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市场调查报告书

基因治疗全球市场 - 产品研发、市场规模、预测

Global Market for Gene Therapy 2019 - Product Pipeline, Market Size, & Forecasts

出版商 BIOINFORMANT WORLDWIDE, LLC 商品编码 893717
出版日期 内容资讯 英文 263 Pages
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基因治疗全球市场 - 产品研发、市场规模、预测 Global Market for Gene Therapy 2019 - Product Pipeline, Market Size, & Forecasts
出版日期: 2019年08月01日内容资讯: 英文 263 Pages
简介

本报告调查分析了全球基因治疗市场,并针对市场概要、新兴市场动向、主要企业、产品、专利情势、临床实验情势等提供系统性资讯。

第1章 报告概要

第2章 基因治疗:简介

  • 基因增强治疗
  • 基因抑制治疗
  • 自杀基因治疗
  • 基因治疗中引进基因的流程
  • 体外 vs. 体内的基因治疗等

第3章 基因治疗产业情况

  • 全球基因治疗企业数量
  • 基因治疗研发数量
  • 大型制药公司参与基因治疗开发
  • 未来3年预期将被认可的基因治疗数量
  • 基因治疗候选产品等

第4章 基因输送载体

  • 病毒载体
  • 逆转录病毒载体
  • 腺病毒载体
  • 单纯疱疹病毒载体
  • 慢病毒属载体,等

第5章 基因治疗临床实验

  • 基因治疗临床实验:地区别
  • 基因治疗临床实验中做为目标的疾病
  • 基因治疗临床实验中使用的载体
  • 针对神经退行性疾病的基因治疗临床实验:种类别,等

第6章 嵌合抗原受体T细胞(CAR-T细胞)疗法

  • CAR-T细胞疗法企业的地理分布
  • CAR-T领域的主要活动
  • CAR-T疗法的开发时程
  • CAR-T的生产工程
  • CAR-T细胞:作用机制

第7章 CAR-T专利情势

  • CAR-T专利数
  • CAR-T专利类型
  • CAR-T专利情势概要
  • 有关固态肿瘤的CAR-T专利
  • 主要的CAR-T专利申请

第8章 CAR-T临床实验情势

  • CAR-T临床实验数急速增加
  • CAR-T临床实验比例:目标抗原别
  • CAR-T临床实验聚焦:适应症别
  • 商用被授权人的CAR-T专案
  • 产学合作的CAR-T专案,等

第9章 提供CAR-T疗法的医疗设施

  • 美国
  • 德国
  • 英国

第10章 基因治疗产品

第11章 基因治疗价格、支付模式

第12章 细胞、病毒载体的制造

第13章 CMO、CDMO

第14章 市场分析

第15章 公司档案

目录

Gene therapy is a novel area of medicine that involves the insertion of functional genes into cells to replace faulty or missing genes in order to treat genetic diseases. This report reviews key developments from across the gene therapy sector, evaluating the industry's commercial successes and failures, regulatory issues across key global markets, manufacturing and scale-up challenges, pricing and reimbursement considerations, and requirements for long-term technology adoption. It evaluates trends in scientific publications, clinical trials, and patent activity, as well as presents market size determinations with 5-year projections.

Representing a "new frontier of medicine," gene therapy made its grand arrival in the U.S. in 2017 through the FDA approval of chimeric antigen receptor (CAR) T-cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), as well as the gene therapy Luxturna (voretigene neparvovec). Kymriah and Yescarta represent gene-modified cell therapies in which the T cells of the patient are genetically modified ex vivo by the addition of a gene and infused back into the patient's body to seek out and destroy cancer cells. Luxturna is a gene therapy product consisting of modified viral particles loaded with a therapeutic gene that is injected into the cornea of a patient to improve functional vision. These products have revolutionized the gene therapy industry, giving hope to thousands of patients suffering from incurable diseases and expanding interest in gene therapy technology adoption on a global basis. Thus far, China, Australia, New Zealand, South Korea, India, Japan, Europe, Canada, and the US. have approved gene therapy products, indicating worldwide adoption is underway.

Since 2012, hundreds of clinical trials have confirmed the efficacy and safety of gene therapy for a range of indications, including blindness, hemophilia, immunodeficiencies, inherited anemia and cancer immunotherapy with CAR-T cells. Because some of these clinical trials have successfully reached Phase III, it is anticipated that the gene therapy industry will see up to 50 or 60 approved products by 2030. The handful of currently approved gene therapies and anticipated future approvals will likely be used to treat more than a half million patients around the world by 2030.

The successful launch of gene therapy products has also lured substantial investment into the industry and attracted a growing number of market competitors, ranging from spin-offs to large conglomerates. Pharmaceutical companies are showing an appetite for gene therapy technologies, engaging in licensing agreements, R&D collaborations, and M&A deal making aimed to bring new gene therapies to market. Ranging from small to large, gene therapy companies are emerging in major healthcare markets worldwide.

