Public-Private Consortium will Fund Three Gene Therapy Clinical Trials at UT Southwestern

Steven Gray, PhD

A consortium of government, industry, and nonprofit partners will fund gene therapy clinical trials for three rare diseases at UT Southwestern Medical Center, where scientists are working on gene therapies to treat neurodevelopmental disorders in children.

Testing on a total of eight genetic diseases was approved for funding by the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership among the National Institutes of Health (NIH), the Food and Drug Administration (FDA), biopharmaceutical and life science companies, and other organizations. Launched in 2021, BGTC aims to create a “playbook” to standardize and streamline the process of developing gene therapies and getting them to patients – an effort that could significantly accelerate the process and lower costs.

“There are over 10,000 rare diseases that affect millions of people worldwide, and most of them are genetic. Because the vast majority of these diseases affect just a small number of patients, they’re viewed as having minimal commercial interest,” said Steven Gray, PhD, Associate Professor of Pediatrics, Molecular Biology, and Neurology in the Eugene McDermott Center for Human Growth and Development and Director of UTSW’s Translational Gene Therapy Core. “By creating an open-access playbook for how to initiate and carry out gene therapy clinical trials for these types of rare diseases, BGTC will make this process substantially easier.”