Global Market for Gene Therapy 2019 – Product Pipeline, Market Size, & Forecasts
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:
Learn how to effectively navigate the market research process to help guide your organization on the journey to success.Download eBook