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

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

EXECUTIVE SUMMARY


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.


  • REPORT OVERVIEW
    • Statement of the Report
    • Executive Summary
  • GENE THERAPY: AN INTRODUCTION
    • Gene Augmentation Therapy
    • Gene Inhibition Therapy
    • Suicide Gene Therapy
    • The Process of Gene Transfer in Gene Therapy
    • Ex vivo vs. in vivo Gene Therapies
      • Ex vivo Gene Therapy
      • In vivo Gene Therapy
    • Autologous vs. Allogeneic Gene Therapies
  • STATUS OF GENE THERAPY INDUSTRY
    • Number of Gene Therapy Companies in the World
      • Table Number of Gene Therapy Companies by Geography
    • Gene Therapy Pipeline Volumes, 1995-2018
    • Big Pharma's Involvement in Gene Therapy Development
    • Number of Gene Therapies Anticipated to be Approved in the Next Three Years
    • Gene Therapy Product Candidates Granted RMAT Designation
      • Table Gene Therapy Product Candidates Granted RMAT Designation, 2018
      • EB-101
      • ABO-102
      • AT132
      • LentiGlobin
      • JCAR017
      • NSR-RE P1
      • P-BCMA-101
      • VY-AADC
    • Gene Therapy Companies Active in Neurodegenerative Disorders
      • Table Gene Therapy Companies in Neurodegenerative Disorders by Geography
      • Gene Therapy Developers for Neurodegenerative Disorders
        • Table Gene Therapy Developers for Neurodegenerative Disorders, 2018
      • Neurodegenerative Diseases Addressed by Gene Therapy Studies
        • Table Neurodegenerative Diseases Addressed by Gene Therapy Studies
      • Anticipated Events in Neurodegenerative Space of Gene Therapy
        • Table Select Anticipated Events in Neurodegenerative Space of Gene Therapy, 2018
    • Important Events in Gene Therapy Space in 2018
      • Table Select Events in Gene Therapy Space in 2017/2018
    • Important Gene Therapy Startups
      • Table Important Gene Therapy Startups
    • Gene Therapy Industry and Global Financings, 2018
      • Table Gene Therapy Industry and Global Financings, 2018
    • Corporate Partnerships in 2018
      • Table Corporate Partnerships in 2018
    • Private Placements and Venture Financings in 2018
      • Table Private Placements and Venture Financings in 2018
    • Public Offerings (IPOs & Follow-Ons) in 2018
      • Table Public Offerings (IPOs & Follow-Ons) in 2018
  • GENE DELIVERY VEHICLES
    • Viral Vectors
      • Physical Properties of Commonly Used Viruses
    • Retrovirus Vectors
      • Table Retroviral Vectors: A Snapshot
    • Adenovirus Vectors
      • Table Adenovirus Vectors: A Snapshot
    • Herpes Simplex Viral Vectors
      • Table Herpes Simplex Viral Vectors: A Snapshot
    • Lentiviral Vectors
      • Table Lentivirus Vectors: A Snapshot
      • Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
      • Companies Developing Lentivirus Vectors
    • Adeno-Associated Virus Vectors
      • Table Adeno-Associated Virus Vectors: A Snapshot
      • Types of Diseases in Clinical Trials using AAV
      • Genes Transduced by AAV for Specific Diseases
        • Table Genes Transduced by AAV for Specific Diseases
      • Companies Developing AAV Vectors
    • The Predominantly Used Viral Vectors
      • Table The Predominantly Used Viral Vectors
    • Non-Viral Vectors
      • Microinjection
        • Table Microinjection Method of Gene Delivery
      • Particle Bombardment
        • Table Particle Bombardment Method of Gene Delivery
      • Laser Irradiation
        • Table Laser Irradiation Method of Gene Delivery
      • Electroporation
        • Table Electroporation Method of Gene Delivery
      • Magnetofection
        • Table Magnetofection Method of Gene Delivery
      • Sonoporation
        • Table Sonoporation Method of Gene Delivery
  • GENE THERAPY CLINICAL TRIALS
    • Table Number of Gene Therapy Clinical Trials, 2017-2018
    • Gene Therapy Clinical Trials by Geography
      • Table Gene Therapy Clinical Trials by Geography, 2018
    • Diseases Targeted by Gene Therapy Clinical Trials
      • Table Diseases Targeted by Gene Therapy, 2018
    • Gene Types Transduced in Clinical Trials
      • Table Gene Types Transduced in Clinical Trials
    • Vectors used in Gene Therapy Clinical Trials
      • Table Vectors used in Gene Therapy Clinical Trials
    • Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
      • Table Gene Therapy Clinical Trials for Neurodegenerative Indications by Type, 2018
    • Domination of Oncology in Gene Therapy Development
      • Table Domination of Oncology in Gene Therapy Development, 2018
    • Dominance of Viral Vectors in Gene Therapy Drug Development
    • Anticipated Clinical Events in Gene Therapy Space, 2019/2020
      • Table Select Anticipated Clinical Data and Events in 2019/2020
  • CHIMERIC ANTIGEN RECEPTOR T CELL (CAR-T CELL) THERAPY
    • Geographical Distribution of CAR-T Cell Therapy Companies
      • Table Geographical Distribution of CAR-T Cell Therapy Companies
    • Select Events in CAR-T Space in 2018
      • Table Select Events in CAR-T Space in 2018
    • Timeline of CAR-T Therapy Development
      • Table Timeline of CAR-T Cell Therapy Development
    • CAR-T Manufacturing Processes
      • Manufacturing of Autologous CAR-T Cells
      • Manufacturing of Allogeneic CAR-T Cells
    • Structure of a CAR-T Cell
      • First Generation CAR-T Cells
      • Second Generation CAR-T Cells
      • Third Generation CAR-T Cells
      • Fourth Generation CAR-T Cells
    • CAR-T Cells: Mechanism of Action
    • Common Designs of CAR-T Constructs
      • Table Different Types of CAR-T Constructs, Corresponding Antigens and Indications
    • Transfection by Vectors
    • CAR-T Cell Targets in Solid Malignancies
      • Table Most Frequent CAR-T Targets in Solid Malignancies
    • Target Diseases for CAR-T Cell Therapy
      • Table Target Diseases and Target Antigens for CAR-T Cell Therapy
  • CAR-T PATENT LANDSCAPE
    • Number of CAR-T Patents, 2013-2018
    • CAR-T Patent Types
      • Table Major CAR-T Patent Holders
    • A Brief Snapshot of CAR-T Patent Landscape
      • Patents for Anti-CD19 CAR-T
      • Patents for Anti-BCMA CAR-T
      • Patents for Regulatable CAR-T
    • CAR-T Patents for Solid Tumors
      • Table Select List of CAR-T Patents
    • Major CAR-T Patent Applicants
      • Table Major CAR-T Patent Applicants
  • THE CAR-T CLINICAL TRIALS LANDSCAPE
    • The Surge in Number of CAR-T Clinical Trials
      • Table The Surge in Number of CAR-T Clinical Trials, 2007-2018
    • Percentage of CAR-T Clinical Trials by Target Antigen
      • Table Percentage of CAR-T Clinical Trials by Target Antigen
    • Research Focus on CAR-T Trials by Indication
      • Table Research Focus on CAR-T Trials by Indication
    • CAR-T Projects with Commercial Licensees
      • Table CAR-T Projects with Commercial Licensees
    • CAR-T Projects with Sole Involvement from Academia
      • Table CAR-T Projects with Sole Involvement from Academia
    • Anti-CD19 Projects
      • Table Anti-CD19 Projects
    • CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
      • Table CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
    • CAR-T Projects in Solid Tumors
      • Table CAR-T Projects in Solid Tumors
    • CAR-T Projects using mRNA Electroporation for Gene Insertion
      • Table CAR-T Projects using mRNA Electroporation for Gene Insertion
    • CAR-T Projects using Suicide Genes
      • Table CAR-T Projects using Suicide Genes
    • Early-Stage CAR-T Assets
      • Table Early-Stage CAR-T Assets
    • CAR-T Clinical Trials in China
      • Table Distribution of CAR-T Clinical Trials in China
      • CD19-Directed CAR-T Projects in China
        • Table CD19-Directed CAR-T Projects in China
      • Chinese CAR-T Projects Targeting Non-CD19 Antigens
        • Table Chinese CAR-T Projects Targeting Non-CD19 Antigens
      • Chinese CAR-T Projects in Solid Tumors
        • Table Select Chinese CAR-T Projects in Solid Tumors
      • Chinese Projects using Fourth Generation CAR-T Constructs
        • Table Chinese Projects using Fourth Generation CAR-T Constructs
      • Clinical-Stage CAR-T Projects in China
        • Table Clinical-Stage CAR-T Projects in China
    • Anticipated Developments in CAR-T Space, 2019-2020
      • Table Anticipated Developments in CAR-T Space, 2019-2020
  • MEDICAL FACILITIES OFFERING CAR-T THERAPIES
    • U.S. Hospitals Offering CAR-T Therapies
      • Table U.S. Medical Facilities Offering CAR-T Therapy
    • Facilities Offering CAR-T Therapies in Germany
    • Hospitals Offering CAR-T Therapies in the U.K.
