Taysha Gene Therapies hit the ground running when it launched this spring, cruising through a $95 million series B straight to a $100 million IPO in September. With its lead program now approved for human trials in Canada, the company is sketching out plans for what it hopes will become its prime commercial manufacturing site.
Taysha leased a 187,000-square-foot commercial-scale manufacturing facility in Durham, North Carolina, planning to invest $75 million to kit out the facility for preclinical, clinical and commercial gene therapy production. It’s also due to receive up to $9.4 million in state and local incentives for the project, Taysha said in a release.
The company will add some 200 jobs over the next two and a half years, building out its development, analytical, manufacturing and quality control testing capability as it advances its portfolio of adeno-associated vector (AAV) gene therapies for monogenic diseases—caused by a defect in a single gene—of the central nervous system. The new plan is expected to be fully operational in 2023.
Taysha aims to cultivate a diverse workforce of bioprocess engineers and manufacturing associates, as well as biochemists and molecular biologists—and it’s already recruited some top talent, Fred Porter, Ph.D., chief technical officer at Taysha, said in an interview. Having innovators and experts in manufacturing in the same place is “central to the success of a gene therapy startup like Taysha,” he said.
Small but very ambitious, Taysha launched in April with a team of former AveXis execs in charge, $30 million in seed funding and a partnership with the University of Texas (UT) Southwestern Medical Center good for 15 AAV gene therapy programs. It’s moved fast, too, bringing three more programs into the fold since then. And just this week, Taysha’s lead program was cleared by Health Canada for a phase 1/2 trial in infantile GM2 gangliosidosis—a rare disorder that progressively destroys nerve cells in the spinal cord and brain.
“Part and parcel to that is we believe that with a portfolio this deep we need to build internal manufacturing capacity,” Porter said. Hence the investment in the Durham space, pegged to shore up Taysha’s primary commercial production needs plus process development, quality control and clinical support for its programs, he said.
With multiple production suites set to come online by 2023, the facility will boast capacity for projects up to 2,000 liters. It will leverage Taysha’s HEK293 suspension process—a next-gen iteration of a cell line manufacturing approach that uses traditional bioprocess manufacturing equipment—to grow cells at high density in a way that’s familiar to any CDMO or biologics manufacturer, Porter said.
The facility will also house a “scaled-down” development lab, which the company will use to model commercial-scale processes in smaller reactors. But manufacturing is only half the battle, Porter figures. Equally important is an arsenal of analytical tools like qPCR, digital PCR, immunoassays, biochemical assays and potency assays, which Taysha will position at the new facility.
“For products like AAV gene therapies, such as the ones we’re working on, the process is the product,” Porter said. “As much as there’s a focus on the manufacturing itself, understanding the product you make is really important."
Meanwhile, the company expects to benefit by housing development, quality control and manufacturing under one roof.
“That’s one of the other strategic benefits we see in building this facility with this scope—that the transfer is across the hall, not across the country or to another facility or another organization,” Porter said.
The facility rounds out Taysha’s three-pillar manufacturing strategy, which comprises its manufacturing collaborations with UT Southwestern and Catalent, tapped in November to boost capacity across Taysha’s portfolio, including treatments for CLN1—which causes nerve cells in the brain to die over time—and the neurodevelopment disorder Rett syndrome.
Taysha will depend on those relationships for clinical supply as it gets its Durham facility up and running, though the company intends to rely heavily on its internal manufacturing once the site is operational, with those partnerships continuing to augment capacity, Porter said.
Taysha isn’t letting the momentum die down, either. Four months after the company uncloaked, it went public with a $100 million IPO in September. That move followed a $95 million series B in July. The company also sketched out plans to hit the clinic by year-end.
Then, just four days after the company announced its lease, Taysha revealed Health Canada had cleared Queen’s University in Ontario to run a phase 1/2 study of its lead program, TSHA-101, in infantile GM2 gangliosidosis.
“TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy,” Suyash Prasad, chief medical officer and head of R&D at Taysha, said in a statement.