A gene therapy gives boys their night vision back
X-linked retinitis pigmentosa mostly strikes boys and young men, and it takes their night vision first, often years before it takes the rest. In the VISTA trial, 85 males between 12 and 48 received a single injection of Beacon Therapeutics' gene therapy, laru-zova, into the retina.
A year later, 31 percent of the high-dose group and 24 percent of the low-dose group could read 15 or more additional letters on a low-light vision test. In the untreated control group, the number who hit that mark was zero 224. It's the first pivotal trial win this disease has ever had; a Biogen-acquired therapy failed in 2021, and MeiraGTx missed its primary goal in its own pivotal study. Beacon plans to start filing for approval by the end of the year 224.