Solid Updates Strategy to Develop SGT-001 & SGT-003 for Duchenne

Solid Bio

Today, Solid Biosciences announced a change to its corporate strategy to better align resources with goals to bring SGT-001 and SGT-003 to patients.

Key Takeaways:

  • Enrollment in IGNITE DMD, its Phase 1/2 gene therapy trial for SGT-001 is complete 
    • In March 2022, Solid released data showing individuals demonstrated a sustained benefit compared to natural history out to 2 years
    • These individuals will now be monitored for 5 years post-treatment
  • SGT-001 will advance to late-stage trials with a new manufacturing process in early 2023, transitioning to commercially scaled transient transfection
  • SGT-003 is moving to IND submission, also with new manufacturing, in early 2023
    • SGT-003 packages the same microdystrophin in a novel capsid with better muscle tropism
    • Newly released preclinical data from a non-human primate study indicated a 2x increase in muscle targeting with decreased liver uptake, and a more than 10x increase in reporter gene expression in muscle and heart compared to AAV9

For more information, please read Solid’s letter to the community here.

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