Funding for HSP gene therapy

Expansion of SPG50 clinical trials

DALLAS – June 27, 2023

A consortium of government, industry, and nonprofit partners in the USA will fund gene therapy clinical trials for three different rare diseases at UT Southwestern Medical Center and Children’s Health, 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.

Dr Susan Iannaccone

At UT Southwestern and Children’s Health, the funding will expand an ongoing clinical trial on a gene therapy for spastic paraplegia type 50 (SPG50) involving two patients and being led by Susan Iannaccone, M.D., Professor of Pediatrics and Neurology. The new funds will allow the trial to include more patients at additional sites.

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SOURCE: UT Southwestern Medical Centre

Public-private consortium will fund three gene therapy clinical trials at UT Southwestern and Children’s Health

News release, June 27, 2023

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