Gene Therapy for Rare Disease Market 2018 - Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics,

A new report published by Coherent Market Insights. Robust pipeline, recent success of newly launched gene therapies such as Kymriah, and Yescarta, and absence of robust therapies for several rare diseases is expected to boost growth of the global gene therapy for rare disease market.

Key players in the market are focused on research and development of gene therapies for rare diseases in order to introduce their pipeline products in the market. For instance, in October 2018, bluebird bio, Inc. received European Medicines Agency (EMA) marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.

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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. Moreover, in September 2018, Spark Therapeutics Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of LUXTURNA (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss

Increasing research and development by leading players in gene therapy for rare disease is expected to support growth of the gene therapy for rare disease market. Several companies have novel gene therapies in the clinical stages for treatment of rare diseases ranging from rare cancers to rare genetic diseases. For instance, Spark Therapeutics, Inc. has SPK-7001 (Choroideremia/ Phase ½), SPK-8011 (Hemophilia A/Phase ½), and SPK-3006 (Pompe Disease/preclinical) in the pipeline. Moreover, Orchard Therapeutics has multiple therapies in the pipeline such as OTL-101, OTL-103, OTL-102, OTL-200, and OTL-201 for various indications. Some of these therapies are expected to receive the U.S. Food & Drug Administration approvals during the forecast period. These factors are expected to boost the global gene therapy for rare disease market growth over the forecast period.

Launches of such novel therapies in near future is expected to significantly create lucrative opportunity for growth of the market. Moreover, increasing reimbursement support as well as innovative payment options are being adopted by manufacturers such as pay for performance. However, high prices of these therapies and potential challenges in commercialization of these therapies in emerging economies such as Asia Pacific and Latin America may restrain growth of the 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.

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Detailed Segmentation:

Global Gene Therapy for Rare Disease Market, By Drug:
Approved Drugs
Tisagenlecleucel (Kymriah)
Axicabtagene ciloleucel (Yescarta)
Voretigene neparvovec (Luxturna)
Strimvelis
Pipeline Drugs
GT-AADC
Fidanacogene elaparvovec (SPK-9011)
OTL-200
bb2121
AMT-061
Others
Global Gene Therapy for Rare Disease Market, By Therapeutic Application:
Oncology
Neurological Disorders
Ophthalmic Disorders
Hematological Disorders
Immunodeficiency Disorders
Metabolic Disorders
Others

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.

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