Beacon Therapeutics Reports Positive Phase 2 Results for XLRP AAV Gene Therapy

LONDON and CAMBRIDGE, Mass., September 4, 2025 — Beacon Therapeutics has released new results from two Phase 2 clinical trials, SKYLINE and DAWN, evaluating its lead program, laruparetigene zovaparvovec (laru-zova), for the treatment of X-linked retinitis pigmentosa (XLRP). The results, presented at the EURETINA 2025 Conference in Paris, France, demonstrated that the AAV gene therapy was well-tolerated and showed sustained improvements in visual function.

Laru-zova, an adeno-associated virus (AAV) vector-based gene therapy, was found to be generally well-tolerated by SKYLINE participants through month 36 and DAWN participants at 9 months or beyond. Data from both trials, which utilize AAV technology, showed sustained improvements across several key measures of visual function, including low luminance visual acuity (LLVA) and microperimetry.

“We are pleased to be sharing key data from our DAWN and SKYLINE trials, building on one of the most significant bodies of evidence for an AAV gene therapy in ocular diseases,” said Daniel Chung, D.O., M.A., Chief Medical Officer of Beacon Therapeutics. “These new data updates reinforce our belief in the potential for laru-zova to be a meaningful treatment option for people living with XLRP.”

XLRP is an inherited retinal disease that often leads to blindness and currently has no approved treatments. It is typically caused by mutations in the RPGR gene, affecting approximately 1 in 25,000 males in the U.S., Europe, and Australia. Laru-zova is an AAV-based gene therapy designed to restore the natural function of both rods and cones in the retina by delivering a functional copy of the RPGR ORF15 gene to produce the full-length protein.

Key DAWN and SKYLINE Data Highlights

DAWN Trial:

• Data from this AAV-based trial continued to show early and sustained improvements in low luminance visual acuity (LLVA) and mean sensitivity, representing enhanced visual function in participants evaluated at month 9 or beyond.
• Laru-zova continued to be well-tolerated by all participants evaluated at month 9 or beyond.

SKYLINE Trial:

• Participants who received the high dose of laru-zova showed durable improvements in retinal sensitivity through month 36.
• A greater response rate was observed in the high-dose study eyes compared to the low-dose group or untreated fellow eye.
• Laru-zova, an AAV gene therapy, continued to be well-tolerated by participants in both low- and high-dose groups through month 36.

Beacon is also evaluating LLVA as the primary endpoint in its ongoing pivotal VISTA trial of laru-zova for the treatment of XLRP, with topline data expected in the second half of 2026.

Laru-zova has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA), as well as other key regulatory designations in Europe and the UK. Laru-zova is currently an investigational AAV gene therapy and has not been approved for use.