Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results
Pfizer’s gene therapy group is back up at bat with a fresh update of data from its Phase Ib study for a gene therapy intended as a once-and-done treatment for Duchenne muscular dystrophy. And they had some good news, and some bad news, to report.
The good news was primarily in the dystrophin expression they tracked among the now 9 evaluable patients in their first clinical foray for PF-06939926.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 119,800+ biopharma pros reading Endpoints daily — and it's free.