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Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.

Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.

Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose.

The FDA has placed a clinical hold on Solid Biosciences' IGNITE DMD trial for its Duchenne muscular dystrophy (DMD) gene therapy, SGT-001, after a serious adverse event was reported in a participant. This marks the second clinical hold for the trial, raising concerns over the therapy's safety profile. The event involved complement activation and other severe reactions, prompting a significant drop in Solid Bio's share price.

Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.