Which gene therapy companies got funding?

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Gene therapy funding experienced a dramatic rebound in 2025 after a challenging 2024.

Five companies alone raised $534.4 million in the first four months of 2025, compared to $773 million across 20 rounds for the entire 2024. This shift reveals which technologies and approaches are capturing investor attention and where the smart money is flowing in advanced therapeutics.

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Which gene therapy companies received the most funding in 2024 and 2025?

Tune Therapeutics leads 2025 funding with a $175 million Series B round for their epigenome editing platform targeting oncology applications.

In 2024, Beacon Therapeutics captured the largest round at $170 million Series B, backed by Forbion, Syncona, Oxford Science Enterprises, TCGX, and Advent Life Sciences. Their AAV-based gene therapy laru-zova targets X-linked retinitis pigmentosa and dry age-related macular degeneration, representing a clear path to proof-of-concept in ophthalmology.

Atsena Therapeutics secured $150 million in Series C funding in 2025, though specific technology details remain undisclosed. SpliceBio from Spain raised $135 million Series B co-led by EQT Life Sciences and Sanofi Ventures for their dual-AAV protein-splicing platform addressing Stargardt disease.

Purespring Therapeutics raised $105 million Series B in 2024 from Sofinnova Partners, Gilde Healthcare, Forbion, British Patient Capital, and Syncona. Their AAV platform specifically targets podocyte delivery for IgA nephropathy and other glomerular diseases.

The funding concentration in fewer, larger rounds indicates investors are backing proven technologies with regulatory clarity rather than spreading capital across numerous early-stage ventures.

How much total capital flowed into gene therapy startups in each year?

Gene therapy startup funding dropped 45% in 2024 to $773 million across 20 rounds, down from $1.4 billion in 2023.

However, 2025 shows remarkable recovery momentum with $534.4 million raised by just five companies in the first four months. At this pace, 2025 could exceed 2024's total by mid-year, suggesting investor confidence has returned to the sector.

The 2024 decline reflected broader biotech market corrections and investor caution around early-stage gene therapy ventures. Investors became more selective, focusing on companies with advanced clinical data and clear regulatory pathways rather than platform plays without proven efficacy.

The 2025 rebound concentrates capital in mature technologies like epigenome editing and enhanced AAV delivery systems. This shift indicates the market has moved beyond proof-of-concept funding toward supporting technologies ready for clinical validation and commercial development.

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Which investors and pharmaceutical companies are backing these gene therapy ventures?

European venture capital firms dominate gene therapy funding, with Forbion, Syncona, and Sofinnova Partners leading multiple rounds.

Forbion participated in both Beacon Therapeutics ($170M) and Purespring Therapeutics ($105M) rounds in 2024, indicating strong conviction in AAV-based platforms. Syncona, the UK's specialist life sciences investor, co-led Beacon's round and invested in Purespring, focusing on companies with clear clinical development paths.

Pharmaceutical corporations engage primarily through venture arms rather than direct strategic investments. Sanofi Ventures co-led SpliceBio's $135 million round, while Roche Venture Fund and Novartis Venture Fund maintain active portfolios in the space. This approach allows pharma companies to evaluate technologies before committing to larger partnerships.

Strategic licensing deals reached significant scale in 2024, with Astellas providing upfront payments of $10-30 million plus milestone commitments up to $1.8 billion for platforms from AviadoBio and Sangamo. These deals offer alternative capital sources beyond traditional venture rounds.

US-based New Enterprise Associates represents the major American presence in European-dominated funding syndicates, reflecting the global nature of gene therapy investment.

What core technologies and breakthroughs are attracting the most investment?

Epigenome editing platforms captured the largest funding rounds in 2025, with Tune Therapeutics ($175M) and Epicrispr Biotechnologies ($68M) leading this emerging category.

