IVB304

IVB304 is an adeno-associated virus (AAV) gene therapy being developed by InnoVec Biotherapeutics for the treatment of Amyotrophic Lateral Sclerosis (ALS). The therapy utilizes a microRNA (miRNA) approach to target and silence the expression of the superoxide dismutase 1 (SOD1) gene. Mutations in the SOD1 gene are a significant cause of familial ALS, leading to the production of misfolded proteins that are toxic to motor neurons. By delivering SOD1-targeting miRNA via an AAV vector directly to the central nervous system, IVB304 aims to reduce the levels of mutant SOD1 protein, thereby potentially slowing or halting the neurodegenerative process. The program is currently in the discovery stage of development.