Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation AAV micro-dystrophin gene therapy candidate, SGT-003, for the treatment of Duchenne.

Based on early study results, the trial has expanded its enrollment to a total of 43 participants and broadened the age range to include individuals from 4 to less than 12 years old. Additionally, the evaluation of functional endpoints has been adjusted from 12 months to 18 months, allowing for a better assessment of SGT-003’s potential to impact disease progression. They also announced the addition of three new clinical trial sites. Data from the first three patients will be shared in early 2025.

Read Solid’s community letter here.

PPMD is excited to be hosting a webinar with Solid on Tuesday, November 12th at 1:00 PM ET, to discuss these updates. Register here and submit questions in advance here.