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Preliminary Data and Intention to Dose Escalate in IGNITE DMD


Today we announced our intention to move to a higher dose of SGT-001 in IGNITE DMD, our Phase I/II clinical trial evaluating SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. This decision is based on our initial look at the muscle biopsies from the first three boys who received the starting dose of SGT-001 in the clinical trial. You can find the press release here. We wanted to take a moment to share with the Duchenne community what these findings mean and how we are moving forward.

First, we want to let you know that all three boys who received SGT-001 continue to do well, and we are following them per our study protocol. As for the biopsy data, we were able to see microdystrophin expression across some measures, although it was low. These initial results support moving forward with evaluation of higher doses in the clinical trial.

Preliminary Data And Intention To Dose Escalate In IGNITE DMD