Penn spinout Ascidian Therapeutics has achieved a significant milestone with the FDA’s acceptance of their Investigational New Drug (IND) application for ACDN-01, a treatment targeting the rare genetic eye condition Stargardt disease and other ABCA4 retinopathies based on technology developed by Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology at Penn Medicine.
The therapy, which is the first of its kind to enter clinical development, has also received Fast Track and Rare Pediatric Disease Designations from the FDA.
ACDN-01’s unique approach allows for precise post-transcriptional editing of genes to produce full-length, functional proteins, with the ultimate goal of providing a persistent therapeutic effect with a single dose. Ascidian anticipates initiating patient enrollment in its Phase 1/2 study for ACDN-01 in the first half of 2024. Read more here.