Solid Biosciences Touts Encouraging Preclinical Data From Potential Duchenne Gene Therapy

 

• Solid Biosciences Inc 
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   presented additional data characterizing AAV-SLB101, a novel adeno-associated virus (AAV) vector designed for improved transduction efficiency and biodistribution to muscle cells. 
• In studies in wild-type mice, the mdx mouse model of Duchenne (DMDmdx) and non-human primates (NHPs) AAV-SLB101 demonstrated superior transduction efficiency compared with AAV9.
• In DMDmdx mice, the biodistribution of AAV-SLB101 to the quadriceps was significantly increased, and biodistribution to the liver and brain was decreased compared with AAV9.
• In DMDmdx mice, microdystrophin protein expression was significantly higher with AAV-SLB101 than with AAV9.
• Across multiple mouse studies, muscle tissues treated with SGT-003 showed approximately 2- to 3-fold higher levels of microdystrophin protein. 
• The company says the AAV-SLB101 capsid may be a superior candidate for muscle-targeted gene therapies, with the potential to achieve higher levels of efficacy with lower total doses.
• The company expects to submit an investigational new drug application (IND) for SGT-003 in mid-2023 and, subject to IND clearance, initiate patient dosing in late 2023. 

https://www.benzinga.com/general/biotech/22/10/29289577/solid-biosciences-touts-encouraging-preclinical-data-from-potential-duchenne-gene-therapy