The main objectives of this report are to provide the reader with the following details:

  • Approved gene therapy products in the global market.
  • Number of gene therapy products to gain FDA approval by 2024.
  • Market value of the forthcoming gene therapy products.
  • Leading gene therapy startups.
  • Select anticipated clinical data and events in gene therapy space.
  • Gene therapy products approved in Australia, New Zealand, China, South Korea, India, Japan, Europe, Canada and the U.S.
  • Gene therapy products with RMAT designation.
  • Pricing of gene therapy products.
  • Manufacturing cost of gene therapy products in manual, partially-automated and fully-automated facilities.
  • Pharma companies having in-house capability for gene therapy manufacture.
  • Big and Mid-Pharma companies relying on outsourced manufacturing of gene therapies.
  • Gene therapy manufacturing capacities in the U.S. and Europe.
  • Select CMOs/CDMOs in gene therapy space.
  • Projected outsourcing market for the manufacture of gene therapy products, 2018-2025.
  • Global market for gene therapy products by therapy type and geography, 2018-2025.
  • Profiles of companies that are developing products for gene therapy.

In summary, this report analyzes emerging market forces, highlights industry partnerships and deal-making, and captures the progression of gene therapy products across various states of development. Because this market is rapidly expanding, this report is your guide to understanding gene therapy research advances, clinical breakthroughs, and future directions.

Claim this report to:

  • 1.Identify the preclinical and clinical product pipeline for gene therapy products globally.
  • 2.Reveal the identities of competitors developing gene therapies and supporting technologies.
  • 3.Determine company strategy or purse external funding from investors for gene therapy technologies.
  • 4.Access gene therapy market size determinations, market segmentation, and 5-year projections through 2024.
  • 5.Make smarter decisions, faster.

BioInformant, the publisher of this report, has a unique understanding of the rapidly evolving Gene Therapy (GT) Industry. Founded in 2006, BioInformant has collected more than decade of historical data on the 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 (NIH), U.S. FDA, and the Maryland Biotech Corridor, among other critical resources. This experience provides BioInformant with access to Key Opinion Leaders (KOLs) from across the gene therapy sector, including dozens of executives who were interviewed for this report.

Table of Contents

1. REPORT OVERVIEW

  • 1.1 Statement of the Report
  • 1.2 Executive Summary

2. GENE THERAPY: AN INTRODUCTION

  • 2.1 Gene Augmentation Therapy
  • 2.2 Gene Inhibition Therapy
  • 2.3 Suicide Gene Therapy
  • 2.4 The Process of Gene Transfer in Gene Therapy
  • 2.5 Ex Vivo vs. In Vivo Gene Therapies
    • 2.5.1 Ex Vivo Gene Therapy
    • 2.5.2 In Vivo Gene Therapy
  • 2.6 Autologous vs. Allogeneic Gene Therapies

3. STATUS OF GENE THERAPY INDUSTRY

  • 3.1 Number of Gene Therapy Companies in the World
  • 3.2 Gene Therapy Pipeline Volumes, 1995-2018
  • 3.3 Big Pharma's Involvement in Gene Therapy Development
  • 3.4 Number of Gene Therapies Anticipated to be Approved in the Next Three Years
  • 3.5 Gene Therapy Product Candidates Granted RMAT Designation
    • 3.5.1 EB-101
    • 3.5.2 ABO-102
    • 3.5.3 AT132
    • 3.5.4 LentiGlobin
    • 3.5.5 JCAR017
    • 3.5.6 NSR-RE P1
    • 3.5.7 P-BCMA-101
    • 3.5.8 VY-AADC
  • 3.6 Gene Therapy Companies Active in Neurodegenerative Disorders
    • 3.6.1 Gene Therapy Developers for Neurodegenerative Disorders
    • 3.6.2 Neurodegenerative Diseases Addressed by Gene Therapy Studies
    • 3.6.3 Anticipated Events in Neurodegenerative Space of Gene Therapy
  • 3.7 Important Events in Gene Therapy Space in 2018
  • 3.8 Important Gene Therapy Startups
  • 3.9 Gene Therapy Industry and Global Financings, 2018
  • 3.10 Corporate Partnerships in 2018
  • 3.11 Private Placements and Venture Financings in 2018
  • 3.12 Public Offerings (IPOs & Follow-Ons) in 2018

4. GENE DELIVERY VEHICLES

  • 4.1 Viral Vectors
    • 4.1.1 Physical Properties of Commonly Used Viruses
  • 4.2 Retrovirus Vectors
  • 4.3 Adenovirus Vectors
  • 4.4 Herpes Simplex Viral Vectors
  • 4.5 Lentiviral Vectors
    • 4.5.1 Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
    • 4.5.2 Companies Developing Lentivirus Vectors
  • 4.6 Adeno-Associated Virus Vectors.
    • 4.6.1 Types of Diseases in Clinical Trials using AAV
    • 4.6.2 Genes Transduced by AAV for Specific Diseases
    • 4.6.3 Companies Developing AAV Vectors
  • 4.7 The Predominantly Used Viral Vectors
  • 4.8 Non-Viral Vectors
    • 4.8.1 Microinjection
    • 4.8.2 Particle Bombardment
    • 4.8.3 Laser Irradiation
    • 4.8.4 Electroporation
    • 4.8.5 Magnetofection
    • 4.8.6 Sonoporation

5. GENE THERAPY CLINICAL TRIALS

  • 5.1 Gene Therapy Clinical Trials by Geography
  • 5.2 Diseases Targeted by Gene Therapy Clinical Trials
  • 5.3 Gene Types Transduced in Clinical Trials
  • 5.4 Vectors used in Gene Therapy Clinical Trials
  • 5.5 Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
  • 5.6 Domination of Oncology in Gene Therapy Development
  • 5.8 Anticipated Clinical Events in Gene Therapy Space, 2019/2020

