by: Parent Project Muscular Dystrophy
Solid Biosciences has shared an update with the community on dosing as part of the IGNITE DMD Phase I/II clinical trial, developments for next generation gene therapy pipeline initiative SGT-003 and their collaboration with Ultragenyx.
It was reported that the eighth patient in IGNITE DMD, the fifth patient in the high dose 2E14 vg/kg cohort, that was treated with SGT-001 and experienced a serious adverse event has fully resolved. The patient experienced an SAE that was not unexpected. Following dosing of two patients with second-generation manufacturing process and clinical strategy, Solid conducted an extensive review of all clinical data, resulting in a strengthened risk mitigation plan including new patient management guidance which has been submitted to the FDA.
Activities are underway to advance IGNITE DMD with the next patient dosing anticipated later this year. No new drug-related safety findings have been identified in Patients 1 through 8 in post-dosing periods of 90-days to more than 3 years. Dosed patients continue to be followed in order to collect data to support the potential benefit from dosing SGT-001.
Solid also reports that preclinical activities progressed on the internally developed next-generation Duchenne microdystrophin gene transfer program, SGT-003, including proof of concept research; manufacturing, regulatory and clinical strategies; and development and validation of screening assays to support clinical dosing. The company is targeting an IND submission for SGT-003 in early-2023.
Read Solid’s Community Letter:
Dear Duchenne Community,
This afternoon, we issued a press release providing updates on Solid’s business, including dosing as part of the IGNITE DMD Phase I/II clinical trial, and developments for our next generation gene therapy pipeline initiative SGT-003 and our collaboration with Ultragenyx. We are pleased to have made progress on these key strategic priorities and advance our mission of developing meaningful treatments for patients.
IGNITE DMD Clinical Trial Update
As previously reported, in April 2021, the eighth patient in IGNITE DMD, and fifth patient in the 2E14 vg/kg cohort, was treated with SGT-001. The patient experienced an SAE that was not unexpected and which we’re pleased to share has fully resolved.
Following dosing of two patients with our second-generation manufacturing process and clinical strategy, we conducted an extensive review of all clinical data, resulting in a strengthened risk mitigation plan including new patient management guidance which has been submitted to the FDA. Activities are underway to advance IGNITE DMD with the next patient dosing anticipated later this year.
No new drug-related safety findings have been identified in Patients 1 through 8 in post-dosing periods of 90-days to more than 3 years. We continue to follow dosed patients and collect data to support the potential benefit from dosing SGT-001.
R&D Pipeline Update
Preclinical activities progressed on our internally developed next-generation Duchenne microdystrophin gene transfer program, SGT-003, including proof of concept research; manufacturing, regulatory and clinical strategies; and development and validation of screening assays to support clinical dosing. We are targeting an IND submission for SGT-003 in early-2023.We’ve also made progress in our ongoing collaboration with Ultragenyx.
Upcoming Community Presentations
CureDuchenne FUTURES Conference: October 2021
Action Duchenne Annual Conference: November 2021
The team at Solid Biosciences would like to reiterate our continued dedication to the Duchenne community, and to express our gratitude for your unwavering support. The community has been the greatest driving force behind our dedication and commitment to advance meaningful treatments for patients.
#TogetherWeAreSolid
Sincerely,
Your Solid Biosciences Team
