CHARLESTOWN, Mass. — Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today shared that the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), the list of conditions recommended for universal newborn screening across the United States.
For nearly a decade, Solid has been a staunch supporter and a steering committee member of Parent Project Muscular Dystrophy’s (PPMD) committee dedicated to implementing newborn screening for Duchenne, beginning with a state-level pilot program in New York that was completed in 2021. Adding Duchenne to the RUSP is expected to accelerate detection efforts, leading to earlier access to specialists and supportive intervention and treatment options, potentially improving outcomes for those living with Duchenne.
“At Solid, we are honored to stand alongside PPMD in championing the inclusion of Duchenne on the RUSP, a milestone born from years of rigorous evidence generation, tireless advocacy, and the unwavering commitment of families, clinicians, researchers, and industry partners,” said Annie Ganot, SVP of Patient Advocacy and Co-founder of Solid Biosciences. “This achievement marks a transformative moment for newborn screening, ensuring earlier diagnosis and access to vital resources for newly diagnosed families. As we celebrate this progress, we remain deeply focused on delivering our differentiated, investigational gene therapy, SGT-003, to the Duchenne community with urgency and care, driven by the belief that continued innovation in the treatment landscape can bring new hope to families everywhere.”
About Duchenne Muscular Dystrophy
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.
About SGT-003
SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies. SGT-003’s microdystrophin construct uniquely includes the R16/17 binding domain, which localizes nNOS to the muscle membrane. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com or follow the company on X.
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Solid Biosciences Inc.
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