6. CHIMERIC ANTIGEN RECEPTOR T CELL (CAR-T CELL) THERAPY

  • 6.1 Geographical Distribution of CAR-T Cell Therapy Companies
  • 6.2 Select Events in CAR-T Space in 2018
  • 6.3 Timeline of CAR-T Therapy Development
  • 6.4 CAR-T Manufacturing Processes
    • 6.4.1 Manufacturing of Autologous CAR-T Cells
      • 6.4.1.1 Source of T Cells
      • 6.4.1.2 T Cell Activation
      • 6.4.1.3 Genetic Modification of T Cells
      • 6.4.1.4 Expansion of CAR-T Cells
    • 6.4.2 Manufacturing of Allogeneic CAR-T Cells
    • 6.5 Structure of a CAR-T Cell
      • 6.5.1 First Generation CAR-T Cells
      • 6.5.2 Second Generation CAR-T Cells
      • 6.5.3 Third Generation CAR-T Cells
      • 6.5.4 Fourth Generation CAR-T Cells
  • 6.6 CAR-T Cells: Mechanism of Action
  • 6.7 Common Designs of CAR-T Constructs
  • 6.8 Transfection by Vectors
  • 6.9 CAR-T Cell Targets in Solid Malignancies
  • 6.10 Target Diseases for CAR-T Cell Therapy

7. CAR-T PATENT LANDSCAPE

  • 7.1 Number of CAR-T Patents, 2013-2018
  • 7.2 CAR-T Patent Types
  • 7.3 A Brief Snapshot of CAR-T Patent Landscape
    • 7.3.1 Patents for Anti-CD19 CAR-T
    • 7.3.2 Patents for Anti-BCMA CAR-T
    • 7.3.3 Patents for Regulatable CAR-T
  • 7.4 CAR-T Patents for Solid Tumors
  • 7.5 Major CAR-T Patent Applicants

8. THE CAR-T CLINICAL TRIALS LANDSCAPE

  • 8.1 The Surge in Number of CAR-T Clinical Trials
  • 8.2 Percentage of CAR-T Clinical Trials by Target Antigen
  • 8.3 Research Focus on CAR-T Trials by Indication
  • 8.4 CAR-T Projects with Commercial Licensees
  • 8.5 CAR-T Projects with Sole Involvement from Academia
  • 8.6 Anti-CD19 Projects
  • 8.7 CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
  • 8.8 CAR-T Projects in Solid Tumors
  • 8.9 CAR-T Projects using mRNA Electroporation for Gene Insertion
  • 8.10 CAR-T Projects using Suicide Genes
  • 8.11 Early-Stage CAR-T Assets
  • 8.12 CAR-T Clinical Trials in China
    • 8.12.1 CD19-Directed CAR-T Projects in China
    • 8.12.2 Chinese CAR-T Projects Targeting Non-CD19 Antigens
    • 8.12.3 Chinese CAR-T Projects in Solid Tumors
    • 8.12.4 Chinese Projects using Fourth Generation CAR-T Constructs
    • 8.12.5 Clinical-Stage CAR-T Projects in China
  • 8.13 Anticipated Developments in CAR-T Space, 2019-2020

9. MEDICAL FACILITIES OFFERING CAR-T THERAPIES

  • 9.1 U.S. Hospitals Offering CAR-T Therapies
  • 9.2 Facilities Offering CAR-T Therapies in Germany
  • 9.3 Hospitals Offering CAR-T Therapies in the U.K.

10. GENE THERAPY PRODUCTS

  • I0.1 Gendicine
  • 10.2 Oncorine
  • 10.3 Rexin-G
  • 10.4 Neovasculgen (vascular endothelial growth factor gene)
  • 10.5 Imlygic (talimogene laherparepvec)
  • 10.6 Strimvelis (autologous CD34+ enriched cells)
  • 10.7 Zalmoxis
  • 10.8 Invossa (tonogenchoncel-L)
  • 10.9 Kymriah (tisagenlecleucel-t)
    • 10.9.1 Overall Remission Rates in Patients Treated with Kymriah
    • 10.9.2 Overall Event-Free Survival Rate with Kymriah
  • 10.10 Yescarta (axicabtagene ciloleucel)
    • 10.10.1 Effectiveness of Yescarta
    • 10.10.2 Clinical Benefits of Yescarta
    • 10.10.3 Objective Response Rates (ORR) Reported for Yescarta
    • 10.10.4 Complete Remission Rate (CRR) for Yescarta
  • 10.11 Luxturna (voretigene neparvovec-rzyl)
  • 10.12 Patient Impact of the Three Products Approved in the U.S. in 2017
  • 10.13 Anticipated Number of Product Launches between 2018 and 2030
  • 10.14 Number of Gene Therapies Available in Eight Major Markets as of January 2019
  • 10.15 Five Forthcoming Gene Therapies

11. PRICING AND PAYMENT MODELS FOR GENE THERAPIES

  • 11.1 CMS and Payments for Kymriah and Yescarta
  • 11.2 Kymriah's Outcome-Based Contracts (OBCs)
  • 11.3 Luxturna's Installment Payment Plan
  • 11.4 Other Suggested Payment Options for Gene Therapies

