLE 13.30: CureVac's RNA-Based Product Pipeline
  • TABLE 13.31: CytomX's Pipeline of Probody Therapeutics
  • TABLE 13.32: Cytori's Cell Therapy Development Pipeline
  • TABLE 13.33: Editas' Diverse Product Pipeline
  • TABLE 13.34: Elixirgen's Pipeline for Genetic and Age-Related Diseases
  • TABLE 13.35: Eureka's Development Pipeline
  • TABLE 13.36: Fate Therapeutics' Cell Therapy Product Candidates Pipeline
  • TABLE 13.37: Fibrocell's Personalized Biologics Pipeline
  • TABLE 13.38: Freeline's Pipeline of Gene Therapy Treatments in Development
  • TABLE 13.39: Gamida Cell's Pipeline Programs
  • TABLE 13.40: Current Status of GC Cell's Clinical Activities
  • TABLE 13.41: Gemini's Pipeline
  • TABLE 13.42: Genethon's Products in Development
  • TABLE 13.43: Hemostemix's Clinical Pipeline
  • TABLE 13.44: Horama's Product Pipeline
  • TABLE 13.45: Immune Design's Product Pipeline
  • TABLE 13.46: ICT's Pipeline Programs
  • TABLE 13.47: Intellia's In Vivo and Ex Vivo R&D Pipeline
  • TABLE 13.48: Iovance's Clinical Pipeline
  • TABLE 13.49: JCR's RM Products in Development
  • TABLE 13.50: Kite's CAR-T Pipeline
  • TABLE 13.51: Kolon's Gene Therapeutics in Development
  • TABLE 13.52: Krystal Biotech' Pipeline Programs for Orphan Skin Disease
  • TABLE 13.53: Lion TCR's Product Candidates
  • TABLE 13.54: Magenta's Product Candidates
  • TABLE 13.55: Medigene's Immunotherapy Pipeline Projects
  • TABLE 13.56: MeiraGTX's Pipeline
  • TABLE 13.57: Mesoblast's Advanced Product Candidates
  • TABLE 13.58: MolMed's Product Candidates
  • TABLE 13.59: Mustang Bio's Product Pipeline
  • TABLE 13.60: Myonexus' Clinical Stage LGMD Gene Therapy Pipeline
  • TABLE 13.61: NantKwest's Clinical Trials using Cryopreserved haNK Cells
  • TABLE 13.62: NexImmune's Adoptive Cellular Therapy (ACT) Programs
  • TABLE 13.63: Nightstar Therapeutics' Pipeline Programs
  • TABLE 13.64: Neuralstem's Cell Therapy Programs
  • TABLE 13.65: Nohla's Donor Cell Therapy Programs
  • TABLE 13.66: Noveome's Clinical Pipeline
  • TABLE 13.67: Orchard Therapeutics' Pipeline
  • TABLE 13.68: Oxford BioMedica's Proprietary Product Pipeline
  • TABLE 13.69: Oxford BioMedica's Partnered Products
  • TABLE 13.70: Oxford BioMedica's IP Enabled and Royalty Bearing Products
  • TABLE 13.71: Pharmicell's Clinical Trials for Commercialization
  • TABLE 13.72: Posedia's Product Pipeline
  • TABLE 13.73: PROMETHERA's Product Pipeline
  • TABLE 13.74: PTC Therapeutics' Gene Therapy Programs for CNS Disorders
  • TABLE 13.75: REGENXBIO's Product Pipeline
  • TABLE 13.76: REGENXBIO's Partners & Licensees Pipeline
  • TABLE 13.77: ReNeuron's Program Pipeline
  • TABLE 13.78: Renova's Product Pipeline
  • TABLE 13.79: Rubius' Pipeline Programs
  • TABLE 13.80: Sangamo's Proprietary Programs
  • TABLE 13.81: Sangamo's Proprietary Programs
  • TABLE 13.82: Sarepta's Huge Product Pipeline
  • TABLE 13.83: SCM Lifesciences' Product Pipeline
  • TABLE 13.84: Sentien's SBI-101 Clinical Programs
  • TABLE 13.85: Spark Therapeutics' Pipeline
  • TABLE 13.86: Translate Bio's Two Lead Product Candidates
  • TABLE 13.87: Triumvira's Product Pipeline
  • TABLE 13.88: uniQure's Product Pipeline
  • TABLE 13.89: Vertex Pharmaceuticals' Product Candidates
  • TABLE 13.90: ViaCyte's Product Pipeline
  • TABLE 13.91: Vybion's Drug Pipeline
  • TABLE 13.92: Voyager's Pipeline Programs
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