Positive Fabry Data Should Smooth Way For Sangamo’s Partnering Efforts

Just days after Roche stopped shipments of the DMD gene therapy following safety concerns raised by two patient deaths, its EU approval application has hit a block.

The biotech will lay off nearly one-third of its workforce and focus on its cardiovascular gene therapy programs.

Regenxbio and Solid Biosciences believe they have a next-generation approach to AAV-based gene therapies – but must distance themselves from Sarepta’s safety and efficacy problems.

The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.