Taysha Gene Therapies is launching with an ambitious plan to advance at least 15 projects developed by specialists at University of Texas-Southwestern's medical school.
The Dallas-based company officially unveiled itself Wednesday, announcing it had received $30 million in venture backing. Taysha is led by an executive team that helped develop the second gene therapy to receive U.S. approval, Zolgensma, a treatment for the rare neuromuscular disorder spinal muscular atrophy.
The large arsenal of programs is part of a strategy to build a sustainable company, breaking with the past where gene therapy developers were primarily built to be purchased by large pharmaceutical companies. The first two to achieve Food and Drug Administration approval, AveXis and Spark Therapeutics, were acquired by the Swiss drugmakers Novartis and Roche, respectively.
In the small rare disease populations being targeted by many gene therapies, sales are likely to reach their peak and plateau, or even fall off, as already sick, or "prevalent," patients are treated with a one-and-done medicine, to be replaced only by newly diagnosed, or "incident" patients, R.A. Session II, Taysha's CEO, told BioPharma Dive in an interview.
Taysha hopes to counteract that challenge by starting up with a large slate of programs in development. The company's highly ambitious goal is to launch a product every two to three years, Session said, so "as soon as one product is starting to go down and treat its incident population, we'll be starting to launch a new product to treat its prevalent population."
"It's a really cool model but you're only able to do it if you have multiple programs in development that are appropriately staged," he said.
Sessions said the experience of AveXis, where he headed up corporate strategy and business development, was instructive. As big pharma buyers were circling, he was asked to seek out other gene therapies to add to the AveXis pipeline to help build the company's value.
Ultimately, Novartis bid $8.7 billion to buy AveXis largely for the product that would become Zolgensma. However, Session said he doesn't view single-asset companies as the future of gene therapy: “You're starting to see less and less single-asset deals. Either the company has a lead program, a manufacturing capability and a platform to create new programs, or companies that people are getting excited about have multiple shots on goal to create sustainability.”
The company represents the first gene therapy spinout from the University of Texas-Southwestern. Its center is headed by Steven Gray, who did his postdoctoral work in the University of North Carolina's well-known gene therapy laboratories.
On the business and financial side, the board will be chaired by former AveXis CEO Sean Nolan, and the lead investor in the funding round is PBM Capital, the first institutional investor in AveXis.
UT-Southwestern is also an equity investor in the company, a partnership that is growing more common in gene therapy development, and Claire Aldridge, the university medical center's associate vice president of commercialization, will serve on the board.
Taysha has disclosed the first four programs it plans to advance. The lead product is called TGTX-101, a gene replacement treatment for a rare condition called GM2-Gangliosidosis, which is sometimes called Tay–Sachs and Sandhoff disease. Human trials should begin later this year.
The three others are aimed at the rare neurological disorders SURF1 deficiency, SLC6A1 haploinsufficiency and Rett syndrome. Taysha plans to file applications to begin human testing for each by the end of 2021.
UT-Southwestern will be responsible for pre-clinical discovery, development and manufacturing work. Once the programs advance, Taysha will take over clinical development, regulatory, commercialization and commercial-scale manufacturing.
The hand-off between the two may be smoother than has happened in the past, as biopharma companies have attempted to replicate gene therapies born in small, university-based manufacturing facilities in larger commercial-scale plants.
The university uses a gene therapy delivery tool — a type of adeno-associated virus — that can be scaled for both small and large manufacturing needs. That may eliminate the need for comparability studies before advancing into human research and potentially speed products toward market.
“There's no secret sauce in it,” he said. “At some point we'll be tech transferring that clinical process over to our commercial facility.”