Gene Therapy for Rare Disease Market - Size, Share, Outlook, and Opportunity AnaPosted by priya saraf on July 30th, 2019 Gene Therapy for Rare Disease Market – Insights Gene therapy involves insertion of genetic material into cells of the patients to provide new function or to restore missing or inappropriate functioning of the cells due to cancer or gene mutations. According to National Center for Advancing Translational Sciences, although around 7,000 rare diseases are identified, only a few hundred have approved treatments. Moreover, developing gene therapy for rare diseases is gaining significant traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause. Small molecule drugs often treat symptoms only rather than curing underlying cause of the disease. This requires frequent administration of the drugs to manage the condition. Gene therapy holds the potential to correct underlying genetic defects. Moreover, a clinically sound and successful gene therapy may only require a single dose to cure a disease rather than requiring a lifetime treatment. Commercially approved gene therapies include Kymriah (Novartis AG), Yescarta (Gilead Sciences, Inc.), Luxturna (Spark Therapeutics), and Strimvelis (Orchard Therapeutics). Request A Sample Copy: http://bit.ly/2KgCfRh Increasing drug approvals and launches of novel gene therapies for treatment of various rare diseases are expected to boost the gene therapy for rare disease market growth over the forecast period In recent past, key players in the market have gained regulatory approvals and have launched their therapies in the market. Frequent approvals of novel gene therapies and their launches in the developed regions are expected to significantly drive gene therapy for rare disease market growth over the forecast period. For instance, in December 2017, Spark Therapeutics, Inc. received the U.S. Food and Drug Administration (FDA) approval for its LUXTURNA (voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Furthermore, in August 2018, Novartis International AG received European Commission’s (EC) approval for Kymriah (tisagenlecleucel, formerly CTL019) indicated for treatment of patients up to 25 years of age with relapse or refractory B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. Moreover, in August 2018, Kite Pharma, Inc., a Gilead Company, received European Commission (EC) Marketing approval for its novel Yescarta (axicabtagene ciloleucel), as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. The global gene therapy for rare disease market size was valued at US$ 16.3 million in 2017, and is expected to witness a robust CAGR of35.0% over the forecast period (2018–2026). Ongoing research and development in gene therapies for rare diseases is expected to boost growth of the global gene therapy for rare disease market Some of the major players operating in the global gene therapy for rare disease market include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. Click To Read More On Gene Therapy for Rare Disease Market About Coherent Market Insights: Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity. Contact Us: Mr.Shah Coherent Market Insights 1001 4th Ave, #3200 Seattle, WA 98154 Tel: +1-206-701-6702 Like it? Share it! More by this author |