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The Global Gene Therapy for Rare Disease Market Analysis| Coherent Market Insights

The Global Gene Therapy for Rare Disease Market is expected to witness a robust CAGR of 35.0% during 2018u20132026.

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The Global Gene Therapy for Rare Disease Market Analysis| Coherent Market Insights

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  1. Coherent Market Insights Market Research and Business Consulting Services https://www.coherentmarketinsights.com/ COVID-19 Impact Tracker

  2. GENE THERAPY FOR RARE DISEASE MARKET ANALYSIS Gene Therapy for Rare Disease Market,By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtageneciloleucel (Yescarta), Voretigeneneparvovec (Luxturna), Strimvelis), Pipeline Drugs (GT-AADC, Fidanacogeneelaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others),   By Therapeutic Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), and Region (North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2018 – 2026

  3. 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.Accordingto National Center for Advancing Translational Sciences, although around 7,000 rare diseases are identified,onlya few hundred have approved treatments. Moreover,developinggene therapy for rare diseases is gaining significant traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause.Smallmolecule drugs often treat symptoms only rather than curing underlying cause of the disease.Thisrequires frequent administration of the drugs to manage the condition.Genetherapy 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.Commerciallyapproved gene therapies include Kymriah (Novartis AG), Yescarta (Gilead Sciences, Inc.), Luxturna (Spark Therapeutics), and Strimvelis (Orchard Therapeutics).

  4. Figure No.1: Global Gene Therapy for Rare Disease Market Share, By Drug (%), 2018 -2026 

  5. Market Growth In recent past,keyplayers 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.Forinstance,inDecember 2017, Spark Therapeutics,Inc. received the U.S.Foodand Drug Administration (FDA) approval for its LUXTURNA (voretigeneneparvovec-rzyl),a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.Furthermore,inAugust 2018,Novartis International AG received European Commission’s (EC) approval for Kymriah (tisagenlecleucel,formerlyCTL019) 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.

  6. Moreover, in August 2018, Kite Pharma, Inc., a Gilead Company, received European Commission (EC) Marketing approval for its novel Yescarta (axicabtageneciloleucel), 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 of 35.0% over the forecast period (2018–2026).

  7. Table No.1: Gene Therapy for Rare Disease Pipeline

  8. Ongoing research and development in gene therapies for rare diseases is expected to boost growth of the global gene therapy for rare disease market • Presence of multiple novel gene therapies for various rare disease treatment in pipeline, which are expected to gain approval is expected to boost the market growth over the forecast period. For instance, Spark Therapeutics, Inc. is developing gene therapy namely FidanacogeneElaparvovec, a novel, investigational gene therapy indicated for the treatment of patients suffering from hemophilia B. Currently, the therapy is under Phase 3 clinical trial and is expected to receive approval in near future.

  9. In August 2018, uniQure N.V. treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In May 2018, Orchard Therapeutics Plc. received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate OTL-200, indicated for treatment of patients with metachromatic leukodystrophy (MLD).  PTC Therapeutics, In. has GT-AADC in phase 3 clinical trials and is indicated for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.

  10. However, lack of awareness among population regarding rare diseases and their treatment is expected to negatively affect growth of the gene therapy for rare disease market. For instance, in 2017, uniQure N.V. dropped their plant to pursue the renewal of its gene therapy Glybera, which was approved in 2012, for treatment for a small subset of patients with familial lipoprotein lipase deficiency (LPLD). The company dropped their plan due to very low demand of therapy in the market

  11. Competitive Section 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.

  12. Request Sample Report • Request Customization • Download PDF Brochure • Talk to Analyst Reference: https://www.coherentmarketinsights.com/market-insight/gene-therapy-for-rare-disease-market-2321

  13. About Us Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. What we provide: • Customized Market Research Services • Industry Analysis Services • Business Consulting Services • Market Intelligence Services • Long term Engagement Model • Country Specific Analysis

  14. Contact Us Mr. Shah Coherent Market Insights Pvt.Ltd. Address: 1001 4th Ave, #3200 Seattle, WA 98154, U.S. Phone: +1-206-701-6702 Email: sales@coherentmarketinsights.com

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