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
The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split