BB-401

BB-401 is an investigational gene-silencing therapy consisting of a recombinant DNA plasmid construct designed to produce antisense RNA targeting the Epidermal Growth Factor Receptor (EGFR). Developed by Benitec Biopharma through strategic licensing agreements with NantWorks and the University of Pittsburgh, BB-401 is primarily intended for the treatment of recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). The therapeutic approach involves intratumoral injection of the DNA plasmid, which then facilitates the local production of antisense RNA to inhibit the translation of EGFR mRNA. Since EGFR is frequently overexpressed in HNSCC and is linked to poor clinical outcomes, silencing its expression aims to reduce tumor proliferation and improve patient survival in refractory cases.

Other names

EGFR antisense DNAEGFR ASEGFR antisense gene therapy