Cyagen Biosciences and Research Partners Announce AI-Engineered AAV Platform for Next-Generation Retinal Gene Therapy
Guangzhou, China, July 14, 2026 – Cyagen Biosciences, in collaboration with YIMA Gene, Guangdong Provincial Biotechnology Research Institute, and the Institute of Medical Microbiology at Jinan University, has announced a major breakthrough in ophthalmic gene delivery. The joint research team recently published their findings, entitled "AI-engineered AAV Capsid Enables Intravitreal Delivery for the Treatment of Diverse Retinal Degenerations" in Molecular Therapy Advances.
The study represents a significant milestone in AI-driven AAV capsid engineering, with the technology receiving international peer recognition through publication in a leading gene therapy research journal. By combining Cyagen's proprietary AI-driven biological computing and standardized disease models with the deep preclinical research expertise of its partners, the alliance has successfully introduced AAV2.PN168—a novel adeno-associated virus (AAV) variant poised to overcome the most pressing limitations of conventional retinal gene therapy.
The study was jointly led by Lance Han, Founder of Cyagen Biosciences; Dr. Sheng Ren, Head of YIMA Gene; and Dr. Yu Zhang, Deputy Director of Guangdong Provincial Biotechnology Research Institute, who served as corresponding authors. Mochen Cui, Huaqing Liu, and Lei Cai contributed as co-first authors.
Overcoming Delivery Barriers Through Collaborative Innovation
Historically, retinal gene therapy has relied on subretinal injections—a procedure fraught with surgical complexity and the risk of tissue damage. Recognizing this industry-wide bottleneck, the collaborative team engineered AAV2.PN168 to achieve efficient and widespread gene delivery throughout the retinal layers via a single, minimally invasive intravitreal injection.
Leveraging the combined resources of the partnership, the team rigorously validated the vector's efficacy across diverse preclinical models. AAV2.PN168 demonstrated exceptional retinal delivery capabilities not only in mouse models but also in non-human primates (cynomolgus monkeys). Furthermore, it exhibited sustained, long-term therapeutic efficacy in treating models of Leber congenital amaurosis type 1 (LCA1) and wet age-related macular degeneration (wAMD).
Integrating Artificial Intelligence with Pipeline Development
A key advancement of this research is the establishment of an AI-powered multi-objective iterative screening platform, which integrates computational design, high-throughput screening, and in vivo validation to accelerate AAV capsid discovery and optimization.
By leveraging artificial intelligence and advanced genetic engineering technologies, the platform enables efficient identification of AAV variants with improved retinal targeting capabilities and therapeutic potential. This approach provides a new framework for developing next-generation gene delivery systems for ophthalmic diseases and other therapeutic areas.
"AI-driven biological innovation is transforming the future of gene therapy development," said Lance Han, Founder of Cyagen Biosciences. "Through the integration of AI computation, AAV engineering, standardized disease models, and preclinical evaluation capabilities, Cyagen is committed to advancing innovative gene delivery technologies and accelerating the translation of promising therapeutic candidates."
Advancing the Future of Retinal Gene Therapy
The international peer recognition garnered by this publication serves as a powerful validation of the alliance's AI-enabled engineering capabilities. Moving forward, Cyagen will continue integrating AI-powered biological computing with gene delivery platform development. Through its proprietary AAV technologies, standardized disease animal models, and comprehensive preclinical efficacy evaluation systems, Cyagen aims to collaborate with global biopharmaceutical partners to advance the development of retinal gene therapy candidates.
By delivering innovative gene delivery solutions, Cyagen seeks to contribute to the development of safer, longer-lasting, and more accessible therapeutic approaches for patients affected by retinal degenerative diseases.
Publication Information
Title: AI-engineered AAV Capsid Enables Intravitreal Delivery for the Treatment of Diverse Retinal Degenerations
Journal: Molecular Therapy Advances
About Cyagen Biosciences
Founded in 2006, Cyagen Biosciences is an AI-enabled biotechnology company dedicated to accelerating biomedical innovation through proprietary gene-editing technologies, advanced disease models, computational platforms, and integrated preclinical research solutions.
Cyagen develops and applies cutting-edge technologies in gene editing, humanized models, AAV engineering, and AI-driven biological research to support global drug discovery and translational medicine programs. The company's research capabilities span multiple therapeutic areas, including ophthalmology, oncology, neuroscience, metabolic diseases, autoimmune disorders, and other fields.
With advanced research platforms and global collaboration networks, Cyagen is committed to empowering researchers and biopharmaceutical companies with innovative solutions that bridge scientific discovery and therapeutic development.
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