The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and they’re wasting no time. With 15 programs, $30 million in seed funding and an “unrivaled” partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.
It’s working on adeno-associated vector (AAV) gene therapies for monogenic diseases—that is, diseases caused by a defect in a single gene—of the central nervous system. Taysha’s lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the company’s portfolio includes conditions that are more prevalent.
The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.
And that’s not all—Taysha has the option to pick up four more prospects from UT Southwestern.
“The reason why we can tackle 15 programs with the option to four additional programs is we’re able to focus on what we do best and we’re able to allow our collaborators to focus on what they do best,” Session said.
Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. It’s a skill set that Taysha’s management team honed at AveXis as it developed the gene therapy that eventually became Novartis’ spinal muscular atrophy treatment Zolgensma.
“We essentially flew the plane and built it at the same time when we were developing AveXis … We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and we’re marrying that with a best-in-class academic research institution,” Session said.
Dividing the labor “creates this engine for innovation” that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsid—the protein shell of a virus—for treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Taysha’s side.
For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.
“AAV9 is the best way we have to treat monogenic CNS disease. If it’s not broke, don’t fix it,” Session said. “We know how to effectively dose AAV9; we know it’s safe, effective and efficient. And we know it’s scalable. These are problems we had to solve at our previous company.”
But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.