      • Table Facilities Offering CAR-T Therapies in the U.K.
  • GENE THERAPY PRODUCTS
    • Table Approved Gene Therapy Products
    • Gendicine
    • Oncorine
    • Rexin-G
    • Neovasculgen (vascular endothelial growth factor gene)
    • Imlygic (talimogene laherparepvec)
    • Strimvelis (autologous CD34+ enriched cells)
    • Zalmoxis
    • Invossa (tonogenchoncel-L)
    • Kymriah (tisagenlecleucel-t)
      • Overall Remission Rates in Patients Treated with Kymriah
        • Table Overall Remission Rates in Patients Treated with Kymriah
      • Overall Event-Free Survival Rate with Kymriah
        • Table Event-Free Survival at Six and 12 Months in ALL Patients
    • Yescarta (axicabtagene ciloleucel)
      • Table Yescarta's Timeline of Development
      • Effectiveness of Yescarta
      • Clinical Benefits of Yescarta
      • Objective Response Rates (ORR) Reported for Yescarta
        • Table Objective Response Rates (ORR) Reported for Yescarta
      • Complete Remission Rate (CRR) for Yescarta
        • Table CRR for Yescarta Compared with CRR of SCHOLAR-1 Trial
    • Luxturna (voretigene neparvovec-rzyl)
      • Table The Development Timeline of Luxturna
    • Patient Impact of the Three Products Approved in the U.S. in 2017
      • Table Patient Impact of the Three Products Approved in the U.S. in 2017
    • Anticipated Number of Product Launches between 2018 and 2030
    • Number of Gene Therapies Available in Eight Major Markets as of January 2019
      • Table Number of Gene Therapies in Eight Major Markets as of January 2019
    • Five Forthcoming Gene Therapies
      • Table Details of the Five Forthcoming Gene Therapies
  • PRICING AND PAYMENT MODELS FOR GENE THERAPIES
    • Table Cost and Market Size for Organ Transplants in the U.S.
    • Table Cost and Market Size for Three Gene Therapies in the U.S.
    • CMS and Payments for Kymriah and Yescarta
    • Kymriah's Outcome-Based Contracts (OBCs)
    • Luxturna's Installment Payment Plan
    • Other Suggested Payment Options for Gene Therapies
      • Table Suggested Financing and Payment Options for Gene Therapies
  • MANUFACTURING OF CELLS AND VIRUS VECTORS
    • Table Expenditure Categories in Cell Manufacturing
    • Cost of Manufacture of Cells and Virus Vectors
      • Table The Impact of Head Count on Labor Cost
    • Cost of Manufacture in a Partially-Automated Facility
      • Table Cost of Manufacture in a Partially-Automated Facility
    • Cost of Manufacture in a Fully-Automated Facility
      • Table Cost of Manufacture in a Fully-Automated Facility
    • Net Present Cost (NPC) of the Three Methods
    • In-House Gene Therapy Manufacturing Capabilities
      • Table Companies with In-House Gene Therapy Manufacturing Capability
    • External Gene Therapy Manufacturing
      • Table External Gene Therapy Manufacturing
    • Manufacturing Capacity in North America
      • Table Manufacturing Capacity in North America
    • Manufacturing Capacity in Europe
      • Table Manufacturing Capacity in Europe
  • CMOs & CDMOs
    • Table Select CDMOs
    • Appropriate Phase for Outsourcing
      • Table Appropriate Phase for Outsourcing
    • Important Points Considered While Fixing a CDMO
      • Table Important Points Considered While Fixing a CDMO
    • In-House vs. Outsourcing
      • Table Share of In-House and Outsourcing
    • Number of CMPs and CDMOs in Europe
    • Projected Growth of Outsourcing Market
    • Projected Growth of Manufacturing Capacity
  • MARKET ANALYSIS
    • Table Global Market for Gene Therapy Products, 2018-2025
    • Global Market for Gene Therapy by Product Category
      • Table Global Market for Gene Therapy by Product Category, 2018-2025
    • Competitive Landscape in Gene-Modified Cell Therapy Products
      • Table Competitive Landscape in Gene-Modified Cell Therapy Products
    • Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
      • LentiGlobin to be on Top among the Top Ten
        • Table Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
    • Global Market for Viral Vector and Plasmid DNA Manufacturing
      • Table Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
    • Top Nine Assets of Gene Therapy between 2018 and 2024
      • Table Top Nine Assets of Gene Therapies between 2018 and 2024
      • Zolgensma (AVXS-101)
      • JCAR017, bb2121
      • Lentiglobin (BB305)
      • Valoctocogene roxaparvovec
      • ABO-102
      • Tabelecleucel
    • Net Present Value (NPV) of Select Gene Therapy Product Candidates
      • Table Net Present Value (NPV) of Select Gene Therapy Product Candidates
      • Table Net Present Value (NPV) of Yescarta and Kymriah
  • COMPANY PROFILES
    • 4D Molecular Therapeutics, LLC
      • Key Capabilities
        • Table 4D Molecular Therapeutics' Product Pipeline
    • Abeona Therapeutics, Inc.
