Taysha’s Gene Therapy Programs Get $50M Funding Boost From Astellas

Gene therapies being developed for Rett syndrome and giant axonal neuropathy

Vanda Pinto, PhD avatar

by Vanda Pinto, PhD |

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Taysha Gene Therapies is partnering with Astellas Pharma to support the development of its gene therapy programs for Rett syndrome and giant axonal neuropathy, another progressive neurodegenerative disorder.

Astellas will invest $50 million to acquire 15% of Taysha and to receive an exclusive option to license Taysha’s experimental gene therapies: TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy.

The agreement will support the development of both treatments by harnessing Astellas’ expertise in gene therapies. Astellas recently opened a state-of-the-art commercial Good Laboratory Practice manufacturing facility in North Carolina.

“We are excited to enter this strategic investment with Astellas, a premier biopharmaceutical company with global R&D, manufacturing and commercial capabilities,” RA Session II, Taysha’s CEO, said in a press release.

“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,” he added.

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Nearly all cases of Rett syndrome are caused by mutations in the MECP2 gene which results in insufficient or defective MECP2 protein. This protein is important for brain development and function.

TSHA-102 uses a harmless adeno-associated virus to deliver a shorter but functional version of MECP2 that is only switched on in nerve cells. It is also designed with a self-regulatory suppressor called a miRNA-responsive auto-regulatory element (miRARE), a feature that prevents the excess production of the MECP2 protein.

The therapy is administered by injection into the spinal canal fluid (intrathecally).

Recently, Taysha launched the clinical program for TSHA-102 following the approval of a clinical trial application by Health Canada. Preliminary Phase 1/2 clinical findings are expected by the end of this year.

Preclinical results, which supported the application, showed positive safety and efficacy data. In a Rett mouse model that lacked the mouse equivalent of the MECP2 gene, a one-time injection increased survival, and improved body weight, motor function, and lung assessments.

Additionally, a six-month toxicology study in nonhuman primates showed that TSHA-102 was distributed in several areas of the brain and spinal cord. No toxic overexpression of the MECP2 protein, changes in behavior, or adverse tissue findings were reported.

Under the new agreement, Astellas will receive one board observer seat on Taysha’s board of directors, as well as certain rights related to any potential change of control of Taysha.

“Gene therapy is the corner stone of Astellas’ Primary Focus, Genetic Regulation,” said Naoki Okamura, chief strategy officer at Astellas. “Our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options.”

“Taysha is an industry leader in [central nervous system, comprising the brain and spinal cord] 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,” added Okamura.