Lysogene Shifts Sanfilippo Focus To Subgroup Following Pivotal Trial Flop
As Cash Runway Dries Up
Executive Summary
The French firm’s late-stage trial of its gene therapy candidate for Sanfilippo syndrome missed several endpoints but promising data from a cohort of young infants could help pave a way forward - if it can get more cash.
You may also be interested in...
Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance
Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.
Finance Watch: SPAC Deals Stumble, Layoffs Continue As Valuations Struggle To Recover
Aesther Healthcare Acquisition extended the deadline to merge with Ocean Biomedical, while Intrinsic Medicine no longer plans to go public by merging with a SPAC. In restructuring news, Otonomy begins liquidation, Celyad cuts last clinical program with cash running out and Clovis files for bankruptcy.