NINDS initiative will fast-track gene therapy for ultra-rare diseases

The National Institute of Neurological Disorders and Stroke (NINDS) is enhancing its Ultra-Rare Gene-based Therapy (URGenT) network to accelerate the development and clinical delivery of gene-based therapies for ultra-rare neurological disorders, according to a poster presented at the 2024 International Congress of Parkinson’s Disease and Movement.

By integrating resources and support through partnerships with the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) network, NINDS aims to facilitate a smoother transition from preclinical research to clinical trials, ultimately improving access to innovative treatments for patients with rare diseases.

Established in 2021, URGenT aims to support late-stage pre-clinical development and facilitate clinical trials for gene-targeted treatments.

Rare diseases are often rooted in genetic anomalies, with movement disorders among the most common classifications. The expanded URGenT network now collaborates with the NeuroNEXT to enhance resources and guidance for clinical trial challenges specific to gene therapy.

Currently, 4 pre-clinical projects are underway, focusing on therapies for Menkes disease, prion disease, amyotrophic lateral sclerosis (ALS), and aspartylglucosaminuria. NINDS has also issued a Research Opportunity Announcement (ROA), inviting broader clinical trial applications beyond those developed through URGenT.

Reference
Cho HJ. Videnovic M. Cudkowicz, et al. The NINDS Approach to Therapeutic Development for Ultra-Rare Neurological Disorders, Bridging Translational and Clinical Frontiers in Gene-based Therapy [abstract]. Mov Disord. 2024; 39 (suppl 1). https://www.mdsabstracts.org/abstract/the-ninds-approach-to-therapeutic-development-for-ultra-rare-neurological-disorders-bridging-translational-and-clinical-frontiers-in-gene-based-therapy/. Accessed September 27, 2024.