dry-amd
(2)
Beacon Therapeutics
Beacon Therapeutics is a clinical-stage ophthalmic gene therapy company building AAV-based treatments to preserve and restore sight for people with rare and prevalent retinal diseases that cause blindness. Formed in 2023 by Syncona around the assets of Applied Genetic Technologies Corp (AGTC) and complementary science from the University of Oxford, its lead program, laru-zova (AGTC-501), is a one-time gene therapy for X-linked retinitis pigmentosa now in registrational trials, with additional programs targeting geographic atrophy (dry AMD) and cone-rod dystrophy.
Character Biosciences
Character Biosciences is a precision medicine company developing targeted therapies for progressive polygenic diseases, starting in ophthalmology with age-related macular degeneration (AMD). By pairing human genetics with longitudinal clinical and retinal imaging data from thousands of consented patients - and layering AI-enabled biomarker analysis on top - Character reclassifies broad diagnoses into genetically defined subtypes so it can design drugs that target the root drivers of disease progression. Its lead programs, CTX114 (a complement inhibitor for geographic atrophy) and CTX203 (a lipid regulator to prevent progression to advanced AMD), are advancing toward the clinic on the strength of a Series B that grew past $110 million.