Topic: Duchenne muscular dystrophy (DMD)
Preliminary signs of improved muscle function have been seen in Sarepta's Duchenne muscular dystrophy gene therapy trial.
The early-stage alliance comes a week after Pfizer halted development of domagrozumab in DMD because of lackluster midphase data.
Using CRISPR to correct a DMD gene mutation restored up to 92% of muscle function in dogs.
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
A trial of Sarepta’s gene therapy for muscular dystrophy was placed on clinical hold by the FDA but could be upgraded to a pivotal study as a result.
Two AI firms, Insilico Medicine and A2A Pharmaceuticals, launched a joint company aimed at Duchenne muscular dystrophy and other rare orphan diseases.
Despite posting promising early data, Summit Therapeutics' Duchenne muscular dystrophy drug missed its primary and secondary endpoints in a phase 2 trial.
The data, taken from three patients, showed a 38% increase in a muscle protein that boys with DMD usually lack.
The FDA lifted the second of two clinical holds on Solid Bio’s Duchenne muscular dystrophy trial, following safety and manufacturing concerns.
At BIO, we chatted with Doug Ingram about Sarepta's pipeline, its latest hires and how the healthcare landscape must change.