AAV-based delivery systems remain the dominant funded technology, but with enhanced specificity features. Beacon Therapeutics' laru-zova demonstrates improved retinal targeting, while Purespring's platform specifically delivers genes to podocytes in kidney glomeruli. Sangamo's STAC-BBB system enables blood-brain barrier crossing, expanding AAV applications to neurological conditions.

In vivo CAR-T represents a breakthrough approach attracting $67 million for Interius BioTherapeutics. Their platform delivers CAR transgenes directly to patients via AAV vectors, eliminating complex ex vivo cell manufacturing processes that plague traditional CAR-T therapies.

Split-gene delivery systems like SpliceBio's dual-AAV platform solve payload size limitations that restrict single-vector approaches. This technology enables delivery of larger therapeutic proteins by splitting them across two vectors that reassemble in target cells.

CRISPR-based genome editing continues attracting capital through Arbor Biotechnologies' $73.9 million Series C, though specific technological advantages over existing platforms remain undisclosed.

Which diseases and therapeutic areas are receiving the most funding?

Ophthalmology dominates funded indications, capturing $305 million across Beacon Therapeutics ($170M) and SpliceBio ($135M) for inherited retinal disorders.

Oncology applications secured significant investment through Tune Therapeutics' $175 million round for solid tumor targets and Interius BioTherapeutics' $67 million for CD20-positive B-cell malignancies. The epigenome editing approach offers potential advantages over traditional gene replacement by modulating existing gene expression rather than introducing new genetic material.

Rare kidney diseases attracted $105 million through Purespring Therapeutics' focus on IgA nephropathy and other glomerular conditions. This represents a relatively underserved therapeutic area with clear regulatory pathways through orphan drug designations.

Cardiovascular disease entered the funded landscape through XyloCor Therapeutics' $67.5 million Series B, though specific indications remain undisclosed. This area offers large patient populations but faces higher regulatory hurdles than rare disease applications.

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Where are these funded gene therapy companies located geographically?

North American companies captured 80% of disclosed funding, with seven of nine major rounds going to US-based ventures.

European presence centers on the UK through Purespring Therapeutics ($105M) and Spain via SpliceBio ($135M). The UK benefits from established life sciences infrastructure and Syncona's active investment strategy, while Spain's emergence reflects growing European gene therapy expertise outside traditional hubs.

Boston and San Francisco Bay Area dominate US gene therapy funding geography, though specific company locations aren't disclosed for all ventures. These regions offer access to specialized talent, clinical research infrastructure, and proximity to major investors and pharmaceutical partners.

The geographic concentration reflects practical considerations around clinical trial execution, regulatory expertise, and manufacturing capabilities. Gene therapy development requires specialized infrastructure that exists primarily in established biotech clusters.

Asia-Pacific remains notably absent from major gene therapy funding rounds, despite significant activity in cell therapy and traditional biotech sectors in this region.

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What deal terms and partnership structures are companies using?

Standard venture equity financing dominates gene therapy funding, with Series B and C rounds representing the majority of disclosed capital.

Strategic licensing deals offer alternative structures with $10-30 million upfront payments and milestone commitments reaching $1.8 billion. Astellas' partnerships with AviadoBio and Sangamo include worldwide licensing rights and tiered royalties on net sales, providing significant capital without equity dilution.

Equity stakes in venture rounds typically follow standard VC terms for early- to mid-stage companies, though specific percentages and liquidation preferences aren't publicly disclosed. The large round sizes suggest investors are taking substantial positions to justify the capital commitments.

Exclusivity provisions appear standard in pharma partnerships, with worldwide rights granted in most cases. This global scope reflects the international nature of gene therapy development and the need for partners to recoup substantial R&D investments across major markets.

Milestone structures tie payments to clinical and regulatory achievements, aligning investor and company interests around development progress rather than purely financial metrics.

Which companies are positioned to raise significant funding in 2026?

Companies with FDA accelerated approval pathways represent the most likely candidates for major 2026 funding rounds, including uniQure, Sangamo, Rocket Pharmaceuticals, and Precigen.

