Abstract
The NeuroNEXT Gene Therapy Consortium: Designing and Advising on Quality Gene Therapy Trials in Rare and Ultra-rare Neurological Disorders
Neurotherapeutics, Vol.22(4), e00645
07/2025
DOI: 10.1016/j.neurot.2025.e00645
Abstract
The NeuroNEXT network for Excellence in Neuroscience Clinical Trials was initially funded by NINDS in 2011, and funded for its third cycle in 2023, with the goal of increasing efficiency and accelerating trials in promising new therapies across the field of neurology. As part of the 2023 funding cycle, the NeuroNEXT Gene Therapy Consortium (GTC) was established to provide expertise and guidance for proposed clinical neurology trials utilizing gene-targeted treatments. The GTC consists of support from the NeuroNEXT clinical and data coordinating centers for overall study design and statistical expertise, and 9 members with professional expertise in clinical and pre-clinical gene therapy development, IND and regulatory, natural history studies, ultra-rare disorder treatment, patient advocacy, and outcome measures. They work directly with applicants referred from NINDS who are interested in applying for grants to test promising gene-based therapies and provide expertise, including projects developed from research in the NINDS Ultra-Rare Gene-based Therapy (URGenT) network, which supports late-stage pre-clinical development of gene-based therapies for ultra-rare neurological diseases. The NeuroNEXT GTC provides guidance on potential challenges related to safety and efficacy research, regulatory approval, and business processes unique for gene-based therapy trials. The application process has been designed to move quickly from initial idea through approval to first patient enrolled. Application and protocol reviews are rolling and iterative, thereby maximizing the speed with which promising trials can be launched. The GTC plans to contribute to many trials studying promising gene therapy treatments over the remaining years of the NeuroNEXT network funding period.
Details
- Title: Subtitle
- The NeuroNEXT Gene Therapy Consortium: Designing and Advising on Quality Gene Therapy Trials in Rare and Ultra-rare Neurological Disorders
- Creators
- David Klements - Massachusetts General HospitalMarianne Chase - Massachusetts General HospitalHyun Joo (Sophie) Cho - National Institutes of HealthAleksander Videnovic - Massachusetts General HospitalChristopher Coffey - University of IowaDixie Ecklund - University of IowaMerit Cudkowicz - Massachusetts General Hospital
- Resource Type
- Abstract
- Publication Details
- Neurotherapeutics, Vol.22(4), e00645
- DOI
- 10.1016/j.neurot.2025.e00645
- ISSN
- 1878-7479
- eISSN
- 1878-7479
- Publisher
- Elsevier Inc
- Language
- English
- Date published
- 07/2025
- Academic Unit
- Biostatistics
- Record Identifier
- 9984948006802771
Metrics
4 Record Views