Cyagen, Neurophth Ink $140 Million Deal For AI-Designed AAV Capsids

Cyagen, a Santa Clara, California-based contract research organization (CRO), and China’s Neurophth have entered a deal estimated to be worth $140 million to co-develop the next generation of AAV gene therapy vectors for genetic disorders of the eye. 

Terms Of The Deal 

Under the collaboration, Cyagen will leverage its AI-powered AAV vector discovery platform to identify AAV capsids that can target tissue with higher efficiency and specificity. 

Both companies will assess the functional properties of the capsids in rodent and non-human primate (NHP) models. Neurophth will take over for clinical trials and commercialization of gene therapy products birthed in the deal. 

The Wuhan, China-based biotech has promised Cyagen research and clinical milestone payments plus sales royalties that could exceed $140 million. 

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AI-Powered AAVs And Animal Models 

Cyagen’s AAV discovery platform utilizes AI and single-cell RNA sequencing technologies. The company has fed large amounts of experimental data to train their AI models and developed machine learning algorithms to speed up the AAV capsid identification and optimization processes. The platform is also designed to overcome the limitations of AAV gene therapies through better tissue targeting. 

The company further specializes in developing humanized animal models of rare diseases for preclinical research, with a focus on genetic eye and nervous system disorders. 

Cyagen’s specialties complement Neurophth’s goals for developing gene therapy-based eye treatments. The Chinese biotech’s lead candidate NR082 for ND-4 mediated Leber’s hereditary optic neuropathy (LBON) has been granted Orphan Drug status in the US and Europe. It is also the first Chinese gene therapy to secure IND approval from the NMPA, China’s drug regulator. In September, the first patient was dosed with NR082 in its Phase 3 clinical trial for LBON. 

“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,” said Lance Han, president of Cyagen.

“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.”

Author

Joy Lin

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