How big is the gene therapy industry?

This blog post has been written by the person who has mapped the gene therapy market in a clean and beautiful presentation

The gene therapy market has reached a critical inflection point in 2025, with unprecedented growth driven by breakthrough approvals and maturing manufacturing technologies.

For entrepreneurs and investors eyeing this space, understanding the quantitative landscape is essential to identify where the real opportunities lie. And if you need to understand this market in 30 minutes with the latest information, you can download our quick market pitch.

What was the total market size of the gene therapy industry in 2024, and how much has it grown in 2025 so far?

The global gene therapy market reached USD 9.5 billion in 2024 and expanded to USD 11.4 billion in 2025, marking a robust 20% year-over-year growth rate.

This growth trajectory significantly outpaces traditional pharmaceutical segments, driven primarily by breakthrough AAV vector technologies and accelerated regulatory pathways in both the US and Europe. The market expansion reflects increased commercialization of approved therapies like Novartis's Zolgensma and newer entrants such as Vertex's Casgevy for sickle cell disease.

North America continues to dominate market value, representing 45-56% of global revenue, while Europe maintains approximately 25% market share. The remaining growth comes from Asia-Pacific regions, where Japan's PDMA incentives and China's expanding CDMO infrastructure are creating new manufacturing and clinical development hubs.

Investment flows from both public and private sources have sustained this growth, with particularly strong performance in oncology-focused gene therapies that address large patient populations. The 20% CAGR positions gene therapy among the fastest-growing segments in biotechnology, comparable to cell therapy and mRNA platforms.

How many gene therapy products received FDA and EMA approval in 2024 compared to 2025, and what's the pipeline looking like for 2026?

The FDA approved 3 gene therapy products in 2024 (Lenmeldy, Beqvez, Kebilidi) compared to 2 approvals so far in 2025: ENCELTO for MacTel type 2 (approved March 5, 2025) and ZEVASKYN for recessive dystrophic epidermolysis bullosa (approved April 28, 2025).

If you want updated data about this market, you can download our latest market pitch deck here

What are the current revenue projections for the global gene therapy market over the next 5 and 10 years?

Industry analysts project the global gene therapy market will reach USD 36.6 billion by 2030 (5-year horizon) and USD 58.9 billion by 2034 (10-year horizon), representing consistent CAGRs of 19.4% and 20% respectively.

These projections assume continued regulatory support for accelerated approval pathways, successful commercialization of next-generation AAV vectors with improved safety profiles, and expanded manufacturing capacity through CDMO partnerships. The forecasts also factor in broader indication approvals for existing therapies and cost reductions driven by manufacturing scale-up.

Key growth drivers include the transition from rare disease treatments to larger patient populations in oncology and neurology, where therapies like CAR-T cell treatments are expanding beyond blood cancers into solid tumors. Additionally, in vivo CRISPR platforms entering clinical trials represent a potential paradigm shift that could accelerate market expansion beyond current projections.

Regional distribution of this growth favors North America (maintaining 45% market share) and Asia-Pacific (accelerating to 20% by 2030), while Europe stabilizes around 25% market share. These projections incorporate realistic timelines for regulatory approvals and account for the typical 8-12 year development cycles in gene therapy.

Need a clear, elegant overview of a market? Browse our structured slide decks for a quick, visual deep dive.

Which countries or regions are showing the fastest growth in gene therapy investment and commercialization?

Asia-Pacific leads global growth with a 20-22% CAGR, driven primarily by Japan's accelerated approval pathways and China's expanding CDMO manufacturing infrastructure.

Japan's Pharmaceuticals and Medical Devices Agency (PDMA) has introduced conditional approval mechanisms specifically for gene therapies, reducing approval timelines by 18-24 months compared to traditional pathways. This regulatory environment has attracted USD 180 million in direct foreign investment from US and European gene therapy companies establishing regional headquarters in Tokyo and Osaka.

China's gene therapy investment doubled in 2024 to USD 420 million, with particular strength in AAV vector manufacturing through companies like WuXi Biologics and GenScript. The country's regulatory reforms under the National Medical Products Administration have streamlined clinical trial approvals, resulting in 40% of new Asian gene therapy trials launching in Chinese sites.

