The 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, 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) was valued at US$ 16.3 million in 2017, and is projected to exhibit a CAGR of 35.0% over the forecast period (2018–2026).

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. 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.

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Browse 26 Market Data Tables and 37 Figures spread through 240 Pages and in-depth TOC on 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, 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) - Global Forecast to 2026

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.

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Key Takeaways of the Global Gene Therapy for Rare Disease Market:

The global gene therapy for rare disease market is expected to exhibit a CAGR of 35.0% over the forecast period, owing to increasing research and development by key players in gene therapy.

Price for therapies are highly dependent on prevalence of the disease and therefore, some drugs (which are in development for ultra-rare disease) may be expensive

There could be a significant delay in commercialization of approved therapies in regions such as Latin America, Asia Pacific, Middle East, and Africa, as these therapies have novel technologies (as patient cells may be needed for modification and reintroduction into the patient) and high prices of gene therapy treatment.

Asia Pacific is expected to witness highest growth in gene therapy for rare disease market over the forecast period

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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