retinal-disease
(3)
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.
Neurotech Pharmaceuticals, Inc.
Neurotech Pharmaceuticals is a Cumberland, Rhode Island biotechnology company that develops long-acting therapies for chronic retinal diseases using its Encapsulated Cell Technology (ECT) platform - a tiny implant of genetically engineered cells that continuously secretes a therapeutic protein inside the eye. In March 2025 its lead product ENCELTO (revakinagene taroretcel-lwey, formerly NT-501/Renexus) became the first and only FDA-approved treatment for macular telangiectasia type 2 (MacTel), delivering ciliary neurotrophic factor (CNTF) to slow the loss of photoreceptors.
Therini Bio, Inc.
Therini Bio is a clinical-stage biotechnology company developing fibrin-targeting immunotherapies for neurodegenerative and retinal diseases driven by vascular dysfunction. Founded in 2019 on the neurovascular research of Dr. Katerina Akassoglou at UCSF and the Gladstone Institutes, the company's lead antibody THN391 selectively blocks fibrin's inflammatory epitope to halt chronic neuroinflammation without interfering with normal blood clotting. Backed by roughly $145M in total funding from investors including Eli Lilly, Sanofi Ventures, Merck's MRL Ventures and the Dementia Discovery Fund, Therini is advancing THN391 into Phase 1b trials for Alzheimer's disease and diabetic macular edema.