MedPage Today September 13, 2024
— “Mind-boggling” effects in small group with Leber congenital amaurosis 1, expert says
A subretinal gene therapy was well tolerated 1 year after treatment, with no drug-related serious adverse events, in a small group of patients with Leber congenital amaurosis 1 (LCA1), a rare inherited retinal disorder that causes blindness in early childhood, a phase I/II study suggested.
Among the 15 adults and children with LCA1, which is caused by mutations in GUCY2D, 68 treatment-emergent adverse events (TEAEs) were observed with three different doses of unilateral subretinal injections of ATSN-101, 56 of which were related to the procedure, reported Paul Yang, MD, of the Casey Eye Institute at Oregon Health & Science University in Portland, and colleagues in The Lancet.
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