Roche has partnered with Ascidian Therapeutics, a startup focused on editing RNA to address mutations driving diseases. Ascidian’s technology edits RNA exons to potentially solve issues in devastating diseases. Unlike traditional gene editing, Ascidian’s approach focuses on protein-coding segments of RNA to replace mutated exons with functional ones. This avoids immune responses and off-target edits. Ascidian’s lead program, ACDN-01, targets Stargardt disease by editing the ABCA4 gene. Roche’s interest in RNA therapies for neurological disorders has led to partnerships with other startups as well. The alliance with Ascidian involves collaboration in discovery and preclinical work, with potential milestone payments of $1.8 billion.
Source link
Roche Adds Another Neuro Alliance, Inking R&D Pact With RNA-Editing Startup Ascidian
