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
A gene therapy for heart failure being developed by Bayer’s AskBio unit has been granted fast-track status by the FDA, shortly after it showed preliminary signs of efficac
An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer
Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-
Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr