IVB503

IVB503 is an adeno-associated virus (AAV) gene therapy candidate developed by InnoVec Pharmaceutical Technology for the treatment of Myotonic Dystrophy type 1 (DM1). DM1 is a multisystemic neuromuscular disorder caused by an unstable CTG repeat expansion in the 3' untranslated region of the DMPK gene, which results in the accumulation of toxic RNA foci that sequester essential splicing factors. IVB503 utilizes an AAV vector to deliver a therapeutic payload designed to mitigate the effects of these toxic RNA species or reduce DMPK expression. The program is currently in the preclinical stage of development as part of InnoVec's neuromuscular disease pipeline.