Biotechnology company Applied Genetic Technologies Corporation (AGTC) recently released data evaluating an experimental recombinant AAV vector gene delivery in patients with Leber congenital amaurosis (LCA) or severe early-childhood-onset retinal degeneration (SECORD).
Both LCA and SECORD are related retinal diseases that are caused by mutations in the RPE65 gene, causing severe loss of vision in infancy. The study reportedly enrolled four children and eight adults with LCA or SECORD who were given subretinal injections of a recombinant AAV expressing RPE65 (rAAV2-CB-hRPE65) at one of two dose levels in the poorer seeing eye. According to AGTC, best-corrected visual acuity (BCVA) increased in the treated eye of five subjects, static perimetry hill of vision measurements for the central 30° of the visual field increased in six subjects, total visual field hill of vision measurements increased in five subjects, and kinetic visual field area improved in three subjects.
Results from the study were recently published online in the journal Ophthalmology and will appear in the journal's April print issue.
Click here to read the full press release.
Source: Applied Genetic Technologies Corporation