Gene Therapy Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2021-2031

The Global Gene Therapy Market is projected to expand from USD 7.54 Billion in 2025 to USD 13.02 Billion by 2031, reflecting a Compound Annual Growth Rate (CAGR) of 9.53%. Gene therapy is characterized as a medical intervention that alters the genetic instructions within a patient's cells - by replacing, inactivating, or introducing genes - to treat or prevent diseases. This market growth is primarily fueled by the increasing prevalence of rare genetic disorders and chronic conditions, which generate an urgent need for long-lasting therapeutic solutions. Additionally, advancements in viral vector technologies and streamlined regulatory designations are accelerating the approval process, facilitating the transition of clinical research into commercially viable products.

Despite this progress, the high complexity of manufacturing and its associated costs present a significant barrier to market scalability and accessibility. These economic factors often lead to pricing structures that burden healthcare reimbursement systems and restrict patient adoption. According to the American Society of Gene & Cell Therapy, oncology indications comprised 64% of the 80 newly initiated gene therapy clinical trials in the second quarter of 2025. This statistic underscores the sector's continued clinical focus on complex indications, even in the face of substantial financial hurdles.

Market Drivers

Supportive regulatory frameworks and expedited approval pathways are fundamentally transforming the market by accelerating the commercialization of novel treatments. Regulatory agencies have implemented specialized designations, such as the FDA's Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy status, which streamline the review process for complex biologic products and shorten the timeline between clinical validation and patient access. This pro-innovation environment has driven a tangible increase in successful market entries, effectively validating emerging viral vector and gene-editing technologies. According to Fierce Pharma in January 2026, the FDA approved nine new cell and gene therapies in 2024, setting a record for the sector; these regulatory milestones reduce risks for future programs and encourage sustained industrial participation by demonstrating a clear path to commercialization.

Concurrent with regulatory advancements, a surge in investment and funding for life sciences research is propelling market expansion by providing the essential capital needed for high-cost clinical development and manufacturing scale-up. Biopharmaceutical developers continue to secure significant financial backing to advance their pipelines, ensuring that innovative candidates can progress through rigorous testing phases despite broader economic fluctuations. According to the Alliance for Regenerative Medicine in January 2025, the sector attracted $15.2 billion in global investment in 2024, representing a 30% year-over-year increase. This influx of capital supports a diverse and expanding ecosystem of therapeutic options, with the American Society of Gene & Cell Therapy reporting in November 2025 that the global pipeline included more than 3,200 active clinical trials for gene, cell, and RNA therapies.

Market Challenges

The high complexity of manufacturing and the associated costs constitute a formidable barrier that directly impedes the scalability and commercial potential of the Global Gene Therapy Market. Unlike small-molecule drugs, gene therapies depend on intricate biological processes, such as viral vector production, which are inherently difficult to standardize and scale. This lack of manufacturing efficiency results in exorbitant Cost of Goods Sold (COGS), forcing companies to set high list prices that strain healthcare budgets. Consequently, payers often impose strict reimbursement criteria, which limits patient access and reduces the revenue potential necessary to sustain further innovation.

Furthermore, the capital-intensive nature of overcoming these manufacturing hurdles creates a challenging financial environment for developers. Establishing facilities that comply with Good Manufacturing Practice (GMP) requires massive upfront capital, draining resources that could otherwise fund pipeline expansion. According to the Alliance for Regenerative Medicine, the sector raised approximately $10.9 billion in investment during the first half of 2024. While this funding is substantial, the extreme costs associated with biomanufacturing and supply chain logistics mean that a significant portion of this capital is absorbed by operational expenses rather than clinical advancement, thereby slowing the overall rate of market growth and product commercialization.

Market Trends

The proliferation of strategic partnerships with Contract Development and Manufacturing Organizations (CDMOs) is currently addressing critical manufacturing bottlenecks. By outsourcing to CDMOs, developers gain access to specialized viral vector infrastructure without the prohibitive capital costs of building internal facilities. This strategy mitigates technical risks and expedites clinical validation through established regulatory pathways, allowing innovators to focus resources on pipeline development rather than process engineering. According to a press release from Matica Biotechnology in October 2025 titled 'Matica Biotechnology Emerges as the Go-To CDMO,' the company secured over 10 new manufacturing projects in the first half of 2025, reflecting the sector's rapid adoption of externalized production to support expanding pipelines.

Simultaneously, the shift from ex vivo to direct in vivo administration routes is transforming the therapeutic landscape for complex tissue targets. Unlike ex vivo methods, which are limited to accessible cells, direct in vivo delivery utilizes advanced vectors to treat deep anatomical sites such as the central nervous system. This approach simplifies logistics and extends the reach of gene therapy to previously untreatable neurological conditions. According to the International Society for Cell & Gene Therapy's January 2025 article 'Cell & Gene Therapy Approvals in 2024,' the approval of Kebilidi marked a milestone as the first gene therapy directly administered to the brain, confirming the viability of this administration route.

Key Players Profiled in the Gene Therapy Market

• REGENXBIO Inc.
• Oxford Biomedica plc
• Voyager Therapeutics Inc.
• Human Stem Cells Institute
• Dimension Therapeutics, Inc.
• Bristol-Myers Squibb Company
• Sanofi
• F. Hoffmann-La Roche Ltd.
• bluebird bio, Inc.
• Novartis AG
• uniQure NV.

Report Scope

In this report, the Global Gene Therapy Market has been segmented into the following categories:

Gene Therapy Market, by Vector Type:

• Viral Vector
• Non-Viral Vector

Gene Therapy Market, by Delivery Method:

• In-Vivo
• Ex-Vivo

Gene Therapy Market, by Indication:

• Rare Diseases
• Cancer
• Neurological Diseases
• Others

Gene Therapy Market, by Region:

• North America
• Europe
• Asia-Pacific
• South America
• Middle East & Africa

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Gene Therapy Market.

Available Customization

The analyst offers customization according to your specific needs. The following customization options are available for the report:

• Detailed analysis and profiling of additional market players (up to five).

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