At BiopharmIQ, we’ve mapped 377 companies in the global AAV landscape. This work gave us a broad view of the ecosystem, from vector engineering innovators to clinical-stage pioneers and emerging players in new geographies. While big pharma has made major moves into AAV, this list focuses on bio/pharmas and mid-sized innovators that stand out for their technology novelty, recent funding, promising clinical results, or regulatory momentum.

Do you have them on your radar?

👁️ 4D Molecular Therapeutics (US)

Recognition point: Late-stage ophthalmology pipeline

4DMT is advancing 4D-150 for wet AMD, which entered Phase 3 studies in 2025. With a history of strong interim results and a sharpened strategic focus on ophthalmology, 4DMT is one of the few AAV companies with a clear late-stage trajectory in a major indication.

🔬 REGENXBIO (US)

Recognition point: Pivotal trials + pending BLA

REGENXBIO is running a pivotal program for RGX-314 in diabetic retinopathy with AbbVie, while its BLA for RGX-121 in Hunter syndrome is under FDA review. This dual catalyst, pivotal retina and rare-disease regulatory progress, makes it one of the most advanced AAV players worldwide.

💰 Capsida Biotherapeutics (US)

Recognition point: AbbVie opt-in milestone

Capsida’s engineered capsids are designed for better tissue specificity. In 2025, AbbVie exercised its option in their neurodegeneration collaboration, triggering a $40M milestone. That external validation of platform and pipeline cements Capsida’s position as a rising CNS-focused AAV biotech.

🧠 Jaguar Gene Therapy (US)

Recognition point: Niche CNS focuses on autism

Jaguar is targeting SHANK3 haploinsufficiency autism, a rare and high-impact indication. With FDA clearance to begin dosing pediatric patients, Jaguar is carving out a distinctive spot in neurodevelopmental disorders—an area largely untouched by other AAV players.

🧠 uniQure (Netherlands)

Recognition point: Accelerated approval pathway in Huntington’s disease uniQure, one of the earliest pioneers in AAV, continues to innovate with AMT-130 for Huntington’s disease, which received RMAT designation and is on track for a BLA submission in 2026. The potential for accelerated approval makes it a standout in neurodegenerative gene therapy.

💰 Purespring Therapeutics (UK)

Recognition point: Big Series B for kidney-targeted AAV

Purespring closed a $105M Series B to advance its kidney-directed AAV pipeline, including therapies for IgA nephropathy. As one of the few companies applying gene therapy to renal disease, Purespring is addressing a major unmet need and broadening the reach of AAV.

🔬 SparingVision (France)

Recognition point: First-in-human data in retinal dystrophy

SparingVision reported favorable Phase I/II results for its SPVN06 program targeting rod-cone dystrophy. The company also invests in rigorous CMC innovation (new assays for AAV quality), underscoring its strength in both clinical and manufacturing fronts.

👁️ ViGeneron (VeonGen) (Germany)

Recognition point: Ocular focus with rare pediatric designation

Rebranded as VeonGen, the company develops vgAAV and vgRNA REVeRT platforms for inherited retinal diseases. With the FDA Rare Pediatric Disease designation and ongoing clinical progress, VeonGen is solidifying itself as a European leader in AAV-based ocular therapies.

🫀 AaviGen (Germany)

Recognition point: Novel heart-derived AAV vectors

AaviGen is pioneering human heart–derived AAV vectors, a unique approach aimed at improving cardiac gene delivery. This innovative capsid source has the potential to open new therapeutic possibilities in cardiovascular medicine, a field where AAV still struggles with efficient targeting.🚀

🚀 BRL Medicine (China)

Recognition point: First-mover in China’s AAV clinical space

BRL Medicine is advancing CRISPR + AAV therapies and has already reported early clinical data for BRL-101 in hematologic diseases. As one of the first Chinese biotech companies pushing AAV-based treatments into human studies, BRL adds important geographic and scientific diversity to the field.

Why These 10?

• Late-stage momentum: 4DMT, REGENXBIO, uniQure

• Partnerships & funding: Capsida (AbbVie), Purespring (Series B)

• Novel platforms: AaviGen (heart-derived AAV), VeonGen (vgAAV, vgRNA)

• Unique niches: Jaguar (autism), BRL (China-first mover), SparingVision (rod-cone dystrophy)

Together, these companies highlight how AAV innovation is evolving beyond big pharma and beyond the US, with global biotech leaders pushing into new disease areas, new capsid technologies, and new geographies.

Closing Thoughts

The AAV field has grown rapidly, and with 377 companies mapped in BiopharmIQ’s landscape, it’s clear the space is no longer dominated by just a handful of players. The companies highlighted here represent a diverse mix of late-stage clinical programs, breakthrough capsid engineering, novel therapeutic niches, and geographic expansion beyond the US.

While challenges remain, such as manufacturing scalability, safety, and regulatory alignment, the momentum across these innovators signals that AAV continues to be one of the most active and transformative areas in biotech. At BiopharmIQ, we’ll keep tracking this evolving landscape, surfacing the next wave of companies and data points that shape the future of gene therapy.

👉 Want to explore the broader landscape? Explore our free list of 377 AAV companies mapped by BiopharmIQ, covering innovators, scale-ups, and global leaders.

How can you get the most out of this list?

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You’re also welcome to book a demo with our team, who can walk you through the platform and show you how to tailor insights to your specific business development needs.

Article History:

9/26/25 MD, DG

Not legal, investing, or tax advice.