Insights:Letter to the Duchenne Community: IGNITE DMD Phase I/II Clinical Trial and Business Update

Dear Duchenne Community,

This morning we issued a press release providing a full-year 2022 business update. Among the highlights, Solid completed the acquisition of AavantiBio, creating a precision genetic medicines company with a focus on cardiac and neuromuscular diseases, including Duchenne.

IGNITE DMD Phase I/II Clinical Trial Update

On March 19, Solid presented one-year post-treatment data relating to safety, efficacy and microdystrophin expression in muscle biopsies for patients enrolled in the IGNITE DMD Phase I/II clinical trial evaluating SGT-001 in a poster presentation at the Muscular Dystrophy Association Clinical and Scientific Conference. Other than the required ongoing observation of patients in IGNITE DMD and the completion of already in process preclinical experiments, the SGT-001 program has concluded.

Also at the MDA conference, Solid presented data previously reported at World Muscle Society Congress in October 2022. These data demonstrate patients administered SGT-001, on average, showed improved spontaneous walking velocity at one-year post-treatment as measured by the SV95C assessment through the ActiMyo wearable device.

Additional updates on the IGNITE DMD study are expected to be provided following completion of the 5-year follow up timepoint for all subjects. Per study protocol, all patients dosed will continue with their scheduled follow-up visits five years post-infusion. We plan to make information related to the full study, as well as individual patient data, available to all study participants.

We would like to take this opportunity to express our deep gratitude for the patients who have participated in IGNITE DMD, and for all patients and families who choose to participate in clinical trials. With your bravery and willingness to participate in clinical trials, we are able to advance important science in our pursuit to develop meaningful therapies for Duchenne.


The Company is on track for an anticipated Investigational New Drug (IND) submission for SGT-003, a next generation gene therapy for patients with Duchenne, in the second half of 2023 and, pending IND acceptance, first patient dosing in late-2023.

Upcoming Community Presentations

  • CureDuchenne FUTURES National Conference: April 20-23, 2023
  • Parent Project Muscular Dystrophy Annual Conference: June 30-July 2, 2023

The Solid Biosciences team remains committed to the Duchenne community and to our purpose of improving the lives of patients living with Duchenne and other rare neuromuscular and cardiac diseases.


Your Solid Biosciences Team