Rare Daily Staff

Solid Biosciences said that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne muscular dystrophy, a rare, progressive fatal muscle disease.

Solid’s lead candidate, SGT-001, is an experimental adeno-associated viral vector-mediated gene transfer being studied to address the underlying genetic cause of DMD. The viral vector is used to deliver microdystrophin, a synthetic dystrophin gene that encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase.

Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or halt the progression of DMD, regardless of the specific genetic mutation of a patient or disease stage.

SGT-001 is based on research in dystrophin biology by Dongsheng Duan of the University of Missouri and Jeffrey Chamberlain of the University of Washington. The U.S. Food and Drug Administration granted SGT-001 Rare Pediatric Disease Designation and Orphan Drug Designations. SGT-001 also has an orphan drug designation in the European Union.

“For more than three years, we’ve been working on a preclinical package and scalable manufacturing process for SGT-001 that would allow us to responsibly move into the clinic,” said Ilan Ganot, founder and CEO of Solid Biosciences. “This work, as well as decades of research by our scientific advisors and insight from the DMD community, helped us design a clinical trial that will efficiently characterize the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients, regardless of their underlying genetic mutation.”

The phase 1/2 study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with DMD. Enrollment will begin at the first clinical trial site in the United States in the coming days.

IGNITE DMD is an adaptive study. It will use a single-ascending dose. Solid will adjust the dose and number of participants as the study progresses to efficiently assess the safety and efficacy of SGT-001. Solid expects to enroll approximately 16 to 32 patients in the study, all of whom will receive a systemic dose of SGT-001. The starting dose was selected based on Solid’s extensive preclinical program across multiple animal species. All clinical drug product is being produced utilizing Solid’s scalable manufacturing process.

November 30, 2017

Photo: Ilan Ganot, founder and CEO of Solid Biosciences 

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