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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Report: https://www.coherentmarketinsights.com/market-insight/gene-therapy-for-rare-disease-market-2321
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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