ADVM-062

ADVM-062 is a recombinant adeno-associated virus (AAV)-based gene therapy vector, specifically utilizing the AAV.7m8 capsid variant, designed to deliver and express the human long-wave-sensitive opsin 1 (OPN1LW) gene in cone photoreceptors of the retina[1][2][4]. The therapy uses a synthetic cone cell-specific expression cassette to drive targeted expression of L-opsin protein in foveal cones[1][4]. It is administered as a single intravitreal injection and aims to restore L-opsin function in patients with blue cone monochromacy (BCM), an X-linked inherited retinal disorder characterized by loss of visual acuity, photophobia, myopia, nystagmus, and impaired daylight vision due to mutations in the L or M opsin genes[4][6][7]. Preclinical studies have demonstrated effective transduction of cone photoreceptors and functional restoration in animal models. The U.S. FDA has granted Orphan Drug Designation for this candidate for BCM[4].

Other names

AAV.7m8-L-opsin