BB-301

BB-301 is an investigational gene therapy developed for the treatment of oculopharyngeal muscular dystrophy (OPMD). It utilizes a modified adeno-associated virus serotype 9 (AAV9) vector to deliver a single bifunctional construct that both silences the expression of mutant Poly-A Binding Protein Nuclear-1 (PABPN1) using two small inhibitory RNAs (siRNAs), and simultaneously provides muscle cells with a codon-optimized, functional version of PABPN1. This "silence and replace" mechanism aims to halt disease progression by reducing toxic mutant protein levels while restoring normal protein function in affected tissues. The therapy is administered via direct injection into target muscles and has shown promising results in early clinical trials, improving swallowing function in OPMD patients without significant adverse events. BB-301 has received Orphan Drug Designation in both the United States and European Union[1][4][5][6][8].

Other names

Pabparna