      • Table Abeona's Product Pipeline
    • Adaptimmune Therapeutics, plc
      • Table Adaptimmune's Pipeline Programs
    • Advanced Bioscience Laboratories, Inc.
      • Biomanufacturing Services
      • Manufacturing of Virus-Based Products
      • Manufacturing of Protein-Based Products
      • Asceptic Filling Services
      • Immunology and Biomarker Testing
      • Contract Research Assays
      • Clinical Immunology Solutions
      • Preclinical Solutions
      • In vivo and ex vivo Preclinical Studies
      • Product Development Solutions
      • Government Partnering
      • Translational Product Development
    • Adverum Biotechnologies, Inc.
    • Akouos, Inc.
    • Aldevron, LLC
      • Services
    • Allogene Therapeutics, Inc.
      • Table Allogene Therapeutics' Pipeline of Product Candidates
    • Ambys Medicines, Inc.
    • American Gene Technologies International, Inc.
    • Amgen, Inc.
      • Imlygic (talimogene laherparepvec)
    • Amicus Therapeutics, Inc.
      • Table Amicus' Gene Therapy Pipeline
    • AskBio
      • Table AskBio's Therapeutic Pipeline
    • Audentes Therapeutics, Inc.
      • AT132
      • AT342
      • AT982
      • AT307
        • Table Audentes' Product Candidates
    • AveXis, Inc.
      • AVXS-101
    • AVROBIO, INC.
      • AVR-RD-01
      • AVR-RD-02
      • AVR-RD-03
      • AVR-RD-04
        • Table AVROBIO's Gene Therapy Programs
    • Benitec Biopharma Ltd.
      • Table Benitec's Product Pipeline using Gene Silencing Technologies
    • Bioverativ, Inc.
      • Table Bioverativ's Product Pipeline
    • bluebird bio, Inc.
      • Partnering
      • Current Partners
        • Table bluebird bio's Product Pipeline
    • BlueRock Therapeutics
      • BlueRock's Pipeline
    • Brammer Bio, LLC
      • Services
      • Product Development
      • Gene Therapy Manufacturing
      • Viral Vectors
    • Carina Biotech
      • CNA1003
    • CARsgen Therapeutics, Ltd.
      • Table CARsgen Therapeutics' Product Candidates
    • Casebia Therapeutics
      • Table Casebia's Pipeline at a Glance
    • Celyad SA
      • Table Celyad's Program Status at a Glance
    • Cevec Pharmaceuticals GmbH
      • Services
    • CombiGene AB
      • Epilepsy Project
    • CRISPR Therapeutics AG
      • Table CRISPR Therapeutics' Clinical Programs
    • Delphi Genetics S.A.
      • Services
    • DNAlite Therapeutics, Inc.
    • Editas Medicine, Inc.
      • Table Editas' Diverse Product Pipeline
    • Elixirgen Therapeutics, LLC
      • Table Elixirgen's Pipeline for Genetic and Age-Related Diseases
    • Fibrocell Technologies, Inc.
      • Fibrocell's Pipeline
        • Table Fibrocell's Personalized Biologics Pipeline
    • FinVector Vision Therapies Oy
      • Services
    • Freeline Therapeutics, Ltd.
      • Table Freeline's Pipeline of Gene Therapy Treatments in Development
    • Gemini Therapeutics, Inc.
      • Table Gemini's Pipeline
    • GEG Tech
      • Lenti-ONE Vectors
    • Genethon
      • Table Genethon's Products in Development
    • Generation Bio Co.
    • Homology Medicines, Inc.
