Cyagen and Neurophth Enter Global Strategic Collaboration to Develop AI-Designed AAV Gene Therapy Vectors for Ophthalmic Disorders

Cyagen Biomodels LLC

PR98615

 

SANTA CLARA, Calif., Nov. 3, 2022 /PRNewswire=KYODO JBN/ --

 

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.

 

2255 Martin Avenue,Suite E,Santa Clara,CA 95050-2709,US

800-921-8930(8-6pm PST)

animal-service@cyagen.com

 

SOURCE:Cyagen Biomodels LLC

 

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