Beacon Therapeutics

beacon therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. the company has an established scientific foundation that combines a late-stage development candidate to treat x-linked retinitis pigmentosa (xlrp), as well as two preclinical programs, one targeting dry age-related macular degeneration (amd) and another in-licensed from the university of oxford targeting cone-rod dystrophy (crd), an inherited retinal disease. lead development candidate agtc-501, is a gene therapy program in phase ii clinical trials for the treatment of xlrp, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. xlrp is predominantly caused by mutations in the retinitis pigmentosa gtpase regulator (rpgr) gene. agtc-501 expresses the full length rpgr protein, thereby addressing the full complement of photoreceptor damage cause