RARE Daily

Taysha Pauses Programs, Cuts Workforce to Extend Cash Runway

April 1, 2022

Rare Daily Staff

Taysha Gene Therapies has joined several other biotechs in efforts to extend its cash runway. The company, which has a pipeline of 30 CNS-focused programs, said it is prioritizing to focus on its experimental therapies for giant axonal neuropathy and Rett syndrome with activities for other ongoing clinical programs minimized and all additional research and development paused to increase its operational focus and efficiency.

“2021 was a year of accomplishment that included positive data from three clinical programs, including GAN, GM2 gangliosidosis, and CLN7 disease. We are sharpening our strategic focus to prioritize key value-driving registration-directed programs in GAN, which has an estimated addressable patient population of 5,000 worldwide, and Rett syndrome, which affects over 350,000 patients worldwide,” said RA Session II, president, founder, and CEO of Taysha in the company’s fourth quarter and full year 2021 results statement. “To increase operational efficiency, activities for other ongoing clinical programs will be minimized and all additional research and development will be paused. As a result, we have reduced our workforce by approximately 35 percent. Our strategic pipeline prioritization, along with existing cash and financing under our current debt facility is expected to extend cash runway into the fourth quarter of 2023.

Taysha will focus its energies on two programs. TSHA-120, an intrathecally dosed AAV9 gene therapy is currently being evaluated in a clinical trial for the treatment of for giant axonal neuropathy (GAN), a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin. TSHA-120 is designed to deliver a functional copy of the GAN gene to the CNS and PNS.

TSHA-102, a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. TSHA-102 is the first-and-only gene therapy in clinical development for Rett syndrome.

The company will stop further enrollment in the phase 1/2 trial of TSHA-101 for GM2 gangliosidosis, but will continue to follow patients who were previously dosed.

Taysha will continue its collaboration with UT Southwestern on a gene replacement therapy for CLN7 Batten disease, while its CLN1 and SLC13A5 programs will limit patient enrollment to focus on proof of concept.

These changes will result in a 35 percent reduction in its workforce, about 60 people.

Photo: RA Session II, president, founder, and CEO of Taysha

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