      • Gene Editing Platform
    • Horama SAS
      • Table Horama's Product Pipeline
    • Intellia Therapeutics Inc.
      • Table Intellia's in vivo and ex vivo RD Pipeline
    • Kite Pharma, Inc.
      • Yescarta
        • Table Kite's CAR-T Pipeline
    • Kolon Lifesciences, Inc.
      • Invossa-K inj.
        • Table Kolon's Gene Therapeutics in Development
    • Krystal Biotech, Inc.
      • Table Krystal Biotech's P[peline Programs for Orphan Skin Diseases
    • LakePharma
      • Viral Vector Services
    • Lonza Group, Ltd.
      • Custom Development and Manufacturing
    • Lysogene S.A.
      • MPS IIIA
      • GM1 Gangliosidosis
    • MeiraGTx Holdings plc
      • Meira's Pipeline
        • Table Meira's Pipeline
    • MolMed S.p.A
      • MolMed's Clinical Trial Programs
      • MolMed's GMP Solutions
        • Table MolMed's Product Candidates
    • Mustang Bio, Inc.
      • Table Mustang Bio's Product Pipeline
      • MB-101
      • MB-102
      • MB-103
      • MB-104
      • MB-105
      • MB-106
      • MB-107
    • Myonexus Therapeutics, Inc.
      • Table Myonexus' Clinical Stage LGMD Gene Therapy Pipeline
    • Nightstar Therapeutics plc
      • Table Nightstar Therapeutics' Pipeline Programs
    • Novartis AG
      • Kymriah
    • Orchard Therapeutics plc
      • Strimvelis
      • OTL-101
      • OTL-102
      • OTL-103
      • OTL-200
      • OTL-201
      • OTL-202
      • OTL-300
        • Table Orchard Therapeutics' Pipeline
    • OxfordBioMedica plc
      • Oxford BioMedica's Partnering Activities
        • Table Oxford BioMedica's Proprietary Product Pipeline
        • Table Oxford BioMedica's Partnered Products
        • Table Oxford BioMedica's IP-Enabled Royalty-Bearing Products
    • Oxford Genetics, Ltd.
      • Scalable AAV Custom Solutions
    • Paragon Bioservices, Inc.
      • Services
    • Posedia Therapeutics, Inc.
      • Table Posedia's Product Pipeline
    • Precision BioSciences, Inc.
      • ARCUS Genome Editing Platform
    • Prevail Therapeutics, Inc.
      • Prevail's Focus
    • PTC Therapeutics, Inc.
      • Table PTC's Gene Therapy Programs for CNS Disorders
    • Quethera, Ltd.
      • Technology
    • REGENXBIO, INC.
      • NAV Technology
      • Therapeutic Programs
      • Product Candidates
        • Table REGENXBIO'S Product Pipeline
      • Partnerships and Licensing
        • Table REGENXBIO'S & Licensees' Pipeline
    • Rocket Pharmaceuticals, Inc.
    • Sangamo Therapeutics, Inc.
      • Table Sangamo's Proprietary Programs
      • Table Sangamo's Programs for Partners
    • Sarepta Therapeutics, Inc.
      • Table Sarepta's Huge Product Pipeline
    • Sibiono GeneTech Co. Ltd.
      • Gendicine
    • Sirion Biotech, GmbH
      • Viral Vectors
      • Technologies
    • Solid Biosciences, Inc.
      • SGT-001
    • Spark Therapeutics, Inc.
      • Luxturna
      • Spark Therapeutics' Pipeline
        • Table Spark Therapeutics' Pipeline
    • Synpromics, Ltd.
      • Technology
      • PromPT
    • Takara Bio, Inc.
      • Services & Support
    • The Cell and Gene Therapy Catapult
      • Catapult's Industrialization Team
      • Catapult's Manufacturing Center at Stevenage
      • Catapult's Regulatory Team
    • Translate Bio, znc.
      • Table Translate Bio's Lead Product Candidates
    • Ultragenyx Pharmaceutical, Inc.
      • DTX301
      • DTX401
    • uniQure N.V.
      • Table uniQure's Product Pipeline
    • Vybion, Inc.
      • INT41
        • Table Vybion's Drug Pipeline
    • Vigene Biosciences
    • Virovek, Inc.
      • Custom-Made AAVs
    • Vivebiotech SL
      • Lentisoma Technology
      • Services
    • Vivet Therapeutics
      • Vivet's Approach
    • Voyager Therapeutics, Inc.
      • Table Voyager's Pipeline Programs

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