Last week we had the privilege to meet in person with the team at their headquarter in Boston. They presented their very robust preclinical trial data suggesting that SGT-001, has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage. Now they are announcing the initiating of SGT-001, their investigational microdystrophin gene transfer for DMD and we are so excited as this might become a life-changing treatment for people with DMD.
SGT-001 Clinical trial is an Incredible milestone
This is an incredible milestone for this startup company of four years old. This accomplishment is the reflection of years of ongoing collaboration with the top experts in gene therapy. SGT-001 is based on pioneering research in dystrophin biology by Dr. Dongsheng Duan of the University of Missouri and Dr. Jeffrey Chamberlain of the University of Washington.
In case you don’t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted last year in London with Dr Jeffrey Chamberlain:
About the clinical trial:
The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD. IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. The patient screening will begin at their first participating study in one location in the United States in the coming days. Solid Biosciences is working to bring on additional sites in the United States and abroad.
About SGT-001
SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. SGT-001 is a systemically administered* candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.
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Adeno-associated virus > is a small virus which infects humans and some other primate species. Link
- Systemically administered > Systemic forms of administration affect the whole body (in general).
To know more
Open letter de Solid Biosciences
If you are interested in learning more about this trial (IGNITE DMD), further information will be available on clinicaltrials.gov shortly.
Gene transfer has the potential to address the genetic cause of Duchenne muscular dystrophy.