12. MANUFACTURING OF CELLS AND VIRUS VECTORS

  • 12.1 Cost of Manufacture of Cells and Virus Vectors
  • 12.2 Cost of Manufacture in a Partially-Automated Facility
  • 12.3 Cost of Manufacture in a Fully-Automated Facility
  • 12.4 Net Present Cost (NPC) of the Three Methods
  • 12.5 In-House Gene Therapy Manufacturing Capabilities
  • 12.6 External Gene Therapy Manufacturing
  • 12.7 Manufacturing Capacity in North America
  • 12.8 Manufacturing Capacity in Europe

13. CMOs & CDMOs

  • 13.1 Appropriate Phase for Outsourcing
  • 13.2 Important Points Considered While Fixing a CDMO
  • 13.3 In-House vs. Outsourcing
  • 13.4 Number of CMPs and CDMOs in Europe
  • 13.5 Projected Growth of Outsourcing Market
  • 13.6 Projected Growth of Manufacturing Capacity

14. MARKET ANALYSIS

  • 14.1 Global Market for Gene Therapy by Product Category.
  • 14.2 Competitive Landscape in Gene-Modified Cell Therapy Products
  • 14.3 Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
    • 14.3.1 LentiGlobin to be on Top among the Top Ten
  • 14.4 Global Market for Viral Vector and Plasmid DNA Manufacturing
  • 14.5 Top Nine Assets of Gene Therapy between 2018 and 2024
    • 14.5.1 Zolgensma (AVXS-101)
    • 14.5.2 JCAR017, bb2121
    • 14.5.3 Lentiglobin (BB305)
    • 14.5.4 Valoctocogene roxaparvovec
    • 14.5.5 ABO-102
    • 14.5.6 Tabelecleucel
  • 14.6 Net Present Value (NPV) of Select Gene Therapy Product Candidates

