The global GeneTherapy 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 (NorthAmerica, Latin America, Europe, Asia Pacific, the Middle East, and Africa) wasvalued 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 successof newly launched gene therapies such as Kymriah, and Yescarta, and absence ofrobust therapies for several rare diseases is expected to boost growth of theglobal gene therapy for rare disease market. Key players in the market are focused on research and development ofgene therapies for rare diseases in order to introduce their pipeline productsin the market. For instance, in October 2018, bluebird bio, Inc. receivedEuropean Medicines Agency (EMA) marketing authorization application (MAA) forits investigational LentiGlobin gene therapy for the treatment of adolescentsand adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0genotype. In August 2018, Kite Pharma, Inc., a Gilead Company, receivedEuropean Commission (EC) Marketing approval for its novel Yescarta(axicabtagene ciloleucel), as a treatment for adult patients with relapsed orrefractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal largeB-cell lymphoma (PMBCL), after two or more lines of systemic therapy. Moreover,in September 2018, Spark Therapeutics Inc. announced that the Committee forMedicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)adopted a positive opinion recommending approval of LUXTURNA (voretigeneneparvovec), a one-time gene therapy for the treatment of adult and pediatricpatients with vision loss.
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Browse 26 Market Data Tables and37 Figures spread through 240 Pages and in-depth TOC on Global Gene Therapy forRare Disease Market By Drug (ApprovedDrugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta),Voretigene neparvovec (Luxturna), Strimvelis), Pipeline Drugs (GT-AADC,Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, andOthers), By Therapeutic Application(Oncology, Neurological Disorders, Ophthalmic Disorders, HematologicalDisorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), andRegion (North America, Latin America, Europe, Asia Pacific, the Middle East,and Africa) - Global Forecast to 2026
Increasing research anddevelopment by leading players in gene therapy for rare disease is expected tosupport growth of the gene therapy for rare disease market. Several companieshave novel gene therapies in the clinical stages for treatment of rare diseasesranging from rare cancers to rare genetic diseases. For instance, SparkTherapeutics, Inc. has SPK-7001 (Choroideremia/ Phase ½), SPK-8011 (HemophiliaA/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 thesetherapies are expected to receive the U.S. Food & Drug Administrationapprovals during the forecast period. These factors are expected to boost theglobal gene therapy for rare disease market growth over the forecastperiod.
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Key Takeaways of the Global GeneTherapy for Rare Disease Market:
The global gene therapy for raredisease 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 highlydependent on prevalence of the disease and therefore, some drugs (which are indevelopment for ultra-rare disease) may be expensive
There could be a significantdelay in commercialization of approved therapies in regions such as LatinAmerica, Asia Pacific, Middle East, and Africa, as these therapies have noveltechnologies (as patient cells may be needed for modification andreintroduction into the patient) and high prices of gene therapy treatment.
Asia Pacific is expected towitness highest growth in gene therapy for rare disease market over theforecast period
Major players operating in theglobal gene therapy for rare disease market include Kite Pharma, Inc. (GileadSciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (CelgeneCorporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V,Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin PharmaceuticalInc.
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