AAV.GMU01-amiRNA

AAV.GMU01-amiRNA is an adeno-associated virus (AAV) gene therapy designed for the treatment of peripheral neuropathic pain. Developed by Sanofi, the therapy utilizes the AAV.GMU01 vector to deliver artificial microRNAs (amiRNAs) that specifically target and knock down the expression of SCN9A (encoding the NaV1.7 sodium channel) and SCN10A (encoding the NaV1.8 sodium channel) in the dorsal root ganglia (DRG). These sodium channels are critical mediators of pain signal transmission from the periphery to the central nervous system. By reducing the aberrant activity of nociceptive neurons through a one-time intra-cerebrospinal fluid (CSF) administration, AAV.GMU01-amiRNA aims to provide permanent pain relief. Preclinical data in non-human primates and human DRG explants have demonstrated efficient target knockdown and high specificity.