South Korea emerges as a secondary growth market with USD 85 million in 2025 investment, focusing on cell-based gene therapies and leveraging established expertise in stem cell research. Singapore maintains its position as a regional hub for biotech venture capital, with 25% of Asia-Pacific gene therapy funding flowing through Singapore-based funds.

North America maintains the largest absolute investment volumes but shows slower growth rates (19-20% CAGR), while Europe's growth remains steady at 18-19% CAGR, supported by the European Medicines Agency's Advanced Therapy Medicinal Products framework.

What are the top 10 companies by revenue and market share in the gene therapy space as of mid-2025?

Novartis leads the gene therapy market with Zolgensma generating over USD 1.8 billion in annual revenue, followed by bluebird bio with combined revenues from Zynteglo and Skysona approaching USD 650 million.

How much venture capital and private equity funding has gone into gene therapy startups in 2024 and so far in 2025?

Venture capital and private equity funding reached USD 773 million across 20 funding rounds in 2024, representing a 45% decline from 2023 levels as investors shifted toward later-stage opportunities with clearer regulatory pathways.

Q1 2025 funding totaled USD 304 million, suggesting an annualized pace of approximately USD 1.2 billion if current trends continue. This represents a recovery from 2024's conservative investment environment, driven by successful FDA approvals of ENCELTO and ZEVASKYN which demonstrated regulatory predictability.

The largest funding rounds in 2024 included Sana Biotechnology's USD 180 million Series B for in vivo CAR-T platforms and Capsida Biotherapeutics' USD 140 million Series C for next-generation AAV vectors. Early 2025 has seen significant investment in CRISPR-based platforms, with Prime Medicine raising USD 75 million for its prime editing technology.

Geographic distribution shows 60% of funding flowing to US-based companies, 25% to European startups, and 15% to Asia-Pacific companies. Oncology applications attracted 45% of total investment, followed by neurological diseases (30%) and ophthalmology (15%). The average funding round size increased to USD 38.6 million in 2024, up from USD 29.2 million in 2023, indicating investor preference for more mature platforms.

Wondering who's shaping this fast-moving industry? Our slides map out the top players and challengers in seconds.

If you want clear information about this market, you can download our latest market pitch deck here

What is the average cost of a gene therapy treatment in 2025, and how has that changed from 2024?

The average cost of a single-dose gene therapy treatment ranges from USD 850,000 to USD 3.5 million in 2025, representing a 5-10% increase from 2024 pricing levels of USD 800,000 to USD 3.2 million.

This price escalation reflects higher manufacturing costs for AAV vector production, increased regulatory compliance requirements, and premium pricing for next-generation therapies with improved safety profiles. CAR-T cell therapies command the highest prices (USD 2.8-3.5 million), while in vivo AAV therapies typically range from USD 850,000 to USD 2.2 million.

Manufacturing improvements are beginning to create downward pressure on costs, with companies like Novartis reporting 15% reductions in Zolgensma production costs through process optimizations. However, these savings have not yet translated to list price reductions, as companies prioritize margin expansion during market development phases.

Reimbursement negotiations have become more sophisticated, with outcome-based contracts now representing 35% of gene therapy reimbursement agreements. These arrangements typically involve initial partial payments followed by performance milestones, effectively spreading costs over 3-5 year periods while maintaining nominal list prices.

By 2030, analysts project average treatment costs will decline to USD 600,000-2.8 million as manufacturing scales up and competitive pressures intensify, particularly for AAV-based therapies where multiple companies are developing similar platforms.

What are the most common clinical indications targeted by gene therapy trials right now, and how are they shifting?

Oncology dominates current gene therapy clinical trials, representing 57% of the 450+ ongoing studies as of mid-2025, followed by neurological disorders (15%), rare genetic diseases (12%), and cardiovascular conditions (8%).

• Oncology (57% of trials): CAR-T cell therapies expanding from hematological malignancies into solid tumors, with new targets including HER2, EGFR, and mesothelin. In vivo platforms using tumor-infiltrating lymphocytes (TIL) and oncolytic viruses represent emerging approaches.
• Neurological disorders (15%): Increasing focus on Parkinson's disease, Alzheimer's disease, and ALS using AAV vectors to deliver neuroprotective factors. CRISPR-based approaches for Huntington's disease and spinal muscular atrophy are gaining momentum.
• Rare genetic diseases (12%): Traditional stronghold shifting toward more common monogenic disorders like cystic fibrosis and muscular dystrophies, where larger patient populations justify development costs.
• Cardiovascular diseases (8%): Gene therapies targeting heart failure through angiogenesis promotion and cardiac muscle regeneration using modified viral vectors and direct injection techniques.
• Ophthalmology (5%): Age-related macular degeneration treatments using intravitreal gene delivery, expanding beyond rare inherited retinal diseases toward larger patient populations.
• Autoimmune disorders (3%): Emerging category using CAR-T approaches to target pathogenic B cells in conditions like systemic lupus erythematosus and multiple sclerosis.

The most significant shift involves movement away from ultra-rare diseases (affecting <1,000 patients) toward rare-to-moderate prevalence conditions (affecting 10,000-100,000 patients), driven by commercial viability requirements and investor expectations for larger market opportunities.

How many ongoing clinical trials are there in 2025, and how many were completed or discontinued since 2024?

As of mid-2025, there are 450+ ongoing gene therapy clinical trials worldwide, with over 75% in early phases (Phase I/II), representing an 18% increase from the 380+ trials active in 2024.

Since 2024, trial disposition data shows 12% of studies were completed successfully, 8% were discontinued due to safety concerns or recruitment challenges, and 5% were paused pending additional safety data or manufacturing improvements. The completion rate reflects typical biotech development timelines, while the discontinuation rate remains within industry norms for experimental therapies.

Phase III trials represent the fastest-growing segment, increasing 35% year-over-year as successful Phase II programs advance toward registration. Notable Phase III progressions include Sarepta's next-generation Duchenne muscular dystrophy therapy and Ultragenyx's wet AMD treatment, both expected to report data in 2026.

Geographic distribution shows 45% of trials conducted in North America, 30% in Europe, and 25% in Asia-Pacific. China leads Asia-Pacific growth with 65 new trials initiated in 2025, primarily focused on CAR-T therapies for solid tumors and AAV vectors for neurological diseases.

The trial pause rate of 5% primarily reflects manufacturing scale-up challenges and enhanced regulatory scrutiny following adverse events in competing programs. Most paused trials are expected to resume within 6-9 months after addressing manufacturing or safety concerns.

If you want to grasp this market fast, you can download our latest market pitch deck here

What are the major regulatory hurdles or approvals expected in 2026 that could impact market expansion?

The FDA is expected to make PDUFA decisions on 15-20 gene therapy applications in 2026, including next-generation hemophilia A/B vectors, Parkinson's disease treatments, and expanded CAR-T indications for solid tumors.

Critical regulatory milestones include EMA Marketing Authorization Applications for Krystal Biotech's Vyjuvek (dystrophic epidermolysis bullosa) and PTC Therapeutics' Kebilidi expansion into additional metabolic disorders. These approvals could establish precedents for tissue-targeted gene delivery and broaden reimbursement frameworks across European markets.

Manufacturing regulatory guidance represents a significant hurdle, with both FDA and EMA developing new standards for AAV vector production quality and consistency. Companies must demonstrate batch-to-batch reproducibility within 10% variation for critical quality attributes, requiring substantial manufacturing process improvements for many current platforms.

International harmonization efforts through ICH guidelines are progressing toward unified standards for gene therapy clinical trial design and endpoints. Success in these harmonization discussions could reduce regulatory timelines by 12-18 months for companies pursuing simultaneous global approvals.

The most impactful regulatory development involves potential FDA guidance on combination gene therapies (gene therapy plus small molecule or antibody), which could unlock new therapeutic approaches but may require novel clinical trial designs and safety evaluation frameworks.

Looking for the latest market trends? We break them down in sharp, digestible presentations you can skim or share.

What are the major technological breakthroughs or manufacturing challenges currently influencing scalability?

