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Astellas to support development of Taysha’s AAV-based gene therapy programmes

The company will receive an exclusive option to license two clinical stage programmes



Astellas and Taysha Gene Therapies (Taysha) have announced a strategic investment to support the advancement of Taysha’s adeno-associated virus (AAV) gene therapy development programmes for the treatment of Rett syndrome and giant axonal neuropathy (GAN).

Under the terms of the agreement, the Japanese firm will invest a total of $50m to acquire 15% of the outstanding common stock of Taysha and to receive an exclusive option to license two of Taysha’s clinical stage programmes: TSHA-102 for Rett syndrome and TSHA-120 for GAN.

Rett Syndrome and GAN are monogenic diseases of the central nervous system. Rett Syndrome results from a mutation in the MECP2 gene on the X chromosome and causes a broad spectrum of symptoms, including loss of speech, mobility and muscle tone and breathing issues.

GAN is caused by an abnormality in a gene that encodes for the gigaxonin protein found on chromosome 16. The disorder also impacts mobility and motor abilities and can cause seizures and skeletal abnormalities.

The deal gives Astellas the future option to apply its global research and development, manufacturing and commercialisation capabilities in gene therapy to Taysha’s AAV gene therapy development programmes, creating the opportunity for both companies to enhance the development of novel treatment options for patients with Rett syndrome and GAN, who have serious unmet medical needs.

“Taysha is an industry leader in central nervous system 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.

Also commenting on the deal, RA Session II, Taysha’s chief executive officer, said: “We are excited to enter this strategic investment with Astellas, a premier biopharmaceutical company with global R&D, manufacturing and commercial capabilities.

“We believe this investment not only further validates the potential of our technology platform, but also reinforces the therapeutic and market opportunity of our two lead clinical assets.”

The deal is the latest of Astellas’ efforts to build its capability in gene therapy, following a $1.6bn research collaboration with Dyno Therapeutics in December 2021, as well as its $3bn acquisition of Audentes – now Astellas Gene Therapies, California – in January 2020.

Article by
Emily Kimber

26th October 2022

From: Research

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