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, haemophilia, immunodeficiencies, inherited anaemia 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:
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.
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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 Content
Statement of the Report
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
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
Physical Properties of Commonly Used Viruses
Table Retroviral Vectors: A Snapshot
Table Adenovirus Vectors: A Snapshot
Herpes Simplex Viral Vectors
Table Herpes Simplex Viral Vectors: A Snapshot
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
Table Microinjection Method of Gene Delivery
Table Particle Bombardment Method of Gene Delivery
Table Laser Irradiation Method of Gene Delivery
Table Electroporation Method of Gene Delivery
Table Magnetofection Method of Gene Delivery
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
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
Neovasculgen (vascular endothelial growth factor gene)
Imlygic (talimogene laherparepvec)
Strimvelis (autologous CD34+ enriched cells)
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
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
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
4D Molecular Therapeutics, LLC
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.
Manufacturing of Virus-Based Products
Manufacturing of Protein-Based Products
Asceptic Filling Services
Immunology and Biomarker Testing
Contract Research Assays
Clinical Immunology Solutions
In vivo and ex vivo Preclinical Studies
Product Development Solutions
Translational Product Development
Adverum Biotechnologies, Inc.
Allogene Therapeutics, Inc.
Table Allogene Therapeutics' Pipeline of Product Candidates
Ambys Medicines, Inc.
American Gene Technologies International, Inc.
Imlygic (talimogene laherparepvec)
Amicus Therapeutics, Inc.
Table Amicus' Gene Therapy Pipeline
Table AskBio's Therapeutic Pipeline
Audentes Therapeutics, Inc.
Table Audentes' Product Candidates
Table AVROBIO's Gene Therapy Programs
Benitec Biopharma Ltd.
Table Benitec's Product Pipeline using Gene Silencing Technologies
Table Bioverativ's Product Pipeline
bluebird bio, Inc.
Table bluebird bio's Product Pipeline
Brammer Bio, LLC
Gene Therapy Manufacturing
CARsgen Therapeutics, Ltd.
Table CARsgen Therapeutics' Product Candidates
Table Casebia's Pipeline at a Glance
Table Celyad's Program Status at a Glance
Cevec Pharmaceuticals GmbH
CRISPR Therapeutics AG
Table CRISPR Therapeutics' Clinical Programs
Delphi Genetics S.A.
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.
Table Fibrocell's Personalized Biologics Pipeline
FinVector Vision Therapies Oy
Freeline Therapeutics, Ltd.
Table Freeline's Pipeline of Gene Therapy Treatments in Development
Gemini Therapeutics, Inc.
Table Gemini's Pipeline
Table Genethon's Products in Development
Generation Bio Co.
Homology Medicines, Inc.
Gene Editing Platform
Table Horama's Product Pipeline
Intellia Therapeutics Inc.
Table Intellia's in vivo and ex vivo RD Pipeline
Kite Pharma, Inc.
Table Kite's CAR-T Pipeline
Kolon Lifesciences, Inc.
Table Kolon's Gene Therapeutics in Development
Krystal Biotech, Inc.
Table Krystal Biotech's P[peline Programs for Orphan Skin Diseases
Viral Vector Services
Lonza Group, Ltd.
Custom Development and Manufacturing
MeiraGTx Holdings plc
Table Meira's Pipeline
MolMed's Clinical Trial Programs
MolMed's GMP Solutions
Table MolMed's Product Candidates
Mustang Bio, Inc.
Table Mustang Bio's Product Pipeline
Myonexus Therapeutics, Inc.
Table Myonexus' Clinical Stage LGMD Gene Therapy Pipeline
Nightstar Therapeutics plc
Table Nightstar Therapeutics' Pipeline Programs
Orchard Therapeutics plc
Table Orchard Therapeutics' Pipeline
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.
Posedia Therapeutics, Inc.
Table Posedia's Product Pipeline
Precision BioSciences, Inc.
ARCUS Genome Editing Platform
Prevail Therapeutics, Inc.
PTC Therapeutics, Inc.
Table PTC's Gene Therapy Programs for CNS Disorders
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.
Sirion Biotech, GmbH
Solid Biosciences, Inc.
Spark Therapeutics, Inc.
Spark Therapeutics' Pipeline
Table Spark Therapeutics' Pipeline
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.
Table uniQure's Product Pipeline
Table Vybion's Drug Pipeline
Voyager Therapeutics, Inc.
Table Voyager's Pipeline Programs
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