Roche’s gene therapy unit Spark has added to its pipeline in inherited retinal diseases (IRDs) with a $216 million alliance with Spanish biotech SpliceBio.
Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial.
Amryt Pharma has been handed a major blow by the FDA, after the US regulator rejected its marketing application for Oleogel-S10, its drug for rare and debilitating skin disease epidermolysi
Shares in Applied Therapeutics slumped today after it said it would have to delay a US filing for its lead drug programme AT-007, following an FDA request for more data.
The FDA has approved Akebia Therapeutics’ vadadustat as a treatment for anaemia caused by chronic kidney disease (CKD) in dialysis patients, becoming the first rival to GS