15. COMPANY PROFILES

  • 15.1 4D Molecular Therapeutics, LLC
    • 15.1.1 Key Capabilities
  • 15.2 Abeona Therapeutics, Inc.
  • 15.3 Adaptimmune Therapeutics, plc
  • 15.4 Advanced Bioscience Laboratories, Inc.
    • 15.4.1 Biomanufacturing Services
    • 15.4.2 Manufacturing of Virus-Based Products
    • 15.4.3 Manufacturing of Protein-Based Products
    • 15.4.4 Asceptic Filling Services
    • 15.4.5 Immunology and Biomarker Testing
    • 15.4.6 Contract Research Assays
    • 15.4.7 Clinical Immunology Solutions
    • 15.4.8 Preclinical Solutions
    • 15.4.9 In vivo and ex vivo Preclinical Studies
    • 15.4.10 Product Development Solutions
    • 15.4.11 Government Partnering
    • 15.4.12 Translational Product Development
  • 15.5 Adverum Biotechnologies, Inc.
  • 15.6 Akouos, Inc.
  • 15.7 Aldevron, LLC
    • 15.7.1 Services
  • 15.8 Allogene Therapeutics, Inc.
  • 15.9 Ambys Medicines, Inc.
  • 15.10 American Gene Technologies International, Inc.
  • 15.11 Amgen, Inc.
    • 15.11.1 Imlygic (talimogene laherparepvec)
  • 15.12 Amicus Therapeutics, Inc.
  • 15.13 AskBio
  • 15.14 Audentes Therapeutics, Inc.
    • 15.14.1 AT132
    • 15.14.2 AT342
    • 15.14.3 AT982
    • 15.14.4 AT307
  • 15.15 AveXis, Inc.
    • 15.15.1 AVXS-101
  • 15.16 AVROBIO, INC.
    • 15.16.1 AVR-RD-01
    • 15.16.2 AVR-RD-02
    • 15.16.3 AVR-RD-03
    • 15.16.4 AVR-RD-04
  • 15.17 Benitec Biopharma Ltd.
  • 15.18 Bioverativ, Inc.
  • 15.19 bluebird bio, Inc.
    • 15.19.1 Partnering
    • 15.19.2 Current Partners
  • 15.20 BlueRock Therapeutics
    • 15.20.1 BlueRock's Pipeline
  • 15.21 Brammer Bio, LLC
    • 15.21.1 Services
    • 15.21.2 Product Development
    • 15.21.3 Gene Therapy Manufacturing
    • 15.21.4 Viral Vectors
  • 15.22 Carina Biotech
    • 15.22.1 CNA1003
  • 15.23 CARsgen Therapeutics, Ltd.
  • 15.24 Casebia Therapeutics
  • 15.25 Celyad SA
  • 15.26 Cevec Pharmaceuticals GmbH
    • 15.26.1 Services
  • 15.27 CombiGene AB
    • 15.27.1 Epilepsy Project
  • 15.28 CRISPR Therapeutics AG
  • 15.29 Delphi Genetics S.A.
    • 15.29.1 Services
  • 15.30 DNAlite Therapeutics, Inc.
  • 15.31 Editas Medicine, Inc.
  • 15.32 Elixirgen Therapeutics, LLC
  • 15.33 Fibrocell Technologies, Inc.
    • 15.33.1 Fibrocell's Pipeline
  • 15.34 FinVector Vision Therapies Oy
    • 15.34.1 Services
  • 15.35 Freeline Therapeutics, Ltd.
  • 15.36 Gemini Therapeutics, Inc.
  • 15.37 GEG Tech
    • 15.37.1 Lenti-ONE Vectors
  • 15.38 Genethon
  • 15.39 Generation Bio Co.
  • 15.40 Homology Medicines, Inc.
    • 15.40.1 Gene Editing Platform
  • 15.41 Horama SAS
  • 15.42 Intellia Therapeutics Inc.
  • 15.43 Kite Pharma, Inc.
    • 15.43.1 Yescarta
  • 15.44 Kolon Lifesciences, Inc.
    • 15.44.1 Invossa-K inj.
  • 15.45 Krystal Biotech, Inc.
  • 15.46 LakePharma
    • 15.46.1 Viral Vector Services
  • 15.47 Lonza Group, Ltd.
    • 15.47.1 Custom Development and Manufacturing
  • 15.48 Lysogene S.A.
    • 15.48.1 MPS IIIA
    • 15.48.2 GM1 Gangliosidosis
  • 15.49 MeiraGTx Holdings plc.
    • 15.49.1 Meira's Pipeline
      • 15.49.1.1 AAV-CNGB3
      • 15.49.1.2 AAV-RPE65
      • 15.49.1.3 AAV-RPGR
      • 15.49.1.4 AAV-AQP1
      • 15.49.1.5 AAV-GAD
      • 15.49.1.6 AAV-UPF1
  • 15.50 MolMed S.p.A
    • 15.50.1 MolMed's Clinical Trial Programs
    • 15.50.2 MolMed's GMP Solutions
  • 15.51 Mustang Bio, Inc.
    • 15.51.1 MB-101
    • 15.51.2 MB-102
    • 15.51.3 MB-103
    • 15.51.4 MB-104
    • 15.51.5 MB-105
    • 15.51.6 MB-106
    • 15.51.7 MB-107
  • 15.52 Myonexus Therapeutics, Inc.
  • 15.53 Nightstar Therapeutics plc
  • 15.54 Novartis AG
    • 15.54.1 Kymriah
  • 15.55 Orchard Therapeutics plc
    • 15.55.1 Strimvelis
    • 15.55.2 OTL-101
    • 15.55.3 OTL-102
    • 15.55.4 OTL-103
    • 15.55.5 OTL-200
    • 15.55.6 OTL-201
    • 15.55.7 OTL-202
    • 15.55.8 OTL-300
  • 15.56 OxfordBioMedica plc
    • 15.56.1 Oxford BioMedica's Partnering Activities
  • 15.57 Oxford Genetics, Ltd.
    • 15.57.1 Scalable AAV Custom Solutions
  • 15.58 Paragon Bioservices, Inc.
    • 15.58.1 Services
  • 15.59 Posedia Therapeutics, Inc.
  • 15.60 Precision BioSciences, Inc.
    • 15.60.1 ARCUS Genome Editing Platform
  • 15.61 Prevail Therapeutics, Inc.
    • 15.61.1 Prevail's Focus
  • 15.62 PTC Therapeutics, Inc.
  • 15.63 Quethera, Ltd.
    • 15.63.1 Technology
  • 15.64 REGENXBIO, INC.
    • 15.64.1 NAV Technology
    • 15.64.2 Therapeutic Programs
    • 15.64.3 Product Candidates
      • 15.64.3.1 RGX-314
      • 15.64.3.2 RGX-501
      • 15.64.3.3 RGX-111
      • 15.64.3.4 RGX-121
      • 15.64.3.5 RGX-181
    • 15.64.4 Partnerships and Licensing
  • 15.65 Rocket Pharmaceuticals, Inc.
  • 15.66 Sangamo Therapeutics, Inc.
  • 15.67 Sarepta Therapeutics, Inc.
  • 15.68 Sibiono GeneTech Co. Ltd.
    • 15.68.1 Gendicine
  • 15.69 Sirion Biotech, GmbH
    • 15.69.1 Viral Vectors
    • 15.69.2 Technologies
  • 15.70 Solid Biosciences, Inc.
    • 15.70.1 SGT-001
  • 15.71 Spark Therapeutics, Inc.
    • 15.71.1 Luxturna
    • 15.71.2 Spark Therapeutics' Pipeline
  • 15.72 Synpromics, Ltd.
    • 15.72.1 Technology
    • 15.72.2 PromPT
  • 15.73 Takara Bio, Inc.
    • 15.73.1 Services & Support
  • 15.74 The Cell and Gene Therapy Catapult
    • 15.74.1 Catapult's Industrialization Team
    • 15.74.2 Catapult's Manufacturing Center at Stevenage
    • 15.74.3 Catapult's Regulatory Team
  • 15.75 Translate Bio, Inc.
  • 15.76 Ultragenyx Pharmaceutical, Inc.
    • 15.76.1 DTX301
    • 15.76.2 DTX401
  • 15.77 uniQure N.V.
  • 15.78 Vybion, Inc.
    • 15.78.1 INT41
  • 15.79 Vigene Biosciences
  • 15.80 Virovek, Inc.
    • 15.80.1 Custom-Made AAVs
  • 15.81 Vivebiotech SL
    • 15.81.1 Lentisoma Technology
    • 15.81.2 Services
  • 15.82 Vivet Therapeutics
    • 15.82.1 Vivet's Approach
  • 15.83 Voyager Therapeutics, Inc.

