retinal-disease
(5)
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.
Rich Small
Rich Small is the CEO of Neurotech Pharmaceuticals, the Cumberland, Rhode Island clinical-stage biotech that in March 2025 won FDA approval for ENCELTO, the first and only treatment for macular telangiectasia type 2 (MacTel). A CPA and Northeastern University graduate, he joined Neurotech as CFO in 2007 and took over the top job in 2016, guiding the company's encapsulated cell technology platform from experimental science to a commercial retinal implant.
Xuandai Nguyen
Xuandai Nguyen, Ph.D., MBA, is Vice President, Head of Global Program Strategy and Chief of Staff to the CEO at Beacon Therapeutics, an ophthalmic gene therapy company working to restore sight in people with inherited and prevalent retinal diseases. A scientist-turned-strategist, Nguyen has spent more than two decades moving medicines from bench to market, with senior roles at Genentech, Forma Therapeutics and ACELYRIN spanning immunology, ophthalmology, respiratory and sickle cell disease. Trilingual and Berkeley- and Cornell-trained, Nguyen sits at the intersection of hard science and commercial strategy, translating complicated biology into the plans that decide which therapies reach patients.