Shares in Solid Biosciences Inc plunged 71% on Tuesday after a trial testing its gene therapy for a muscle-wasting disorder was halted for the second time in less than two years. A safety scare has rocked Solid Biosciences’ drive to get its Duchenne muscular dystrophy gene therapy program back on track. The latest stage of the gene therapy’s troubled development began in November, when the FDA placed a phase 1/2 trial on clinical hold in response to a case of acute kidney injury and other symptoms in a child who received the high dose of SGT-001.“We will continue to work with the FDA as we believe the clinical development of SGT-001 could offer meaningful benefits to patients with this devastating disease,” Ganot said in a statement.In disclosing the news, Solid Bio said the FDA wants to see more manufacturing information, updated safety and efficacy data for all patients and details of total viral load to be administered per patient. In preclinical studies, a single administration of SGT-001 led to long-term expression of microdystrophin in muscle and significant improvements in muscle function. Duchenne is caused by mutations in the dystrophin gene that result in … Solid Biosciences' gene therapy candidate, SGT-001, and another from Sarepta Therapeutics called AAVrh74.MHCK7.micro-dystrophin use a … Solid Biosciences’ therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. Shares in Solid Bio have traded at below $5 since the FDA imposed the clinical hold.
Disease-focused and founded by a family directly impacted, our mandate is simple yet comprehensive – work to address the disease at its core by correcting the underlying mutation with our gene therapy candidate, SGT-001. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. However, the natural dystrophin gene is too big to fit into AAV.If successful, the body will utilize the newly produced microdystrophin protein similarly to dystrophin, resulting in a healthier muscle function.Join our team of experts to advance treatments for DuchenneGene therapy is a natural area of focus for Solid because it has the potential to address the root cause of Duchenne muscular dystrophy (Duchenne), the absence of the dystrophin protein. Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). The FDA has lifted its clinical hold on Solid Biosciences’ Phase I/II trial for its lead candidate, the Duchenne muscular dystrophy (DMD) gene therapy SGT-001, the company said today. Solid Biosciences Inc (NASDAQ: SLDB) shares pulled back to all-time lows Tuesday after the company said its lead gene therapy candidate to treat … Solid Biosciences (SLDB) amends its phase I/II gene therapy study protocol to expedite the development of its lead candidate, SGT-001, as potential treatment for DMD. Shares of Solid Biosciences were down nearly 9% in premarket trading after the company announced late Friday that the U.S. Food and Drug Administration (FDA) has not yet lifted a clinical hold on its Phase I/II IGNITE Duchenne muscular dystrophy study..
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