Cyagen today announced a strategic collaboration with Neurophth Therapeutics, Inc. 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 (AI)-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 NHP 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.

Solving the Challenges of Gene Therapy Research and Development

Utilizing AI and single-cell RNA-sequencing technologies, Cyagen’s 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 will develop the world’s best AAV ophthalmic gene therapy products and bring brightness back to patients all over the world."

“Neurophth hopes to seek like-minded partners to work together to achieve breakthroughs in ophthalmic gene therapy development, and I think Cyagen is our ideal companion on the road ahead,” said Bin Li, Founder, Chairman and CEO of Neurophth.



About Cyagen

Founded in 2006, Cyagen is a global provider of genetically modified rodent models and innovative one-stop cell and gene therapy solutions for R&D, including: disease model development, AAV discovery, drug efficacy studies, and more. Cyagen currently has over 900 employees and multiple facilities with a total scale of over 40,000 square meters. The company has established extensive cooperations with scientists and institutions in more than 100 countries, leading to the publication of over 6,300 academic articles - including the three major journals of CNS (Cell, Nature, Science). From its foundation in animal model development, to implementation of AI-powered tools for data analysis and therapeutic discoveries, Cyagen provides one-stop solutions for accelerating basic research and new drug R&D with our unique offering of models, data, algorithms, and services.

About Neurophth

Neurophth is China's leading gene therapy company focusing on ophthalmic diseases. NR082 (NFS-01), Neurophth' core product, designed to treat ND4-mediated Leber's hereditary optic neuropathy (ND4-LHON), has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and is the first Chinese gene therapy new drug that has been granted IND approval for clinical trials by Chinese National Medical Products Administration (NMPA) and the US FDA. At present, the first patient has been dosed in the Phase III clinical trial in September 2022. The company's pipeline also includes ND1-mediated LHON (the company's second new drug with ODD granted by the US FDA), autosomal dominant optic atrophy, optic nerve protection, vascular retinopathy and other preclinical candidates.

Preclinical Ophthalmology Research Solutions

As a comprehensive contract research organization (CRO) solution provider, Cyagen recognizes ophthalmic diseases as a breakthrough point for gene therapy and has established an ophthalmic gene therapy platform to overcome the above obstacles. We have equipped the platform with state-of-the-art ophthalmic instruments for small animals and an experienced professional team. With 16 years of gene editing model construction experience, Cyagen can provide you with an array of standardized preclinical research solutions for ophthalmic gene therapy.

Gene Therapy Research Solutions

As a comprehensive solution provider, Cyagen has established an innovative CRO platform that provides researchers with leading-edge genetically modified model services. We have accumulated a large amount of data on biological information and gene editing over the past decade in our continuous dedication to cell and animal models which have allowed us to play a leading role in the gene-editing field.

Cyagen provides one-stop solutions for gene therapy development to meet the needs for all levels of research. Our services include CRISPR Cas9 Screening & Target Validation, Custom Mouse Model Generation, Efficient Adenovirus/Lentivirus Packaging of adeno-associated virus (AAV), lentivirus (LV), adenovirus (ADV), Pharmacology and Pharmacodynamics (PD) Studies, and more. We hope that our services can improve the efficiency of gene therapy research results.

Adeno-associated Virus Packaging

Adeno-associated viruses (AAVs) are promising therapeutic viral vectors. Cyagen can provide adeno-associated virus vectors design and delivery services. For AAV virus packaging, we adopt a three-plasmid co-transfection method. The advanced purification process can provide customized AAV packaging services with high purity, high titer, and different serotypes. It is especially suitable for in vivo animal experiments and can meet the personalized choices of gene therapy researchers.

>> Check out more details on Adeno-associated virus packaging