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An AAV Capsid Viability Prediction Model Published in Molecular Therapy Advances — Cyagen Biosciences’Self-Developed AI-AAV Vector Pioneers a New Minimally Invasive Paradigm for Ocular Gene Therapy
In July 2026, the Gene Therapy Research Team of Cyagen Biosciences, together with Guangdong Institute of Biotechnology and Jinan University, published a research paper titled AI-engineered AAV Capsid Enables Intravitreal Delivery for the Treatment of Diverse Retinal Degenerations in Molecular Therapy Advances. For the first time, this study fully unveiled our in-house AI multi-objective iterative screening platform and AAV2.PN168, a novel AAV variant exclusively optimized for ophthalmic applications. This vector addresses the key drawbacks of conventional retinal gene therapy, namely severe surgical trauma and limited transduction range associated with subretinal injection. A single intravitreal injection delivers robust transduction across the full-thickness retina and macula in both mice and non-human primates (cynomolgus monkeys). Long-term therapeutic potential has been validated in two blinding retinal disease models: Leber Congenital Amaurosis Type 1 (LCA1) and wet Age-Related Macular Degeneration (wAMD). This breakthrough creates an innovative minimally invasive route for the clinical translation of domestic ophthalmic gene therapeutics. Click to view the published article>>>