Global Gene Therapy Markets, 2013-2018 & 2019 & 2025

DUBLIN, July 29, 2019 /PRNewswire/ -- The "Global Market for Gene Therapy 2019 - Product Pipeline, Market Size, & Forecasts" report has been added to ResearchAndMarkets.com's offering.

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.

Key Topics Covered:

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
2.6 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
3.6 Gene Therapy Companies Active in Neurodegenerative Disorders
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
4.2 Retrovirus Vectors
4.3 Adenovirus Vectors
4.4 Herpes Simplex Viral Vectors
4.5 Lentiviral Vectors
4.6 Adeno-Associated Virus Vectors
4.7 The Predominantly Used Viral Vectors
4.8 Non-Viral Vectors

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
6.5 Structure of a CAR-T Cell
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
7.4 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
8.13 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
10.1 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)
10.10 Yescarta (axicabtagene ciloleucel)
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
14.4 Global Market for Viral Vector and Plasmid DNA Manufacturing
14.5 Top Nine Assets of Gene Therapy between 2018 and 2024
14.6 Net Present Value (NPV) of Select Gene Therapy Product Candidates

15. COMPANY PROFILES

For more information about this report visit https://www.researchandmarkets.com/r/h07luu

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