Preliminary results from two studies of gene therapies for achromatopsia have shown them to be safe while improving some patients’ photosensitivity and two-colour dark light-adapted perimetry.
Associate Professor Mark Pennesi, from Oregon Health and Science University’s Casey Eye Institute, said the studies of Applied Genetic Technologies Corporation’s AGTC-401 and AGTC-402 drugs demonstrated visual improvements in three of the 16 patients in the CNGA3 gene study and seven of the 16 patients in the CNGB3 gene study in the higher-dose groups. CNGB3 and CNGA3 are genes whose mutations most frequently cause achromatopsia. The studies are ongoing at Moorfields Eye Hospital in the UK and the Kellogg Eye Center in the US; the drugs being delivered by subretinal injection, with four dosing groups in each study.
