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

Gene Therapy for Rare Disease   Market was valued at US$ 144.5 million in 2024 and is projected to grow at a CAGR of 5.10% to reach US$ 3848.7 million by 2034.

Gene therapy for rare disease involves insertion of genetic material into cells of the patients in order to provide new function as well as to restore missing function cells due to cancer or gene alteration. Gene therapies have ability to cure the genetic defects. Gene therapies can be used to treat rare disorders like Hemophilia B, Hemophilia A, Fabry disease, and neuronopathic disorders.

Increasing approvals of drugs for treatment of rare diseases by the regulatory bodies across the globe is a key factor driving growth of the gene therapy for rare disease market. In addition, increasing launches of various gene therapies across the globe is a factor expected to propel growth of the global market in the near future. Furthermore, ongoing research and development activities in the gene therapies for treatment of rare diseases is another factor expected to boost growth of the Gene Therapy for Rare Disease market over the forecast period.

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Launch of novel therapies with growing drug approvals to fuel market growth

Launch of novel therapies with growing number of drug approvals has contributed in Gene Therapy for Rare Disease market growth. Presence of major key players and strategic partnerships has given rise in launch of new novel therapies which, in turn boost the market growth.

For instance, in October 2021, U.S. FDA (Food and Drug Administration), five profit organizations and ten pharmaceutical companies joined forces to develop gene therapies for rare diseases. By standardizing process and leveraging on experience with platform technology, gene therapy product development will be accelerated to allow more timely access to new therapies for needy patients.

Restrains:

However, high costs associated with treating rare diseases, coupled with lack of awareness regarding gene therapy among the individuals are major factors expected to restrain growth of the Gene Therapy for Rare Disease market over the forecast period.

Gene Therapy for Rare Disease Market is segmented based on Drug, Therapeutic Application and Region.

Drug Insight

On the basis of Drug, Gene Therapy for Rare Disease Market is segmented into Approved Drugs - Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and Strimvelis. On the basis of pipeline drugs market has been segmented as GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and others.

Therapeutic Application Insights

On the basis of Therapeutic Application, Gene Therapy for Rare Disease Market is segmented into Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others. Oncology segment is expected to dominate the market growth due to growing prevalence of cancer diseases.

For instance, in 2019, according to CDC (Centers for Disease Control and Prevention), around 1,752,735 new cancer cases were reported and 599,589 people were died of cancer in United States.

Regional Insights:

On region the Gene Therapy for Rare Disease Market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The North America market is estimated to witness a significantly high revenue share over the forecast period. Moreover, rising prevalence of rare disease, unhealthy diet, sedentary lifestyle, high disposable income, growing research and development activities, new product launch and approval of drugs is anticipated to increase demand for Gene Therapy for Rare Disease in the region.

For instance, according to CDC (Centers for Disease Control and Prevention), in United States rare disease affects fewer than 200,000 individual and as a whole affect about 25 million individual in United States and around 400 million individuals worldwide.

Report Scope:

Segments Covered in the Report:

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends and opportunities in each of the sub-segments from 2022 to 2032. For the purpose of this study, has segmented the Gene Therapy for Rare Disease Market report based on Drug, Therapeutic Application and Region.

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

Gene Therapy for Rare Disease Market, By Therapeutic Application:

• Oncology
• Neurological Disorders
• Ophthalmic Disorders
• Hematological Disorders
• Immunodeficiency Disorders
• Metabolic Disorders
• Others

Gene Therapy for Rare Disease Market, By Region:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Russia
  • Italy
  • Rest of Europe
• Asia Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia Pacific
• Latin America
  • Brazil
  • Mexico
  • Rest of Latin America
• Middle East & Africa
  • GCC
  • Israel
  • South Africa
  • Rest of Middle East & Africa

The key players operating 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.

Recent Development:

• In March 2023, England’s NICE (National Institute for Health and Care Excellence) signs off an PTC Therapeutics “Upstaza” a gene therapy for ultra-rare genetic disease at $3.7 million to patients in U.K.
• In April 2022, Novartis received FDA (Food and Drug Administration) approval for its first “Vijoice” treatment for pediatric and adult patients with severe PIK3CA – PROS (Related Overgrowth Spectrum) a spectrum of rare condition due to growth and blood vessel anomalies.
• In May 2019, AaveXis received FDA (Food and Drug Administration) approval for its first and only gene therapy “Zolgensma” for pediatric patients with SMA (Spinal Muscular Atrophy) a rare genetic disease which leads to paralysis and progressive muscle weakness.

• Kite Pharma, Inc. (Gilead Sciences, Inc.)*
  • Company Overview
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Business Strategies

• Novartis International AG
• Juno Therapeutics Inc.  (Celgene Corporation)
• Bluebird Bio, Inc.
• Spark Therapeutics, Inc.
• UniQure N.V
• Orchard Therapeutics Plc.
• PTC Therapeutics, Inc.
• Biomarin Pharmaceutical Inc.

“*” marked represents similar segmentation in other categories in the respective section