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Global Gene Therapy Market Analysis & Forecast to 2022: $350+ Million Market Strengthened by Recent Approvals of Kymriah, Yescarta and Luxturna in the US

Dublin, March 07, Mar 07, 2018 (GLOBE NEWSWIRE via COMTEX) --

The "Global Gene Therapy Market Analysis & Forecast to 2022" report has been added to's offering.

The global gene therapy market is forecast to hit $363 million by 2022

Strengthened by recent approvals of Kymriah, Yescarta and Luxturna in the US, and a committed European, Japanese and Chinese environment, gene therapy is set to become a significant player in the bio-pharmaceutical industry. The space covers many therapeutic areas specifically, oncology, rare diseases, Parkinson's, HIV, severe combined immuno-deficiencies (SCID) and hemophilia.

Gene therapy is driven by over 2,200 clinical trials globally, with over 55% of this occurring in the US, followed by Europe, Canada and China. Recently, in November 2017, the FDA indicated that gene therapies will now qualify for a fast approval process, which will bring more therapies to market faster. However, the space also has significant challenges, such as manufacturing logistics, reimbursement and its high cost.

Since the FDA approved Kymriah (tisagenlecleucel), Yescarta (Axicabtagene ciloleucel) and Luxturna (voretigene neparvovec-rzyl) in 2017, the US gene therapy space has expanded significantly, underlined by the fact that over 55% of completed and ongoing trials are located in this geographic. Growth in the gene therapy industry has resulted in new commercial initiatives and the emergence of new startups and spin-off biotechs.

Furthermore, gene therapy specifically has raised well over $600 million of venture capital in the last five years. Early stage companies have raised seed, Series A and Series B investment steadily since the market took off, including Spark Therapeutics, Avalanche Biotech, uniQure, Voyager Therapeutics, Editas Medicine and GenSight.

Key Topics Covered:

