TL;DR
Stargardt disease slowly kills the center of the retina, the part of the eye that handles the sharpest vision, usually starting in the teens or twenties, and nothing approved stops it. A company called VeonGen says a one-time gene therapy, VG801, improved the eye-chart scores of all 13 patients it could measure in an early trial, with four of them gaining three full lines. Now, the company says, the FDA has agreed on how the deciding trial should be run. Those early results are the company's own, from a small group with no comparison arm, and they have not been published. The step that matters this week is procedural but real: the trial that could lead to an approval has its blueprint. To see our odds on when a one-time fix for this kind of sight loss reaches patients, and the timeline of breakthroughs that leads there, subscribe.
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