Taysha, Yale to Advance Gene Therapy Program

Note: This story was updated April 22, 2021, to clarify the collaboration between Yale and Taysha at the company’s request. 

Taysha Gene Therapies has established a new research partnership with Yale University to advance its gene therapy program for neurodevelopmental disorders, which includes Rett syndrome, the company announced.

The multi-year collaboration is intended to develop next-generation mini-gene payloads for Taysha’s gene therapies, which use harmless adeno-associated viruses (AAVs) as a gene delivery carrier.

The collaboration follows previously established partnerships with the Cleveland Clinic and the UT Southwestern Medical Center (UTSW) and later with Catalent. The goal of these collaborations is to advance Taysha’s gene therapy program by expanding the company’s manufacturing capacity. Both UTSW and Catalent have manufacturing facilities that follow Good Manufacturing Practices.

“Our collaboration with Yale is a key addition to our established partnerships with Cleveland Clinic and UTSW, designed to advance our breakthrough next-generation mini-gene platform and to potentially expand the range of genetic diseases that can be treated by AAV-based gene therapy,” said Suyash Prasad, chief medical officer and head of research and development at Taysha.

Under the new agreement, researchers at Yale University will generate mini-gene payloads for the treatment of neurodevelopmental disorders. Taysha has exclusive rights on new gene contructs and all intellectual property related to and stemming from the collaboration.

The AAV vectors that will carry the mini-gene payloads will be developed by the researchers at UTSW, who also are responsible for conducting further lab (in vitro) and in vivo studies of effectiveness.

“We look forward to harnessing each partner’s unique capabilities and expertise to better address the challenge of vector capacity and believe our collective efforts may allow significant advancement in the field of gene therapy,” Prasad added.

In the case of Rett syndrome, Taysha is developing a gene therapy known as TSHA-102. It delivers a mini-version of the MECP2 gene together with a “switcher” to turn on the gene exclusively in nerve cells. Notably, most Rett cases are caused by mutations in the MECP2 gene, which provides instructions to make a key protein for normal brain function.

By the time the company announced the collaboration with UTSW, Taysha said it aimed to file for investigational new drug applications, a requirement to start clinical trials in the U.S., to test its gene therapies. The target indications include Rett syndrome and GM2-Gangliosidosis, a rare disorder that affects the central nervous system (brain and spinal cord).