June 8, 2017
Cambridge, Ma

Advancing Toward the Clinic

We are getting ready to initiate a clinical study for our gene transfer candidate, SGT-001, later this year. We understand that there is a lot of excitement around this new and potentially therapeutic approach, as well as a lot of questions.  On behalf of Solid Biosciences’ Clinical Team, I wanted to take the opportunity to outline what you can expect from us over the next several months.

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

Related news


We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD.


Letter to the Duchenne Community: Update on IGNITE DMD Clinical Trial


Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent.


Solid Biosciences is proud to join the global rare disease community again this year to observe Rare Disease Day


Today we are excited to announce the initiation of our first clinical trial for SGT-001.


Solid Biosciences is pleased to announce additional research grant from our Australia-based partner Save Our Sons and an initial research grant from Ireland-based Join Our Boys.


The Duchenne Research Fund (DRF) provided Solid with a research grant of $600,000 to support its LTBP4 program to combat fibrosis and to support novel biomarker work in Duchenne muscular dystrophy (DMD).


It has been an exciting and emotional time for our Duchenne muscular dystrophy (DMD) community.


Solid was proud to sponsor the 8th Annual Gals For Cal team at the recent Max Performance Title 9 Triathlon at Hopkinton State Park in Massachusetts.


Solid Biosciences joined the Jett Foundation and other members of the local Cambridge, MA community to observe World Duchenne Awareness Day.


More information about Solid?

Please subscribe to receive our latest news