PPMD is pleased to learn that Solid Biosciences has received clearance by the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application for SGT-003, the company’s next generation microdystrophin gene therapy for the treatment of Duchenne. SGT-003 combines Solid’s differentiated microdystrophin transgene with a new, novel capsid designed to increase the amount of drug delivered to muscle compared to first generation capsids.

Solid has indicated that the company will now begin working with urgency to enroll patients in INSPIRE DUCHENNE, a Phase 1/2 multicenter clinical trial to primarily evaluate the safety and tolerability of SGT-003. The study will include two separate cohorts based on age, beginning with patients 4 and 5 years old. All participants will receive a one-time intravenous infusion of SGT-003 at a single dose level. Long-term safety and efficacy will be evaluated for a total of five years following treatment.

PPMD will be hosting a community webinar with Solid in the coming weeks, during which members of the Solid team will share more information about the clinical study, including sites and eligibility criteria. Additional details about this webinar will be available soon.

Read Solid’s community letter and press release here.