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.

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

Key features of the study

• This report provides in-depth analysis of gene therapy for rare disease market and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR %) for the forecast period (2026 - 2033), considering 2025, as the base year
• It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategy adopted by the leading players
• It profiles leading players in the global Gene Therapy for Rare Disease Market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies
• Key companies covered as a part of this study 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.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launches, by Technology up-gradation, market expansion, and marketing tactics
• The global Gene Therapy for Rare Disease Market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision making through the various strategy matrices used in analyzing the Gene Therapy for Rare Disease Market

Market Segmentation

• By Drug (Revenue, USD Mn, 2021-2033)
  • 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
• By Therapeutic Application (Revenue, USD Mn, 2021-2033)
  • Oncology
  • Neurological Disorders
  • Ophthalmic Disorders
  • Hematological Disorders
  • Immunodeficiency Disorders
  • Metabolic Disorders
  • Others
• Global Gene Therapy for Rare Disease Market, By Region: (Revenue, USD Mn, 2021-2033)
  • North America
    • U.S.
    • Canada
  • Latin America
    • Brazil
    • Mexico
    • Argentina
    • Rest of Latin America
  • Europe
    • Germany
    • U.K.
    • France
    • Italy
    • Spain
    • Russia
    • Rest of Europe
  • Asia Pacific
    • China
    • India
    • Japan
    • Australia
    • South Korea
    • ASEAN
    • Rest of Asia Pacific
  • Middle East
    • GCC
    • Israel
    • Rest of Middle East
  • Africa
    • South Africa
    • Central Africa
    • North Africa
• Key Players
  • 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.
  • BioMarin Pharmaceutical Inc.