RARE Daily

Cyagen and Neurophth Collaborate to Develop AAV Gene Therapy Vectors for Ophthalmic Disorders

November 7, 2022

Rare Daily Staff

Cyagen entered into a strategic collaboration with Neurophth Therapeutics to co-develop next-generation AAV gene therapy vectors for specific types of genetic ophthalmic disorders.

Under the terms of the agreement, Cyagen will apply its proprietary artificial intelligence-powered high-throughput platform to discover novel AAV vectors with optimized tissue targeting capability, tissue specificity, and productivity. Cyagen and Neurophth will both be responsible for evaluating the functional properties of the novel AAV vectors in rodent and nonhuman primate models, and Neurophth will be responsible for conducting clinical trials and commercialization for gene therapy products developed using Cyagen’s novel AAV capsids. Cyagen could receive research phase and clinical phase milestone payments, as well as sales royalties that may exceed $140 million.

Utilizing AI and single-cell RNA-sequencing technologies, Cyagen says its high-throughput AAV vector discovery platform helps overcome the present limitations of gene therapy R&D by quickly identifying next-generation AAV capsids that have enhanced tissue targeting capability, tissue specificity, and productivity. Cyagen has produced substantial experimental data for AI model training and developed proprietary machine learning algorithms to accelerate the AAV capsid identification and optimization processes compared to traditional directed evolution methods.

Recognizing the growing potential for ophthalmic gene therapy, Cyagen’s Ophthalmology Research Solution platform is fully equipped with state-of-the-art ophthalmic instruments and an experienced professional team.

“Gene therapy has demonstrated great promise and potential for treating genetic ophthalmic diseases, and the ophthalmic gene therapy market has expanded exponentially in the past few years,” Lance Han, president of Cyagen, said. “Together with Neurophth, we hope to develop the world’s best AAV ophthalmic gene therapy products and bring brightness back to patients all over the world.”

Neurophth is a Chinese gene therapy company focusing on ophthalmic diseases. The company’s lead experimental gene therapy is designed to treat ND4-mediated Leber’s hereditary optic neuropathy and has been granted Orphan Drug designation by the U.S. Food and Drug Administration and the European Medicines Agency, and is the first Chinese gene therapy new drug that has been granted IND approval for clinical trials by the Chinese National Medical Products Administration and the FDA. At present, the first patient has been dosed in the phase 3 clinical trial in September 2022.


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