Astellas Invests $50 Million In Taysha, Licenses Two CNS Gene Therapies

Astellas has invested $50 million in Taysha Gene Therapies to help advance Taysha’s AAV gene therapy programs for two rare CNS diseases, Rett syndrome and giant axonal neuropathy (GAN). In return, Astellas gained an option to license the two clinical assets: TSHA-102 for Rett syndrome and TSHA-120 for GAN. 

The move sees Astellas take up a 15% ownership stake in Taysha and a Board observer seat on the company’s board of directors.

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Gene Therapy For Rare CNS Diseases 

Taysha is focused on developing AAV gene therapies for rare CNS diseases caused by a single gene mutation. Taysha’s TSHA-102 is designed to replace the mutated MECP2 gene responsible for Rett syndrome, which causes progressive loss of motor skills and language. Preclinical studies suggested the investigational therapy restored behavior in knockout Rett mice. 

TSHA-102 has received orphan drug designations from the US FDA and the European Commission and a rare pediatric disease designation from the FDA. It is currently undergoing a first-in-human Phase 1/2 trial, with data expected by the end of 2022. 

Taysha’s other candidate TSHA-120 aims to treat GAN, characterized by abnormally large axons that cause progressive loss of motor skills and also impact the autonomic nervous system, leading to problems with excretion. TSHA-120 has been shown to improve neuronal functioning and regenerate nerve fibers. Taysha is anticipating regulatory feedback from the FDA for the gene therapy by the end of 2022.

Taysha’s approach to gene therapies aligns with Astellas’ goals. Gene therapy has been an area of focus for Astellas since its $3 billion acquisition of Audentes in January 2020. The company recently welcomed the opening of its commercial GMP manufacturing plant in North Carolina this June. 

“Taysha is an industry leader in CNS gene therapies and this partnership fits strategically with our long-term vision of expanding Astellas’ gene therapy capabilities, allowing the company to impact the lives of a broader range of patients with urgent unmet medical needs,” said Naoki Okamura, Chief Strategy Officer, at Astellas.

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Joy Lin

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