NINDS $22.8 Million Grant to Fund Gene Editing-Based Therapeutic Development for Four, Rare Neurologic Conditions

Rare Daily Staff

The National Institute of Neurological Disorders and Stroke has issued a five-year, $22.8 Million grant to a group of institutions led by The Jackson Laboratory Rare Disease Translational Center to develop and validate new gene editing-based therapeutic approaches for four neurological conditions.

The groups will seek to advance at least one candidate through preclinical development to an application to conduct human clinical trials. The conditions included effort are spinal muscular atrophy, Friedreich’s ataxia, Huntington’s disease, and Rett syndrome.

One of the new therapeutic approaches of the preclinical genome editing project will be prime editing, a “search-and-replace” genome editing technology that provides the ability to repair DNA without first inducing DNA breaks.

“Our goal is to remove or move past the obstacles, bring a highly promising new therapy strategy to the clinic, and directly benefit individuals with these diseases,” said Cat Lutz, vice president of The Jackson Laboratory Rare Disease Translational Center, who is leading the multi-institutional team, which included Broad Institute, Massachusetts General Hospital, Boston Children’s Hospital and UT Southwestern Medical Center.

Though NINDS focuses on neurological diseases, the research could have implications across the rare disease community and the estimated 10,000 rare genetic diseases that affect patients worldwide.

Lutz will work with associate Professor Steven Murray, who leads the preclinical mouse model core at JAX, to develop, validate, and optimize in vivo mouse models for each disease. Other collaborators have experience and resources for producing virus-based gene-editing therapy delivery to tissues, possess deep expertise in preclinical evaluation of gene-editing therapeutics, and have successfully navigated the regulatory path to IND submission.

Collectively, they said they seek to close the gap between preclinical research that has produced promising rare disease therapy strategies and the actual clinical delivery of safe and effective treatments to patients.

They believe successful completion of the project milestones has the potential to revolutionize treatment for at least one of the diseases and provide a way forward for the other three, as well as a wide range of other rare genetic diseases.

“There are many obstacles to developing safe, effective treatments for rare diseases,” said Lutz, “including small patient populations, high costs, and regulatory barriers. Our goal is to remove or move past the obstacles, bring a highly promising new therapy strategy to the clinic, and directly benefit individuals with these diseases.”