Insights:Advancing Toward the Clinic

From the beginning, we have focused on getting our program right. We partnered with the world’s leading experts in DMD and gene transfer and built a robust preclinical program to understand our candidate as much as possible before it is administered to any patient with DMD. Our objective has been to design a clinical study that can investigate the safety and efficacy of SGT-001 in the most efficient and responsible manner possible. By putting in this work upfront, we expect to minimize the risk of delays later.

Until our clinical study is finally approved to move forward, we are limited in how much detail we can provide. What I can tell you is that it will be an adaptive Phase I/II study to investigate the safety, tolerability and efficacy of intravenously administered SGT-001. We will start the study at clinical sites in the United States, and we are evaluating additional sites abroad. Importantly, we anticipate that the number of patients in this first study will be limited. We commit to communicating all the relevant details, including key eligibility criteria and clinical site locations, to the DMD community as soon as we can.

We share your sense of urgency. We are 100% committed to getting our program going as quickly as possible. It all comes down to one goal: to bring meaningful treatments to all patients.


Jorge Quiroz M.D., Chief Medical Officer, Solid Biosciences