AAVtcm8-coNAGLU

AAVtcm8-coNAGLU is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB; MPS IIIB). Developed at the University of Florida, the vector utilizes a mutated AAV8 capsid (AAVtcm8) to deliver a codon-optimized version of the human alpha-N-acetylglucosaminidase (NAGLU) gene. MPS IIIB is a lysosomal storage disorder caused by NAGLU deficiency, leading to the accumulation of heparan sulfate and progressive neurodegeneration. Preclinical studies in mouse models have demonstrated that neonatal central nervous system administration can rescue hearing loss, restore lysosomal storage levels, and extend lifespan. The program also includes a CpG-depleted variant intended to minimize innate immune activation.