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Solid Biosciences Provides Data Update from SGT-001 Development Program


Solid Biosciences Inc. (Nasdaq: SLDB) today announced biomarker data from two patients dosed in the second cohort of IGNITE DMD, its Phase I/II study of SGT-001. SGT-001 is the company’s gene transfer candidate under investigation for Duchenne muscular dystrophy (Duchenne). The data from these patients show SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase (nNOS) function, providing evidence that SGT-001 has the potential to result in therapeutic benefit for patients with Duchenne. The company also announced that the previously reported serious adverse event experienced by the third patient in the 2E14 vg/kg dose group has fully resolved, and the patient has resumed his normal activities. The company has received the clinical hold letter from the U.S. Food and Drug Administration (FDA) and will continue working internally, and with the FDA and other external experts, to address the clinical hold and determine the path forward.

Solid Biosciences Provides Data Update From SGT-001 Development Program