Beacon Therapeutics Lands $75M to Push Gene Therapy for Blindness

LONDON and CAMBRIDGE, Mass. – January 08, 2026 – Beacon Therapeutics has secured over $75 million in an oversubscribed Series C financing round, a major capital infusion intended to propel its lead gene therapy candidate for a rare blinding disease toward commercialization. The round, led by new investor Life Sciences at Goldman Sachs Alternatives, marks a significant vote of confidence in the company's mission to combat severe ocular diseases.

The financing also saw participation from the Retinal Degeneration Fund (RD Fund), the venture arm of the patient-focused Foundation Fighting Blindness, alongside robust support from existing investors Syncona Limited, Forbion, Oxford Science Enterprises, and Advent Life Sciences. The funds are earmarked to advance laru-zova, a potential first-in-class treatment for X-linked retinitis pigmentosa (XLRP), and to accelerate the development of other promising therapies in the company's pipeline.

“This significant fundraising, led by new blue-chip investor Life Sciences at Goldman Sachs Alternatives, validates our strategy to save and restore sight for people living with rare and prevalent ocular diseases,” said Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics. “With pivotal laru-zova data expected in the second half of 2026, these funds will accelerate our commercial preparations for this potentially life-changing product, as well as advance and expand our pipeline.”

A Beacon of Hope for a Blinding Disease

X-linked retinitis pigmentosa is a devastating inherited retinal disease that causes progressive and irreversible vision loss, primarily affecting males. It is one of the most common causes of blindness in young men, with symptoms often starting in childhood with night blindness. The disease relentlessly constricts the visual field, creating a debilitating “tunnel vision” effect that ultimately leads to legal blindness, often by the time a patient reaches their 40s. In the United States alone, an estimated 11,000 people live with the condition.

Caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene in up to 90% of cases, the genetic defect disrupts the function of the retina's light-sensing photoreceptor cells. To date, there are no approved treatments that can halt or reverse the disease's progression, leaving patients with only supportive care options as their vision fades.

Beacon’s lead candidate, laru-zova, is designed to address this profound unmet need directly. It is an adeno-associated virus (AAV) vector-based gene therapy administered via a one-time subretinal injection. The therapy works by delivering a stable, functional copy of the RPGR gene to retinal cells, aiming to restore the natural function of both rod and cone photoreceptors and thereby preserve or even improve vision.

The Road to Commercialization

The new funding arrives at a critical juncture for laru-zova. The therapy is currently being evaluated in the pivotal VISTA trial, a global, randomized Phase 2/3 study. Beacon announced the completion of enrollment for the 85-participant trial in July 2025, setting the stage for a crucial data readout in the second half of 2026. This data will form the bedrock of planned regulatory submissions in the United States and Europe.

The VISTA trial is supported by a robust body of evidence from several other clinical studies. The SKYLINE and HORIZON trials have already demonstrated durable improvements in retinal sensitivity and a favorable safety profile over 36 months. Furthermore, interim data from the ongoing DAWN study, presented in late 2025, showed sustained functional vision improvements in patients, reinforcing confidence in the therapy's potential.

Laru-zova's promise has already been recognized by global regulators. It has received multiple expedited pathway designations, including Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Priority Medicines (PRIME) from the European Medicines Agency (EMA), and the Innovative Licensing and Access Pathway (ILAP) in the UK, all of which are designed to accelerate the development and review of transformative treatments.

'Blue-Chip' Confidence in a Booming Market

The leadership of a heavyweight investor like Goldman Sachs Alternatives signals strong institutional belief not only in Beacon's approach but also in the broader ocular gene therapy market. This sector is experiencing explosive growth, with market projections estimating it will expand from approximately $1.3 billion in 2024 to over $7.3 billion by 2033. This investment stands out, as it comes at a time when venture funding for the broader gene therapy space has seen a decline, underscoring the perceived potential of targeting ocular diseases.

As part of the financing, Colin Walsh, PhD, a Managing Director at Life Sciences at Goldman Sachs Alternatives, will join Beacon's Board of Directors. “We are excited to work with Beacon Therapeutics at this critical time to support the development of laru-zova as a potential game-changing ocular gene therapy,” said Dr. Walsh. “Beacon has deep experience in gene therapy and its application in ocular disease. With laru-zova's advancement through pivotal trials and the Company's expanding pipeline, the Company is setting the roadmap for how to build successful gene therapies and bring transformative treatments to patients with X-linked retinitis pigmentosa (XLRP).”

The participation of the RD Fund adds another layer of validation, bringing a mission-driven, patient-centric perspective. “RP affects over 100,000 patients in the US, with 14% of those estimated to have XLRP. With no available treatment options, halting XLRP progression and restoring patients' vision would represent a life-changing breakthrough,” commented Rusty Kelley, PhD, MBA, Managing Director of the RD Fund.

Beyond XLRP: Building a Pipeline for Vision

While bringing laru-zova to patients remains the company's top priority, the Series C funds will also be used to build a durable franchise in ophthalmology. Beacon is actively developing other pipeline candidates targeting major causes of vision loss, including geographic atrophy—an advanced form of age-related macular degeneration—and another inherited cone-rod dystrophy. This strategy positions the company as a potential leader in the fight against a range of blinding conditions, not just a single disease.

As the company moves forward with its expanded resources, the biotechnology and investment communities will be watching closely. Beacon Therapeutics is scheduled to present its strategy and progress at the 44th Annual JP Morgan Healthcare Conference in San Francisco next week.