INDEX OF FIGURES

  • FIGURE 2.1: Diagrammatic Representation of Gene Augmentation Therapy
  • FIGURE 2.2: Schematic Representation of Gene Inhibition Therapy
  • FIGURE 2.3: Diagrammatic Representation of Suicide Gene Therapy
  • FIGURE 2.4: Illustration Showing the Viral Vector Transferring Gene into the Patient's Cell.
  • FIGURE 2.5: Autologous and Allogeneic Gene Therapies
  • FIGURE 3.1: Number of Gene and Gene-Based Medicine Companies by Geography
  • FIGURE 3.2: Gene Therapy Pipeline Volumes, 1995-2018
  • FIGURE 3.3: Minor Role Played by Big Pharma in Gene Therapy
  • FIGURE 3.4: Number of Gene Therapies to Gain FDA Approval by 2022
  • FIGURE 3.5: Gene Therapy Companies in Neurodegenerative Disorders by Geography
  • FIGURE 3.6: Gene Therapy Industry and Global Financings, 2018
  • FIGURE 4.1: Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
  • FIGURE 4.2: Companies Developing Lentivirus Vectors
  • FIGURE 4.3: Types of Diseases in Clinical Trials using AAV
  • FIGURE 4.4: Companies Developing AAV Vectors
  • FIGURE 5.1: Number of Gene Therapy Clinical Trials, 2017-2018
  • FIGURE 5.2: Gene Therapy Clinical Trials by Geography, 2018
  • FIGURE 5.3: Diseases Targeted by Gene Therapy, 2018
  • FIGURE 5.4: Gene Types Transduced in Clinical Trials
  • FIGURE 5.6: Vectors used in Gene Therapy Clinical Trials
  • FIGURE 5.7: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type, 2018
  • FIGURE 5.8: Domination of Oncology in Gene Therapy Development, 2018
  • FIGURE 5.9: Dominance of Viral Vectors in Gene Therapy Drug Development
  • FIGURE 6.1: Geographical Distribution of CAR-T Cell Therapy Companies
  • FIGURE 6.3: The Process of CAR-T Cell Manufacturing
  • FIGURE 6.4: Building a CAR-T Cell and its Binding to Tumor Antigen
  • FIGURE 6.5: First Generation CAR-T Cells
  • FIGURE 6.6: Second Generation CAR-T Cells
  • FIGURE 6.7: Third Generation CAR-T Cells
  • FIGURE 6.8: Fourth Generation CAR-T Cells
  • FIGURE 6.9: Recognition and Killing of Cancer Cells by CAR-T Cells
  • FIGURE 6.10: Most Frequent CAR-T Targets in Solid Malignancies
  • FIGURE 7.1: Number of CAR-T Patents, 2013-2018
  • FIGURE 7.1: Major CAR-T Patent Applicants
  • FIGURE 8.1: The Surge in Number of CAR-T Clinical Trials, 2007-2018.
  • FIGURE 8.2: Percentage of CAR-T Clinical Trials by Target Antigen
  • FIGURE 8.3: Research Focus on CAR-T Trials by Indication
  • FIGURE 8.4: CAR-T Cells Targeting Antigens on Solid Tumors
  • FIGURE 10.1: Anticipated Number of Product Launches between 2018 and 2030
  • FIGURE 12.1: Expenditure Categories in Cell Manufacturing
  • FIGURE 12.2: The Impact of Head Count on Labor Cost
  • FIGURE 12.3: Cost of Manufacture in a Partially-Automated Facility
  • FIGURE 12.4: Cost of Manufacture in a Fully-Automated Facility
  • FIGURE 12.5: Net Present Cost (NPC) of the Three Methods
  • FIGURE 13.1: Appropriate Phase for Outsourcing
  • FIGURE 13.2: Important Points Considered While Fixing a CDMO
  • FIGURE 13.3: Share of In-House and Outsourcing
  • FIGURE 13.4: Number of CMPs and CDMOs in Europe
  • FIGURE 13.5: Projected Growth of Outsourcing Market, 2018-2025
  • FIGURE 14.1: Global Market for Gene Therapy Products, 2018-2025
  • FIGURE 14.2: Global Market for Gene Therapy by Product Category, 2018-2025
  • FIGURE 14.3: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025

