Gene Therapy for Rare Disease Market 2021 : Future Growth Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics

In recent past, most of the gene therapies received orphan drug designations. Orphan drugs are generally defined as those medicines with one or more indications approved under the Orphan Drug Act of 1983. The Orphan Drug Act supports the development of innovative treatments for rare disease patients.

The creation of the orphan drug designation with the passage of the Orphan Drug Act in 1983 has facilitated the development and approval of drugs for rare diseases and 2017 and 2018, were marked by the highest number of orphan drug and indication approvals to date. Production of gene therapies is associated with use of high-end technologies, high research and development costs, and skilled scientists and researchers, which reflects in high prices of these therapies.

Request PDF Brochure of this report:
https://www.coherentmarketinsights.com/insight/request-pdf/2321

Market Dynamics

Key players in the market are dedicated to the development of gene therapies for the novel indications such as Huntington’s disease for which there are no specific approved medication. For instance, uniQure N.V. is developing AMT-130, a novel Gene therapy for Huntington’s disease (HD), a rare, fatal, neurodegenerative genetic disorder. AMT-130 is currently in pre-clinical trial and is expected to receive approval in near future.

Furthermore, companies are developing novel gene therapies for rare diseases such as Hemophilia B and Hemophilia A, which can offer long term treatment and can replace lifelong medication for this diseases. 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

Request Here Sample Report:
https://www.coherentmarketinsights.com/insight/request-sample/2321

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

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.

Tailored Information as per niche requirement:
LIMITED TIME OFFER – Hurry Up!
Exclusive offer!!! Purchase the report at a discounted rate!!!
Get Discount for Buyers UPTO 30% OFF On Any Research Report

Buy Now with Discount for Premium Report 2021:
https://www.coherentmarketinsights.com/insight/buy-now/2321

Coherent Market Insights 1001 4th Ave,
#3200 Seattle, WA 98154,U.S.
Phone: US +1-206-701-6702/UK +44-020 8133 4027
Email: sales@coherentmarketinsights.com

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.

This release was published on openPR.