Twelve-month results from a phase 1/2 trial of sepofarsen for Leber congenital amaurosis type 10 showed a manageable safety profile in adults and children, according to a poster presented at the virtual American Academy of Ophthalmology meeting.
Intravitreal sepofarsen (ProQR Therapeutics) is under study “as a potential injectable therapeutic anti-sense messenger RNA sequence that fixes the LCA10 host messenger RNA splicing defect that leads to LCA10 blindness. Preliminary results demonstrate a manageable safety profile and signs of potential vision improvement,” co-author Allen C.
Uncategorized