Regenxbio, Solid report data with Duchenne gene therapies

This week saw both Regenxbio and Solid Bio report new data with their gene therapy candidates for Duchenne muscular dystrophy (DMD), vying to join Sarepta’s Elevidys on the market.

Regenxbio gave an update on the phase 2/3 AFFINITY DUCHENNE trial of its RGX-202, reporting interim results that showed it was well tolerated, achieved robust expression of microdystrophin to replace the mutated dystrophin protein in DMD, and led to early functional improvements in ambulatory DMD patients aged one and over.

The data, from seven subjects in the study treated with the target dose of RGX-202,…