The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor
Almost against the odds, Sarepta’s gene therapy for muscle-wasting disease Duchenne muscular dystrophy (DMD) has been given accelerated approval by the FDA, albeit in a fa
UK life sciences investor Syncona is bankrolling a new gene therapy company, Beacon Therapeutics, that emerged this morning with a pipeline headed by a therapy acquired fr
Astellas’ gene therapy for rare disease X-linked myotubular myopathy (XLMTM) has been hampered by safety concerns, so its decision to license an alternative from start-up
Cell and gene therapy specialist ElevateBio has taken the total fundraising since it launched five years ago to around $1.25 billion, with its latest $401 million round th