Six gene therapies targeting rare genetic diseases aim for 2025-2026 FDA submissions under accelerated approval designations, creating capital needs for commercial preparation and manufacturing scale-up. These companies will require $100-300 million rounds to support regulatory submissions and market launch activities.

Platform companies with validated technologies but undisclosed indications may emerge as funding candidates if they reveal compelling clinical data or strategic partnerships. The success of epigenome editing rounds in 2025 suggests similar technologies could attract large investments.

Companies entering pre-registration phases increased 83% in Q4 2024 versus Q3 2024, indicating a growing pipeline of late-stage ventures approaching commercial readiness. This cohort will need substantial capital for manufacturing, regulatory affairs, and market access preparation.

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What funding trends are emerging in gene therapy approaches?

Platform diversification drives the most significant funding trend, with epigenome editing capturing $243 million across two companies in 2025 compared to zero funding in 2024.

Enhanced AAV delivery systems attract premium valuations when demonstrating tissue-specific targeting capabilities. Beacon's retinal-targeting platform and Purespring's podocyte-specific vectors command higher multiples than generic AAV approaches due to reduced off-target effects and improved safety profiles.

In vivo cell engineering represents an emerging trend through Interius BioTherapeutics' direct CAR-T delivery approach. This eliminates complex manufacturing processes that create cost and scalability challenges for traditional ex vivo CAR-T therapies.

Non-AAV vector systems gain traction through technologies addressing payload limitations and immunogenicity concerns. Split-gene delivery platforms like SpliceBio's dual-AAV system overcome size constraints while maintaining AAV's favorable safety profile.

Strategic pharma partnerships increasingly focus on platform licensing rather than individual asset acquisitions, reflecting interest in broader technology access rather than single-indication bets.

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How do 2025 funding patterns differ from 2024 trends?

2025 shows dramatic consolidation toward fewer, larger rounds compared to 2024's broader distribution across 20 smaller ventures.

Average round size increased from $38.7 million in 2024 to $106.9 million for disclosed 2025 rounds, indicating investor preference for backing advanced-stage companies with proven technologies rather than spreading capital across early-stage platforms.

Technology focus shifted toward epigenome editing and enhanced delivery systems in 2025, while 2024 funding supported a broader range of approaches including first-generation AAV platforms and basic CRISPR applications.

Geographic concentration intensified, with 2025 rounds concentrated in established biotech hubs, while 2024 included more diverse geographic distribution across secondary markets.

Investor syndicate composition changed, with larger specialist funds leading 2025 rounds compared to 2024's broader participation from generalist VCs and smaller biotechnology-focused investors.

What market signals indicate continued gene therapy investment growth?

Regulatory momentum provides the strongest positive signal, with six gene therapies entering accelerated approval pathways targeting 2025-2026 FDA submissions.

The 83% increase in pre-registration gene therapies from Q3 to Q4 2024 indicates a maturing pipeline approaching commercial validation. This regulatory progress reduces investment risk and attracts capital from later-stage investors previously hesitant about gene therapy timelines.

Strategic pharma engagement through licensing deals worth up to $1.8 billion demonstrates corporate confidence in gene therapy commercial potential. These partnerships provide validation and de-risk future venture rounds by establishing potential exit pathways.

Manufacturing infrastructure development enables scalable production, addressing a key investor concern about gene therapy commercialization. Platform companies with validated manufacturing approaches command premium valuations due to reduced execution risk.

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Conclusion

Sources

1. DealForma - Gene Therapy R&D Partnerships, M&A and Ventures in 2024
2. ASGCT-Citeline Q1 2025 Report
3. BioPharma Dive - SpliceBio lands $135M
4. Fierce Biotech - Fundraising Tracker
5. ASGCT-Citeline Q4 2024 Report
6. Alliance for Regenerative Medicine Q1 2025 Sector Snapshot
7. Genetic Engineering & Biotechnology News - Top 10 Best-Selling Gene Therapies