Gene Therapy for Rare disease Market is estimated to be US$ 3848.7 million by 2034; Increase demand for Gene Therapy and its technologies to Fuel Market Growth

Gene therapy for rare disorders like Hemophilia A, Hemophilia B, Fabry disease and Neuronopathic disorder as well. It involves the insertion of genetic material into cells of the patients so as to provide new functions as well as to restore missing function cells due to cancer or gene alterations. The increase in demand for gene therapy and advancement in the field of genetic engineering and healthcare are one of the driving factors that are expected to boost the growth of the target market. 

The growth of Gene Therapy market is influenced by different factors such as strong product portfolio or the expanding investments by key market players. The growing demand of better-quality healthcare for cancers and genetic disorders is expected to foster the demand for Gene Therapy for Rare disease Market growth in coming years.

The report “Gene Therapy for Rare Disease Market, By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and 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 By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2034”

 Key Highlights:

• In February 2022, Sarepta Therapeutics (US) announced a collaboration NanoGalaxy platform and Sarepta’s gene editing technology to develop a gene editing therapeutics for the treatment of neuromuscular diseases.

• In May 2022, Novartis AG (Switzerland) received FDA approval for KYMRIAH which are used in the old age people that are suffering from relapsed or refractory follicular lymphoma.

Analyst View:

The rapid technological advancement in cellular and molecular biology in genomic research have played an important contribution to drive the growth of the Gene Therapy for Rare disease Market. Technological advancement is expected to boost the demand for Gene Therapy for Rare disease Market growth in future. It leads to broadening of the market and eventually beef up the competition. Big international corporations and start-ups are maneuvering to establish position in the Gene Therapy for Rare disease market.

Browse 60 market data tables* and 35 figures* through 140 slides and in-depth TOC on “Gene Therapy for Rare Disease Market, By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and 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 By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2034”

Key Market Insights from the report:

Gene Therapy for Rare disease Market accounted for US$ 144.5 million in 2024 and is estimated to be US$ 3848.7 million by 2034 and is anticipated to register a CAGR of 5.10%. The Gene Therapy for Rare disease Market is segmented based on Drug, Therapeutic Application and Region.

• Based on Drug, Gene Therapy for Rare disease Market is segmented into Approved Drugs- Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and Strimvelis. Pipeline Drugs- GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and others.
• Based on Therapeutic Application, Gene Therapy for Rare disease Market is segmented into Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others.
• By Region, the Gene Therapy for Rare disease Market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.

Competitive Landscape & their strategies of Gene Therapy for Rare disease Market:

The prominent players operating in the Gene Therapy for Rare disease Market includes, 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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