gene therapy

News

The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.

Oncology

At JPM2026, Astellas' chief strategy officer Adam Pearson walked pharmaphorum through the company’s four-pillared R&D strategy.

News

Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.

News

enGene is hoping to file its gene for a form of bladder cancer before the end of 2026, based on phase 2 data revealing a 62% complete response rate.

News

uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.

News

Rocket Pharma has withdrawn a filing in the US for its experimental gene therapy for Fanconi anaemia, but may consider partnering the programme.