Gene Therapy Market Value to Surge at a CAGR of 18.21% over 2025 to 2032; BioMarin Pharmaceutical Received U.S. FDA Approval for ROCTAVIAN to Secure Strong Market Hold

The global gene therapy market size stood at USD 3.43 billion in 2024. The market value is slated to rise from USD 4.29 billion in 2025 to USD 13.83 billion by 2032 at a CAGR of 18.21% during 2025-2032. Fortune Business Insights™ presents this information in its report, titled “Gene Therapy Market Size, Share & Industry Analysis by Product (Zolgensma, Luxturna, Roctavian, and Others), By Vector Type (Viral Vectors and Non-Viral Vectors), By Indication (Genetic Disorders, Ophthalmology, Hematology, and Others), By End User (Hospitals & Clinics, Specialty Clinics, and Others), and Regional Forecast, 2025-2032”.

Gene therapy is used for the treatment of patients ailing with congenital genetic disorders and acquired disorders. Over the forthcoming years, the demand for this technology is touted to depict a spike with the escalating prevalence of rare and genetic diseases. Several industry players are bringing new products to the market to tackle the challenges associated with non-specific and in-efficient delivery of genetic material. An instance is the rollout of three new AAV (adeno-associated virus) capsid gene delivery vectors by Dyno Therapeutics in May 2025. These vectors have been developed for targeting the CNS (central nervous system), musculoskeletal system, and the eye.

Rocket Pharmaceuticals’ RP-A601 Secured U.S. FDA’s RMAT Designation for Intensive FDA Guidance

In July 2025, Rocket Pharmaceuticals, Inc.’s RP-A601 secured U.S. FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation. RP-A601 is Rocket’s investigational AAV-based (adeno-associated virus) gene therapy for treating PKP2-arrhythmogenic investigational cardiomyopathy (ACM). The designation, which would provide the benefits of expedited review and added intensive FDA guidance, was awarded on the basis of positive efficacy and safety data from the Phase 1 RP-A601 clinical trial.

Gene Therapy Demand to Surge amid Globally Escalating Genetic Disorder Burden

In 2023, the number of patients with hemophilia stood at 218,804, according to the World Federation of Hemophilia. Over the recent years, other genetic disorders, including β-thalassemia, spinal muscular atrophy, and inherited retinal diseases have also depicted a rise, resulting in an escalated demand for curative treatments. The heightened demand for effective solutions against the backdrop of rising genetic disorder burden is anticipated to bolster product demand, impelling gene therapy market growth.

However, the adoption barriers that may stem from financial hindrances arising from high costs could hamper the expansion of the industry.

Product Innovation and R&D Have Emerged as Prime Areas of Focus for Major Players

Leading industry players, to augment their gene therapy market share, are emphasizing a range of initiatives. Research and development, product innovation, expansion initiatives, and strategic collaborations are among the key steps market players are deploying to increase their footprint. Some of the leading participants with a comprehensive therapy range comprise Hoffmann-La Roche Ltd, Novartis AG, and BioMarin Pharmaceutical Inc.

Key Players Profiled in the Report

• Novartis AG (Switzerland)
• Hoffmann-La Roche Ltd (Switzerland)
• BioMarin Pharmaceutical Inc. (U.S.)
• CSL (Australia)
• Vertex Pharmaceuticals Incorporated (U.S.)
• Krystal Biotech, Inc. (U.S.)
• Rocket Pharmaceuticals (U.S.)
• Dyno Therapeutics, Inc. (U.S.)

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https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

Key Industry Development

• June 2023 – BioMarin Pharmaceutical Inc., secured the approval of the U.S. FDA for its ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for treating severe hemophilia A patients. The solution is the first single-dose, one-time infusion approved for severe hemophilia A in the U.S.

Further Report Findings

• North America led the global market with a valuation of USD 1.03 billion in 2024. Robust government funding, presence of a strong healthcare infrastructure, and a surge in R&D initiatives are among the key aspects influencing the expansion of the regional market. The region is also characterized by approvals of novel gene therapies by the U.S. FDA an instance of which is the April 2025 approval of Abeno Therapeutics Inc.’s ZEVASKYN gene-modified cellular sheets.
• The Europe market is poised to expand at a rate of 7.82% over 2025 to 2032, impelled by the presence of national reimbursement initiatives, gene therapy research partnerships across academic centers, and favorable regulatory policies. These factors have also resulted in major European countries accounting for a significant valuation in 2025, with the U.K. touching USD 0.37 billion and Germany, a value of USD 0.56 billion.

Table of Segmentation