Global Strategic Report
264 Pages; December 2019
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 a detailed market size determination.
Representing a new frontier of medicine, gene therapy made its grand arrival in the U.S. in 2017 through the FDA approval of 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.
- Market value of the forthcoming gene therapy products.
- Projected number of gene therapy products to gain FDA approval within five years.
- 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.
- Global market for gene therapy products by therapy type and geography.
- 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. It features company profiles for 83 gene therapy competitors worldwide, revealing their core technologies and product pipelines.
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:
- Identify the preclinical and clinical product pipeline for gene therapy products globally.
- Reveal the identities of all known competitors developing gene therapies and supporting technologies.
- Determine company strategy or purse external funding from investors for gene therapy technologies.
- Identify potential collaborators and co-development partners
- Make smarter decisions, faster.
With an online readership of nearly one million viewers per year, BioInformant is a U.S. market research firm with 15 years of experience. As the first and only market research firm to specialize in the Regenerative Medicine (RM)l industry, BioInformant research has been cited by the Wall Street Journal, Xconomy, and Vogue Magazine. Founded in 2006 and headquartered in Washington, DC, BioInformant is strategically positioned to be near the National Institutes of Health (NIH), the U.S. FDA, the Maryland Biotech Corridor, and policy makers on Capitol Hill.
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With the competitive nature of this global market, you do not have the time to do the research. Claim this report to become immediately informed, without sacrificing hours of unnecessary research or missing critical opportunities.
The content within this report was compiled using a diverse range of sources, as described in this Research Methodology.
TABLE OF CONTENTS: Global Market for Gene Therapy– Product Pipeline, Market Size, & Forecasts
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