Paper published in Nature Medicine!

Together with Beam Therapeutics, we have developed a new gene editing therapy that may help treat Stargardt disease—the most common form of inherited macular degeneration. Published in Nature Medicine on 8 January 2025, this method uses “base editing” delivered by a special viral vector to precisely repair the genetic defect behind the disease.

Stargardt disease affects about 1 in 6,500 people and often leads to legal blindness. In lab tests using human tissues and animal models, we were able to correct up to 75% of cone cells and 87% of retinal pigment epithelial cells, with no signs of unwanted edits. These exciting results could pave the way for clinical trials—and potentially offer hope for thousands affected by Stargardt disease and other inherited retinal conditions.

Please see the paper here: https://www.nature.com/articles/s41591-024-03422-8

Watch the video: https://lnkd.in/d326UABB

Press release here: https://lnkd.in/ebhf5xrt