High-titer AAV production using suspension bioreactor systems has increased vector yields by 3-5x compared to traditional adherent cell culture, addressing the primary manufacturing bottleneck for in vivo gene therapies.

Non-viral delivery systems using lipid nanoparticles and polymer conjugates are emerging as alternatives to viral vectors, offering improved safety profiles and simplified manufacturing processes. These platforms have achieved 40-60% transfection efficiency in liver and muscle tissues, approaching viral vector performance levels.

CRISPR-Cas in vivo editing platforms have demonstrated successful gene correction in large animal models for neurological and muscular disorders, eliminating the need for ex vivo cell manipulation and reducing treatment costs by an estimated 60-70%. Prime editing technologies offer increased precision while reducing off-target effects.

Cell-free vector production systems using synthetic biology approaches are eliminating contamination risks and reducing production timelines from 8-12 weeks to 3-4 weeks. These systems also enable rapid vector modifications for personalized medicine applications.

Manufacturing challenges persist in cold chain logistics for autologous cell therapies, where temperature excursions during transport can result in 15-25% product loss. Companies are investing in advanced cryopreservation technologies and real-time monitoring systems to address these logistical constraints.

Quality control analytics remain complex, with current testing requirements adding 2-3 weeks to manufacturing timelines. Development of rapid release assays using artificial intelligence and machine learning could reduce this bottleneck significantly.

What kinds of business models are proving most profitable or scalable in 2025?

Hybrid R&D/CDMO partnership models demonstrate the highest profitability and scalability, combining in-house vector development with external manufacturing capacity to optimize both innovation and operational efficiency.

Companies like Novartis and Spark Therapeutics have successfully implemented this approach, maintaining internal R&D capabilities for core platform technologies while partnering with specialized CDMOs like Lonza and WuXi Biologics for commercial-scale vector production. This model reduces capital expenditure requirements by 40-60% compared to fully integrated approaches.

Milestone and royalty licensing agreements provide sustainable revenue streams with reduced development risk, exemplified by Crispr Therapeutics' partnership with Vertex for Casgevy, where shared development costs and tiered royalty structures (8-15% on net sales) create win-win scenarios for both platform and development partners.

Platform licensing models are emerging as particularly attractive for investors, where companies develop broadly applicable vector or delivery technologies and license to multiple therapeutic developers. Capsida Biotherapeutics and Encoded Therapeutics represent successful examples of this approach, generating upfront payments of USD 20-50 million plus development milestones.

Contract Development and Manufacturing Organizations (CDMOs) specializing in gene therapy show exceptional scalability, with companies like Catalent and Samsung Biologics reporting 25-35% annual revenue growth by serving multiple clients across different therapeutic areas. This model benefits from economies of scale and reduced regulatory risk.

Curious about how money is made in this sector? Explore the most profitable business models in our sleek decks.

Conclusion

Sources

1. Towards Healthcare - Gene Therapy Market Sizing
2. Hitech Health - Advanced Therapeutic Medicinal Products
3. Abeona Therapeutics - ZEVASKYN FDA Approval
4. FDA - ENCELTO Approval Documentation
5. MarketsandMarkets - Gene Therapy Market Report
6. Precedence Research - Gene Therapy Market
7. Research Nester - Gene Therapy Market Report
8. FDA - Approved Cellular and Gene Therapy Products
9. Statista - Gene Therapy Startup Financing
10. Dealforma - Gene Therapy R&D Partnerships
11. ASGCT - Q1 2025 Clinical Trial Report
12. CVS Caremark - Gene Therapy Pipeline Report
13. EMA - CAT Quarterly Highlights May 2025
14. Scripps Research - ENCELTO Research
15. ASGCT - ENCELTO Cell Therapy News
16. CVS Caremark - Q1 2024 Gene Therapy Report
17. IMARC Group - Gene Therapy Market
18. BioSpace - Cell and Gene Therapy Investment Surge
19. Yahoo Finance - Gene Therapy Market Expectations
20. BioSpace - US Gene Therapy Market Projections
21. Globe Newswire - US Gene Therapy Market Analysis
22. Alliance for Regenerative Medicine - Q1 2025 Sector Snapshot
23. Precedence Research - Cell and Gene Therapy Market