Beacon Therapeutics announced positive topline results for its registration-enabling VISTA trial evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa.
VISTA is a randomized, controlled trial designed to evaluate the efficacy, safety and tolerability of laru-zova in male patients with XLRP caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The trial met its FDA-endorsed primary endpoint, demonstrating statistically significant low luminance visual acuity (LLVA) responder rates at month 12 compared to the untreated control group. A statistically significant proportion of trial participants in both the high and low dose groups achieved an improvement of ≥15 letters in LLVA as compared to no responders in the untreated control group.
Based upon these results, Beacon says it will initiate pre-submission discussions with global regulatory authorities.
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.
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