Two-year data with eyeDNA Therapeutics’ gene therapy for retinitis pigmentosa (RP), an inherited form of blindness, has prompted the biotech to seek discussions with regul
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-editing 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.
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year
