Global Gene Therapy Market 2018-2022: $363 Mn Driven by 2,200+ Clinical Trials with Over 55% of this Occurring in the US, Followed by Europe, Canada and China

DUBLIN, March 7, 2018 /PRNewswire/ --

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

Gene therapy products approved between the years 2003 and 2017 include Gendicine, Oncorine, Rexin-G, Neovasculgen, Glybera, Imlygic, Strimvelis, Zalmoxis, Kymriah, Yescarta and Luxturna. Gendicine was approved for head and neck squamous cell carcinoma and has been in the Chinese market since 2003. Rexin-G was approved in the Philippines back in 2007 for the treatment of primary and metastatic cancer. Oncorine was approved in China in 2005 for nasopharyngeal carcinoma. The Russian market has Neovasculgen from 2011 for the treatment of peripheral arterial disease (PAD) and critical limb ischemia.

The first gene therapy approved in E.U. was Glybera in 2012 for the treatment of familial lipoprotein lipase deficiency (LPL), however in October 2017 it was pulled from the market due to lack of patient demand. In 2015, Imlygic was approved in E.U. and also in the U.S. to treat melanoma, and Phase II results released in 2017 indicated its efficacy in combination with the checkpoint-inhibitor, Yervoy. In the E.U., Strimvelis was approved in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID). In 2016, Zalmoxis was approved in E.U. for the treatment of leukemia. 2017 was a bumper year for gene therapy with Kymriah, Yescarta and Luxturna all gaining FDA approval.

Renewed interest has encouraged start-up companies to affiliate with academic centers for tech know-how. As clinical trials advance towards licensure, more meticulous product characterization using improved analytical methods and progressively higher regulatory compliance will be required. Some of the ongoing clinical trials are closing on to produce promising results, including one for hemophilia B caused by the deficiency of Factor IX using a recombinant adeno-associated virus (AAV) as a vector. The product candidate if succeeds will be a relatively cheaper alternative to the expensive and lifelong factor replacement therapy.

A second example of a successful outcome in gene therapy are studies conducted by independent laboratories focusing on sub-retinal delivery of recombinant AAV expressing retinal pigment epithelial RPE65 for Leber Congenital Amaurosis Type 2. A third example is the clinical trial involving nine children with X-linked severe combined immunodeficiency (SCID-X1) treated with autologous bone marrow CD34+ cells transduced with a self-inactivating (SIN) -retroviral vector expressing the IL-2 receptor -chain. The most significant achievement in gene therapy is the spectacular clinical results obtained by many independent teams using CAR-T-cell technology. This novel strategy involves ex vivo gene transfer using recombinant retroviral or lentiviral vectors of chimeric antigen receptors consisting of antibody-binding domains fused to T-cell-signaling domains into patient T lymphocytes.

As gene therapies are generally meant for one time or short duration treatments, they are customized to individuals confined to small patient populations. Therefore, manufacturing firms are expected to seek premium prices for these therapies. Because of this, these therapies will have to face valuation and reimbursement challenges. Stakeholders will show reservations about the hefty price tags and they will require significant data to be convinced.

With the removal of Gylbera from the EU market in 2017, due to the fact that only one patient was treated with the drug, all eyes are focused on the number of end patients that will be treated, and their ability to pay. To that end, launching of new drugs may have to be delayed in order to collect more data for payers. Furthermore, annuity based reimbursement agreements and pay-for-performance scenarios will have to be tackled.

Key Topics Covered:

1.0 Introduction

2.0 Gene Therapy: The Basics

3.0 Historic Overview of Gene Therapy

4.0 Gene Therapy Phase III Product Candidates

5.0 Gene Therapy Phase II Product Candidates

6.0 Commercialization, Cost and Warranty of Gene Therapies

7.0 The Strength of Gene Therapy Industry

8.0 Vectors for Gene Delivery

9.0 Clinical Applications of Gene Therapy

10.0 Market Analysis

11.0 Manufacturing of Viral Vectors and Logistics

12.0 Company Profiles

• AGCT
• Abeona Therapeutics LLC
• Addgene Inc.
• Advanced Cell & Gene Therapy LLC
• Advantagene Inc.
• Adverum Biotechnologies Inc.
• Agilis Biotherapeutics LLC
• Allogeneic Tumor Cell Bank
• Amgen
• AnGes MG Inc.
• Angionetics Inc.
• Applied Genetic Technologies Corporation (AGTC)
• Asklepios BioPharmaceutical Inc.
• Audentes Therapeutics Inc.
• AveXis Inc.
• AvroBio Inc.
• Bayer
• Benitec Biopharma
• BioCancell Therapeutic Inc.
• BioMarin Pharmaceutical Inc.
• BioVex
• Biogen Idec
• Bluebird bio Inc.
• Brammer Bio LLC
• Bristol Myers Squibb
• CRISPR Therapeutics AG
• Cellectis S.A.
• Chiesi
• Clontech Laboratories Inc.
• Cobra Biologics Ltd.
• Copernicus Therapeutics Inc.
• Dimension Therapeutics Inc.
• Editas Medicine Inc.
• Fibrocell Sciences Inc.
• Florida Biologix
• Fondazione Telethon
• Freeline Therapeutics Ltd.
• GTC's Technology
• GenSight Biologics S.A.
• GenVec
• Genable Technologies Ltd.
• Gene Mediated Cytotoxic Immunotherapy (GMCI)
• Genethon
• Genlantis
• Genzyme (Sanofi)
• GlaxoSmithKline
• Glybera
• Ichor Medical Systems Inc.
• Immune Design Corp.
• Immusoft Corp.
• Inovio Pharmaceuticals Inc.
• Intellia Therapeutics Inc.
• Juventa Therapeutics Inc.
• Kite Pharma Inc.
• Kolon Life Sciences Inc.
• Lentigen Technology Inc.
• Lysogene S.A.S.
• Medgenics Inc.
• Mirus Bio LLC
• Molecular Therapeutics LLC
• Mologen AG
• NanoCor Therapeutics Inc.
• Nature Technology Corp. (NTC)
• NightstaRx Ltd.
• Novartis
• Novasep Process SAS
• ORCA Therapeutics B.V.
• Omnia Biologics Inc.
• OrphageniX
• Oxford BioMedica plc
• Oxford Genetics Ltd.
• Pfizer
• REGENXBIO Inc.
• Renova Therapeutics Inc.
• RetroSense Therapeutics LLC
• Sangamo Biosciences Inc.
• Sanofi
• Sarepta Therapeutics Inc.
• Shanghai Sunway Biotech Co. Ltd.
• SiBiono GeneTech Co. Ltd.
• Sirion Biotech GmbH
• Spark Therapeutics Inc.
• Takara Bio Inc.
• Taxus Cardium Pharmaceutical Group Inc.
• Tocagen Inc.
• ToolGen Inc.
• Transgene SA
• uniQure N.V.
• University of Florida
• Vascular Biologics Ltd.
• Vical Inc.
• ViroMed Co., Ltd.
• Vivebiotech SL
• Voyager Therapeutics
• Xenon Pharmaceuticals Inc.

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