Beacon shares positive 6-month data on rare eye disease gene therapy

Beacon Therapeutics announced six-month interim safety and efficacy results from the phase 2 DAWN trial of the company’s lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP).

DAWN is an open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting the RPGR protein. Laru-zova was generally well-tolerated by all DAWN participants evaluated at six months or beyond and initial data showed promising improvements in visual function across several key measures.

Data demonstrated early improvements in low luminance visual acuity, a measure of visual function, with a greater number of two and three line improvements in the study eyes compared to previously treated fellow eyes in participants evaluated at month six or beyond. Data also showed early and sustained improvements in mean sensitivity in study eyes, as observed by microperimetry, indicating enhanced visual function in participants evaluated at month six or beyond.

XLRP is a severe form of retinitis pigmentosa, a group of inherited retinal diseases that that cause serious vision impairment and blindness. It is often caused by mutations to the RPGR gene. Laru-zova is a subretinal gene therapy designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene using a well-established vector with a proprietary capsid designed for high transduction of photoreceptors, and a codon-optimized gene to produce the full-length protein.

Beacon will continue to enroll patients for its pivotal phase 2/3 VISTA trial of laru-zova for patients with XLRP.

Earlier this week, J&J revealed that its experimental AAV gene therapy, bota-vec, failed to meet the primary endpoint of improving vision-guided mobility In a phase 3 trial for XLRP. J&J had purchased the full rights to bota-vec from MeiraGTx in 2023.