INDEX OF TABLES

  • TABLE 3.1: Number of Gene Therapy Companies by Geography
  • TABLE 3.2: Gene Therapy Product Candidates Granted RMAT Designation
  • TABLE 3.3: Gene Therapy Companies in Neurodegenerative Disorders by Geography
  • TABLE 3.4: Gene Therapy Developers for Neurodegenerative Disorders
  • TABLE 3.5: Neurodegenerative Diseases Addressed by Gene Therapy Studies
  • TABLE 3.6: Select Anticipated Events in Neurodegenerative Space of Gene Therapy
  • TABLE 3.7: Select Events in Gene Therapy Space
  • TABLE 3.8: Important Gene Therapy Startups
  • TABLE 3.9: Gene Therapy Industry and Global Financings
  • TABLE 3.10: Corporate Partnerships in 2018
  • TABLE 3.11: Private Placements and Venture Financings
  • TABLE 3.12: Public Offerings (IPOs & Follow-Ons)
  • TABLE 4.2: Retroviral Vectors: A Snapshot
  • TABLE 4.3: Adenovirus Vectors: A Snapshot
  • TABLE 4.4: Herpes Simplex Viral Vectors: A Snapshot.
  • TABLE 4.5: Lentivirus Vectors: A Snapshot
  • TABLE 4.6: Adeno-Associated Virus Vectors: A Snapshot
  • TABLE 4.7: Genes Transduced by AAV for Specific Diseases
  • TABLE 4.8: The Predominantly Used Viral Vectors
  • TABLE 4.9: Microinjection Method of Gene Delivery
  • TABLE 4.10: Particle Bombardment Method of Gene Delivery
  • TABLE 4.11: Laser Irradiation Method of Gene Delivery
  • TABLE 4.12: Electroporation Method of Gene Delivery
  • TABLE 4.13: Magnetofection Method of Gene Delivery.
  • TABLE 4.14: Sonoporation Method of Gene Delivery
  • TABLE 5.1: Number of Gene Therapy Clinical Trials
  • TABLE 5.2: Gene Therapy Clinical Trials by Geography
  • TABLE 5.3: Diseases Targeted by Gene Therapy
  • TABLE 5.4: Gene Types Transduced in Clinical Trials
  • TABLE 5.6: Vectors used in Gene Therapy Clinical Trials
  • TABLE 5.7: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
  • TABLE 5.8: Domination of Oncology in Gene Therapy Development
  • TABLE 5.9: Select Anticipated Clinical Data and Events in 2019/2020
  • TABLE 5.9: (CONTINUED)
  • TABLE 5.9: (CONTINUED)
  • TABLE 5.9: (CONTINUED)
  • TABLE 5.9: (CONTINUED)
  • TABLE 5.9: (CONTINUED)
  • TABLE 5.9: (CONTINUED)
  • TABLE 6.1: Geographical Distribution of CAR-T Cell Therapy Companies
  • TABLE 6.2: Select Events in CAR-T Space
  • TABLE 6.3: Timeline of CAR-T Cell Therapy Development
  • TABLE 6.4: Different Types of CAR-T Constructs, Corresponding Antigens and Indications
  • TABLE 6.5: Most Frequent CAR-T Targets in Solid Malignancies
  • TABLE 6.6: Target Diseases and Target Antigens for CAR-T Cell Therapy
  • TABLE 7.1: Major CAR-T Patent Holders
  • TABLE 7.2: Select List of CAR-T Patents
  • TABLE 7.3: Major CAR-T Patent Applicants
  • TABLE 8.1: The Surge in Number of CAR-T Clinical Trials
  • TABLE 8.2: Percentage of CAR-T Clinical Trials by Target Antigen
  • TABLE 8.3: Research Focus on CAR-T Trials by Indication
  • TABLE 8.4: CAR-T Projects with Commercial Licensees
  • TABLE 8.4: (CONTINUED)
  • TABLE 8.5: CAR-T Projects with Sole Involvement from Academia
  • TABLE 8.6: Anti-CD19 Projects
  • TABLE 8.7: CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
  • TABLE 8.8: CAR-T Projects in Solid Tumors
  • TABLE 8.9: CAR-T Projects using mRNA Electroporation for Gene Insertion
  • TABLE 8.10: CAR-T Projects using Suicide Genes
  • TABLE 8.11: Early-Stage CAR-T Assets
  • TABLE 8.12: Distribution of CAR-T Clinical Trials in China
  • TABLE 8.13: CD19-Directed CAR-T Projects in China
  • TABLE 8.14: Chinese CAR-T Projects Targeting Non-CD19 Antigens
  • TABLE 8.15: Select Chinese CAR-T Projects in Solid Tumors
  • TABLE 8.16: Chinese Projects using Fourth Generation CAR-T Constructs
  • TABLE 8.17: Clinical-Stage CAR-T Projects in China
  • TABLE 8.18: Anticipated Developments in CAR-T Space
  • TABLE 8.18: (CONTINUED)
  • TABLE 9.1: U.S. Medical Facilities Offering CAR-T Therapy
  • TABLE 9.1: (CONTINUED)
  • TABLE 9.1: (CONTINUED)
  • TABLE 9.2: Facilities Offering CAR-T Therapies in the U.K.
  • TABLE 10.1: Approved Gene Therapy Products
  • TABLE 10.1: (CONTINUED)
  • TABLE 10.1: (CONTINUED)
  • TABLE 10.2: Overall Remission Rates in Patients Treated with Kymriah
  • TABLE 10.3: Event-Free Survival at Six and 12 Months in ALL Patients
  • TABLE 10.4: Yescarta's Timeline of Development
  • TABLE 10.5: Objective Response Rates (ORR) Reported for Yescarta
  • TABLE 10.6: CRR for Yescarta Compared with CRR of SCHOLAR-1 Trial
