CGF166

CGF166 is an experimental gene therapy developed for the treatment of severe-to-profound hearing loss. It consists of a recombinant adenovirus 5 (Ad5) vector carrying a cDNA encoding the human Atonal transcription factor (Hath1, also known as ATOH1). The therapy is designed to promote regeneration of sensory hair cells in the inner ear by delivering the atonal gene directly into cochlear supporting cells via intra-labyrinthine infusion. The goal is to enable these supporting cells to become new, functional hair cells, potentially restoring hearing function. Clinical trials have evaluated its safety and efficacy in patients with severe-to-profound unilateral or bilateral hearing loss[1][3][4][5][8]. Development was led by Novartis and GenVec, with Baylor College of Medicine as originator[4]. Trials were suspended after limited efficacy was observed in early-phase studies[6][7].

Other names

Hearing loss gene therapy