Penn spinout Ascidian Therapeutics, a company using technology developed by Jean Bennett, MD, PhD, the F.M. Kirby Emeritus Professor of Ophthalmology at Penn Medicine, has partnered with Swiss multinational healthcare company Roche to develop RNA therapeutics for neurological diseases. The collaboration combines Ascidian’s RNA exon editing technology with Roche’s expertise in CNS delivery to create novel treatments for hard-to-treat neurological conditions.
As part of the agreement Ascidian will receive a $42 million upfront payment and up to $1.8 billion in milestone payments, plus royalties. Ascidian Therapeutics also 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. 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. Read more here and here.