IVB303

IVB303 is an adeno-associated virus (AAV)-based gene therapy being developed by InnoVec Biotherapeutics for the treatment of Neuronal Intranuclear Inclusion Disease (NIID). The therapy utilizes an AAV vector to deliver microRNA (miRNA) sequences designed to target and silence the expression of the *NOTCH2NLC* gene. NIID is a rare neurodegenerative disorder characterized by GGC repeat expansions in the *NOTCH2NLC* gene, leading to the formation of toxic eosinophilic intranuclear inclusions in neurons and other tissues. By employing the RNA interference (RNAi) pathway to reduce the levels of these pathogenic transcripts, IVB303 aims to mitigate protein aggregation and slow the progression of neurodegeneration. The program is currently in the discovery and early preclinical stages, with delivery intended for the central nervous system.