AAV-SLB101

AAV-SLB101 is a proprietary, rationally engineered adeno-associated virus (AAV) capsid developed by Solid Biosciences for enhanced muscle tropism and reduced liver uptake. It is designed to improve the delivery efficiency and safety of gene therapies targeting neuromuscular diseases, particularly Duchenne muscular dystrophy (DMD). Compared to the commonly used AAV9 capsid, AAV-SLB101 demonstrates superior transduction efficiency in skeletal muscle and significantly lower biodistribution to non-target tissues such as the liver and brain. Preclinical studies in mice and non-human primates have shown that vectors using this capsid achieve higher levels of therapeutic protein expression in muscle tissue while minimizing off-target effects. The first clinical application of AAV-SLB101 is as the vector capsid for SGT-003, Solid’s next-generation gene therapy for DMD. Additionally, Armatus Bio has licensed this capsid for use in developing ARM-201, a vectorized RNAi therapy for facioscapulohumeral muscular dystrophy (FSHD)[1][2][3][4][5][6][7][8].