Santa Clara, California.And the November 8, 2022 /PRNewswire/ — Today, Cyagen announced a strategic collaboration with Neurophth Therapeutics, Inc. To participate in the development of the next generation of AAV gene therapy vectors for certain types of genetic eye disorders.
Under the terms of the agreement, Cyagen will implement its high-throughput artificial intelligence (AI)-powered platform to discover new AAV vectors with optimal tissue targeting capability, tissue specificity and throughput. Cyagen and Neurophth will be responsible for evaluating the functional properties of new AAV vectors in rodent and NHP models, and Neurophth will be responsible for conducting clinical trials and marketing of gene therapy products developed using Cyagen’s new AAV capsules. Cyagen can receive research stage and main clinical stage payments, as well as sales revenue that they may exceed 140 million dollars.
Solving research and development challenges in gene therapy
Using artificial intelligence and single-cell RNA sequencing technologies, Cyagen’s high-throughput AAV vector discovery platform helps overcome current limitations of gene therapy research and development by rapidly identifying next-generation AAV capsids that have enhanced tissue targeting ability, tissue specificity and throughput. Cyagen has produced large experimental data to train the AI model and developed special machine learning algorithms to speed up AAV identification and optimization processes compared to traditional directed evolution methods.
Recognizing the growing potential of eye gene therapy, Cyagen’s Ophthalmology Research Solutions platform is fully equipped with the latest ophthalmic tools and an experienced professional team.
“Gene therapy has shown great promise and potential for treating hereditary eye diseases, and the eye gene therapy market has expanded significantly in the past few years,” said Lance Hahn, president of Cyagen. “Together with Neurophth, we will develop the world’s best AAV gene therapy products and bring the brightness back to patients around the world.”
“Neurophth hopes to seek like-minded partners to work together to achieve breakthroughs in the development of eye gene therapy, and I believe Cyagen is our ideal future companion,” said Ben Lee, Founder, Chairman and CEO of Neurophth.
Founded in 2006, Cyagen is a global provider of genetically modified rodent models and comprehensive innovative cell and gene therapy solutions for research and development, including: disease model development, AAV discovery, drug efficacy studies, and more. Cyagen currently has more than 900 employees and multiple facilities with a total scale of over 40,000 square metres. The company has built extensive partnerships with scholars and institutions in more than 100 countries, resulting in the publication of more than 6,300 academic articles – including the three major journals in CNS (The cell, nature and science). From its foundation in animal model development, to the implementation of AI-powered tools for data analysis and therapeutic discovery, Cyagen provides comprehensive solutions to accelerate basic research and research and development of new drugs through our unique offerings of models, data, algorithms, and services.
Neurofth is China A leading gene therapy company focused on eye diseases. NR082 (NFS-01), Neurophth’s primary product, designed for the treatment of ND4-mediated hereditary optic neuropathy (ND4-LHON) has been awarded orphan drug designation (ODD) by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and is the first new Chinese gene therapy drug to be granted IND approval for clinical trials by the China Medicinal Products Administration (NMPA) and the US Food and Drug Administration. Currently, the first patient underwent a dose in a Phase 3 clinical trial in September 2022. The company’s pipeline also includes LHON mediated by ND1 (the company’s second new drug with FDA-granted ODD), autosomal dominant optic atrophy, and protective optic atrophy. , vascular retinopathy and other preclinical candidates.
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