aav
(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.
Cyagen Biosciences
Cyagen Biosciences is a life-sciences contract research organization that builds custom genetically engineered rodent models - knockout, knockin, conditional, and humanized mice and rats - for academic labs and drug developers. Founded in 2006 and headquartered in Santa Clara, California, with major operations in China, Japan, and Europe, Cyagen has grown into one of the world's largest suppliers of custom mouse and rat models, complemented by stem-cell products and a fast-growing gene-delivery arm, VectorBuilder, that designs and manufactures viral vectors from research through clinical CDMO work.
克睿基因 Cure Genetics
克睿基因 Cure Genetics is a clinical-stage biotech founded in Suzhou in 2016 that develops cell and gene therapies for solid tumors and genetic diseases. It runs two proprietary platforms: AIMS, an allogeneic CAR-NKT cell therapy approach aimed at hard-to-treat solid tumors, and VELP, a directed-evolution AAV vector platform that builds high-quality viral libraries for tissue-targeted gene delivery to the nervous system, heart, and kidney. Its lead programs include CGC729 (anti-CD70 CAR-NKT for renal cell carcinoma) and CGC-602 (a PD-1-IL-2v bispecific antibody).