1.0 Introduction

2.0 Gene Therapy: The Basics

2.1 Gene Therapy Techniques

2.2 Gene Therapy Routes

2.3 CRISPR: Latest Technology in Gene Therapy

2.4 Challenges Associated with Developing Gene Therapies

2.5 Disadvantages of Gene Therapy

2.6 Concept to Product Candidates and Products

3.0 Historic Overview of Gene Therapy

3.1 The Formative Years of Gene Therapy

3.2 The Productive Years of Gene Therapy

4.0 Gene Therapy Phase III Product Candidates

4.1 CardioNovo (Generx)

4.2 Collategene

4.3 LentiGlobin

4.4 Lenti-D

4.5 VM-202

4.6 Invossa

4.7 GS010

4.8 ADA-Lentiviral Gene Therapy

5.0 Gene Therapy Phase II Product Candidates

5.1 hF-IX gene/Hemophilia B

5.2 NaGlu gene/San Fillipo B Syndrome

5.3 StarGen/Stargardt disease

5.4 UshStat/Usher Syndrome Type 1B

5.5 Retinostat/Wet AMD

5.6 Lenti-D/Childhood Cerebral ALD

5.7 LentiGlobin/Beta Thalassemia

5.8 SB-728/HIV/AIDS

5.9 CERE-110/Alzheimer's disease

5.10 AATD/Alpha-1 Antitrypsin deficiency

5.11 RS1 gene/X-linked juvenile Retinoschisis (XLRS)

5.12 SPK-CHM/Choroideremia

5.13 SPK-FIX/Hemophilia B

5.14 AMG0001/Primary Lymphedema

5.15 AMG0001/Ischemic heart disease

5.16 WASp /Wiskott-Aldrich syndrome

5.17 X-linked CGD

5.18 GSK2696273/ADA gene

5.19 VM106/Chronic Granulomatous Disease

5.20 VY-AADC01/Parkinson's disease

6.0 Commercialization, Cost and Warranty of Gene Therapies

6.1 Reasons for the Extortionate Price

6.2 Strategies to Make Gene Therapy Affordable

6.3 The Age of First-Ever Warranty for Gene Therapy

7.0 The Strength of Gene Therapy Industry

7.1 Development of Appropriate Gene Delivery Vehicles

7.2 CRISPR-Based Therapy

7.3 Early Clinical Gene Therapy Protocols

7.4 Worldwide Scenario of Gene Therapy Clinical Trials

7.5 Big Pharma's Bold Venturing into Gene Therapy

7.6 Venture Investments by Body Systems

8.0 Vectors for Gene Delivery

8.1 Non-Viral Vectors

8.2 Viral Vectors for Gene Therapy

9.0 Clinical Applications of Gene Therapy

9.1 Leber Congenital Amaurosis (LCA)

9.2 Stargardt Macular Degeneration (SMD)

9.3 Choroideremia

9.4 Leber Hereditary Optic Neuropathy (LHON)

9.5 Parkinson Disease

9.6 Spinal Muscle Atrophy

9.7 Alzheimer Disease

9.8 Cystic Fibrosis

9.10 X-Linked Adrenoleukodystrophy

9.11 Pompe Disease

9.12 Batten Disease (CLN3 Disease)

9.13 Metachromatic Leukodystrophy

9.14 Sanfilippo Syndrome

9.15 Hunter Syndrome

9.16 Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

9.17 X-Linked SCID

9.18 Chronic Granulomatous Disease (CGD)

9.19 Wischott Aldrich syndrome (WAS)

9.20 Hemophilia

9.21 Sickle Cell Anemia

9.22 Beta Thalassemia

9.25 Limb Girdle Muscle Dystrophy 2C/2D

9.26 Duchenne and Becker Muscular Dystrophy

9.27 Human Immunodeficiency Virus (HIV)

9.28 Epidermolysis Bullosa

9.29 Leukemia

9.30 Ovarian Cancer

9.31 Pancreatic Cancer

9.32 Head and Neck Cancers

9.33 Melanoma

9.34 Prostate Cancer

9.35 Breast Cancer

9.36 Fabry Disease

9.37 Hypercholesterolemia

9.38 Huntington Disease

9.39 Tay-Sachs Disease

10.0 Market Analysis

10.1 Global Market for Gene Therapy by Market Segment

10.2 Global Gene Therapy Market by Geography

10.3 Commercialization of Gene Therapy in China

10.4 Commercialization of Gene Therapy in the Philippines

10.5 Commercialization of Gene Therapy in Russia

10.6 Commercialization of Gene Therapy in Europe

10.7 Gene Therapy in the U.S.

10.8 Commercialization of Gene Therapy in South Korea

10.9 Additional Addresseable Markets for Gene Therapy

10.10 Challenges for Existing and Prospective Players

10.11 Future Outlook for Gene Therapy

10.12 Potential Market for Gene Therapy Product Candidates

11.0 Manufacturing of Viral Vectors and Logistics

11.1 Major Manufacturing Companies of Viral Vectors

11.2 Major Diseases Targeted by AAV Vectors in Clinical Trials

11.3 Major Companies Developing Lentiviral Vectors

11.4 Manufacturing Process

11.5 Contract Manufacturing

11.6 Targeted Delivery of Therapeutic Genes

11.7 Logistics Strategies for Gene Therapies

11.7 Cost of Clinical Trials for Biopharmaceuticals including Gene Therapy

12.0 Company Profiles

12.1 4d Molecular Therapeutics LLC

12.2 Abeona Therapeutics LLC

12.3 Advanced Cell & Gene Therapy LLC

12.4 Advantagene Inc.

12.5 Adverum Biotechnologies Inc.

12.6 Addgene Inc.

12.7 Agilis Biotherapeutics LLC

12.8 Angionetics Inc.

12.9 Applied Genetic Technologies Corporation (AGTC)

12.10 AnGes MG Inc.

12.11 Asklepios BioPharmaceutical Inc.

12.12 Audentes Therapeutics Inc.

12.13 AveXis Inc.

12.14 AvroBio Inc.

12.15 Benitec Biopharma

12.16 BioCancell Therapeutic Inc.

12.17 BioMarin Pharmaceutical Inc.

12.18 Bluebird bio Inc.

12.19 Brammer Bio LLC

12.20 Cellectis S.A.

12.21 Clontech Laboratories Inc.

12.22 Cobra Biologics Ltd.

12.23 Copernicus Therapeutics Inc.

12.24 Dimension Therapeutics Inc.

12.26 Editas Medicine Inc.

12.27 Fibrocell Sciences Inc.

12.28 Florida Biologix

12.29 Freeline Therapeutics Ltd.

12.30 Genable Technologies Ltd.

12.31 Genethon

12.32 Genlantis

12.33 GenSight Biologics S.A.

12.35 Ichor Medical Systems Inc.

12.36 Immune Design Corp.

12.37 Immusoft Corp.

12.38 Inovio Pharmaceuticals Inc.

12.39 Intellia Therapeutics Inc.

12.40 Juventa Therapeutics Inc.

12.41 Kite Pharma Inc.

12.42 Kolon Life Sciences Inc.

12.43 Lentigen Technology Inc.

12.44 Lysogene S.A.S.

12.45 Medgenics Inc.

12.46 Mirus Bio LLC

12.47 Mologen AG

12.48 NanoCor Therapeutics Inc.

12.49 Nature Technology Corp. (NTC)

12.50 NightstaRx Ltd.

12.51 Novasep Process SAS

12.52 Omnia Biologics Inc.

12.53 ORCA Therapeutics B.V.

12.54 OrphageniX

12.55 Oxford BioMedica plc

12.56 Oxford Genetics Ltd.

12.57 REGENXBIO Inc.

12.58 Renova Therapeutics Inc.

12.59 RetroSense Therapeutics LLC

12.60 Sangamo Biosciences Inc.

12.61 Sarepta Therapeutics Inc.

12.62 Shanghai Sunway Biotech Co. Ltd.

12.63 SiBiono GeneTech Co. Ltd.

12.64 Sirion Biotech GmbH

12.65 Spark Therapeutics Inc.

12.66 Takara Bio Inc.

12.67 Taxus Cardium Pharmaceutical Group Inc.

12.68 Tocagen Inc.

12.69 ToolGen Inc.

12.70 Transgene SA

12.71 uniQure N.V.

12.72 Vascular Biologics Ltd.

12.73 Vical Inc.

12.74 ViroMed Co., Ltd.

12.75 Vivebiotech SL

12.76 Voyager Therapeutics Inc.

12.77 Xenon Pharmaceuticals Inc.

For more information about this report visit

         Laura Wood, Senior Manager
         For E.S.T Office Hours Call 1-917-300-0470
         For U.S./CAN Toll Free Call 1-800-526-8630
         For GMT Office Hours Call +353-1-416-8900
         Related Topics: Genomics

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