URMC will use funding boost to bet on Batten gene therapies

URMC among first medical centers to join NeuroNEXT

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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The University of Rochester Medical Center (URMC) has been awarded a funding boost and will expand its involvement in a national network of academic medical centers dedicated to advancing clinical trials for rare neurological conditions, such as Batten disease.

URMC was one of the first medical centers to join the Network for Excellence in Neuroscience Clinical Trials, or NeuroNEXT, which gathers academia, private foundations, and industry to respond quickly as opportunities arise to test promising new therapies.

With the funding from the National Institute of Neurological Disorders and Stroke (NINDS), which raises URMC’s total haul for the program to $5.7 million, the focus shifts to the “growing wave of gene therapies that are already revolutionizing care.”

“URMC researchers are poised to help accelerate the development of these drugs by overcoming the recruitment and retention barriers associated with rare diseases,” according to a university press release.

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Presymptomatic, symptomatic CLN5 sheep aided by gene therapy

URMC’s work in Batten disease

Partnering with Neurogene, URMC is one of two sites recruiting children with CLN5, a type of late infantile Batten, for a clinical trial (NCT05228145) testing NGN-101, an experimental gene therapy in the company’s pipeline.

The company is also running a natural history study (NCT03822650) in children and adults with CLN5 to evaluate how the disease progresses without being treated. It is still enrolling at URMC. It’s goal is to determine the most important outcomes to measure in clinical testing.

Robert Holloway, MD, chairman of the Department of Neurology, is the principal investigator responsible for the NeuroNEXT program at URMC — named UR-NEXT. Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White serve as UR-NEXT co-investigators.

The department has been at the forefront of clinical testing of gene therapies for Batten as well as other neurological diseases such as spinal muscular atrophy, Duchenne muscular dystrophy, and myotonic dystrophy.

Besides maintaining patient registries for rare diseases, URMC is home to three Clinical Trial Readiness projects, designed to help move candidate therapies toward clinical testing and increase their chance of success. One of them is in Batten.

In 2020, the University of Rochester joined 15 other centers as a National Institute of Child Health and Human Development-designated Intellectual and Developmental Disabilities Research Center, highlighting URMC’s ability to lead national efforts in translational research and clinical trials for conditions such as Batten disease.

The new funding will let URMC continue reaching out to people in the rare disease community and develop efforts to find people for clinical trials that favor diversity and meet community priorities.