News Breakthrough status for BioMarin ratchets up haemophilia A c... The FDA has granted Breakthrough Therapy Designation to BioMarin’s gene therapy for haemophilia A, helping to intensify competition in the field.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends