Taysha Gene Therapies just emerged out of stealth mode with a $30 million seed round and a strategic partnership with UT Southwestern. The gene therapy-based spinoff specializes in the addressing of diseases caused by a variation in a gene known as monogenic CNS disease. With up to 15 candidates in the pipeline, they are planning up to four investigational new drug (IND) applications.
Who is Taysha Gene Therapies?
Founded in 2020, this brand-new venture is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, they purport to be able to rapidly translate their treatments from bench to bedside. They position their team’s combined experience in gene therapy, drug development and commercialization with the world-class UT Southwestern Gene Therapy Program enabling them to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market infections. Together they leverage their fully integrated platform or what they called an “engine for new cures” to dramatically improve patients’ lives.
Involving previous investors and executives from AveXis (acquired by Novartis), the group has been intensely collaborating with UT Southwestern Gene Therapy Program and the Department of Pediatrics. They are led by CEO RA Session II. In a recent BioPharma Dive interview, he commented, “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.”
Partnership with UT Southwestern
Apparently, the venture inked a strategic alliance with UT Southwestern to translate novel AAV gene therapies in a quest for commercialization. Led by Stephen Gray, PhD, Director of the Viral Vector Southwestern Gene Therapy Program, as well as Berge Minassian, MD, Division Chief of Child Neurology, UT Southwestern has geared up a scalable GMP-compliant viral vector production facility to support Taysha’s preclinical and clinical research initiatives.
The company just completed its large seed funding in just as the Dow “dropped a couple thousand points,” reported EndPoints. The company reports that they have a healthy pipeline of at least 15 candidates focused on adeno-associated virus gene therapy—with options to at least four others. According to reports, the company plans four Investigational New Drug (IND) applications by 2021. They are focusing their attention on an investigational product called TGTX-101, a gene replacement therapy for GM2-Gangliosidosis—this should be associated with previously mentioned first batch INDs. Another experimental product is called SLC6A1 addressing haploinsufficiency, Rett syndrome and SURF1 deficiency.