  • TABLE 10.7: The Development Timeline of Luxturna
  • TABLE 10.8: Patient Impact of the Three Products Approved in the U.S. in 2017
  • TABLE 10.9: Number of Gene Therapies in Eight Major Markets a
  • TABLE 10.10: Details of the Five Forthcoming Gene Therapies
  • TABLE 11.1: Cost and Market Size for Organ Transplants in the U.S.
  • TABLE 11.2: Cost and Market Size for Three Gene Therapies in the U.S.
  • TABLE 11.3: Suggested Financing and Payment Options for Gene Therapies
  • TABLE 12.1: Expenditure Categories in Cell Manufacturing
  • TABLE 12.2: The Impact of Head Count on Labor Cost
  • TABLE 12.3: Cost of Manufacture in a Partially-Automated Facility
  • TABLE 12.4: Cost of Manufacture in a Fully-Automated Facility
  • TABLE 12.5: Companies with In-House Gene Therapy Manufacturing Capability
  • TABLE 12.6: External Gene Therapy Manufacturing
  • TABLE 12.7: Manufacturing Capacity in North America
  • TABLE 12.8: Manufacturing Capacity in Europe
  • TABLE 12.8: (CONTINUED)
  • TABLE 12.8: (CONTINUED)
  • TABLE 13.1: Select CDMOs
  • TABLE 13.2: Appropriate Phase for Outsourcing
  • TABLE 13.3: Important Points Considered While Fixing a CDMO
  • TABLE 13.4: Share of In-House and Outsourcing
  • TABLE 14.1: Global Market for Gene Therapy Products, 2018-2025
  • TABLE 14.2: Global Market for Gene Therapy by Product Category, 2018-2025
  • TABLE 14.3: Competitive Landscape in Gene-Modified Cell Therapy Products
  • TABLE 14.4: Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
  • TABLE 14.4: (CONTINUED)
  • TABLE 14.5: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
  • TABLE 14.6: Top Nine Assets of Gene Therapies between 2018 and 2024
  • TABLE 14.7: Net Present Value (NPV) of Select Gene Therapy Product Candidates
  • TABLE 14.8: Net Present Value (NPV) of Yescarta and Kymriah
  • TABLE 15.1: 4D Molecular Therapeutics' Product Pipeline
  • TABLE 15.2: Abeona's Product Pipeline
  • TABLE 15.3: Adaptimmune's Pipeline Programs
  • TABLE 15.4: Allogene Therapeutics' Pipeline of Product Candidates
  • TABLE 15.5: Amicus' Gene Therapy Pipeline
  • TABLE 15.6: AskBio's Therapeutic Pipeline
  • TABLE 15.7: Audentes' Product Candidates
  • TABLE 15.8: AVROBIO's Gene Therapy Programs
  • TABLE 15.9: Benitec's Product Pipeline using Gene Silencing Technologies
  • TABLE 15.10: Bioverativ's Product Pipeline
  • TABLE 15.11: bluebird bio's Product Pipeline
  • TABLE 15.12: CARsgen Therapeutics' Product Candidates
  • TABLE 15.13: Casebia's Pipeline at a Glance
  • TABLE 15.14: Celyad's Program Status at a Glance
  • TABLE 15.15: CRISPR Therapeutics' Clinical Programs
  • TABLE 15.16: Editas' Diverse Product Pipeline
  • TABLE 15.17: Elixirgen's Pipeline for Genetic and Age-Related Diseases
  • TABLE 15.18: Fibrocell's Personalized Biologics Pipeline
  • TABLE 15.19: Freeline's Pipeline of Gene Therapy Treatments in Development
  • TABLE 15.20: Gemini's Pipeline
  • TABLE 15.21: Genethon's Products in Development
  • TABLE 15.22: Horama's Product Pipeline
  • TABLE 15.23: Intellia's in vivo and ex vivo RD Pipeline
  • TABLE 15.24: Kite's CAR-T Pipeline
  • TABLE 15.25: Kolon's Gene Therapeutics in Development
  • TABLE 15.26: Krystal Biotech's P[peline Programs for Orphan Skin Diseases
  • TABLE 15.27: Meira's Pipeline
  • TABLE 15.28: MolMed's Product Candidates
  • TABLE 15.29: Mustang Bio's Product Pipeline
  • TABLE 15.30: Myonexus' Clinical Stage LGMD Gene Therapy Pipeline
  • TABLE 15.31: Nightstar Therapeutics' Pipeline Programs
  • TABLE 15.32: Orchard Therapeutics' Pipeline
  • TABLE 15.33: Oxford BioMedica's Proprietary Product Pipeline
  • TABLE 15.34: Oxford BioMedica's Partnered Products
  • TABLE 15.35: Oxford BioMedica's IP-Enabled Royalty-Bearing Products
  • TABLE 15.36: Posedia's Product Pipeline
  • TABLE 15.37: PTC's Gene Therapy Programs for CNS Disorders
  • TABLE 15.38: REGENXBIO'S Product Pipeline
  • TABLE 15.39: REGENXBIO'S & Licensees' Pipeline
  • TABLE 15.40: Sangamo's Proprietary Programs
  • TABLE 15.41: Sangamo's Programs for Partners
  • TABLE 15.42: Sarepta's Huge Product Pipeline
  • TABLE 15.43: Spark Therapeutics' Pipeline
  • TABLE 15.44: Translate Bio's Lead Product Candidates
  • TABLE 15.45: uniQure's Product Pipeline
  • TABLE 15.46: Vybion's Drug Pipeline
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