Gene Therapy Network: Expert Guidance for Rare Neurological Gene Treatments
Official title: Gene Therapy Consortium (GTC) for Gene-Based Clinical Trials Conducted within NeuroNEXT
Funded at Massachusetts General Hospital · NIH-11579865 · Data updated 2026-11-12
A specialized group will guide early gene therapy trials for ultra-rare neurological disorders to help adults and children who currently have few or no options.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | March 1, 2024 |
| Project end | August 14, 2027 |
| Funding year | 2026 |
| Award (this year) | $2,981,723 |
| NIH project number | 3OT2NS138339-01S3 |
| Project ID | NIH-11579865 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults and children with ultra-rare neurological disorders who lack effective approved therapies and may be eligible for gene therapy trials within NeuroNEXT.
✕Less likely to help
Patients with conditions outside the ultra-rare scope or those not yet eligible for gene therapy trials may not directly benefit.
What this research studies
This grant supports creating a specialized Gene Therapy Consortium within NeuroNEXT to provide expert advice on designing and running first-in-human gene therapy trials for ultra-rare neurological conditions. The consortium brings together experts in gene therapies, rare diseases, and clinical trial design to help researchers plan trials from conception through analysis. By coordinating with NeuroNEXT, private partners, and government partners, the GTC aims to accelerate safe and well-planned studies that could lead to new treatment options. The work focuses on pediatric and adult patients with genetic neurological diseases that lack FDA-approved therapies.
Why it matters
Potential benefit: If successful, patients with ultra-rare neurological disorders could access more carefully planned gene therapy trials and have a clearer path toward future approved treatments.
How similar studies have gone: This grant builds on existing NeuroNEXT infrastructure and the growing use of gene therapies, with anticipated cautious progression and standard regulatory steps.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Cudkowicz, Merit E — Massachusetts General Hospital
- Study coordinator: Cudkowicz, Merit E
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.