A consortium to advance gene therapy for rare neurological disorders
Official title: Gene Therapy Consortium (GTC) for Gene-Based Clinical Trials Conducted within NeuroNEXT
Funded at Massachusetts General Hospital · NIH-10953878 · Data updated 2026-11-12
This study is creating a team of experts to help develop new gene therapies for very rare neurological disorders in both kids and adults, making it easier for researchers to turn their discoveries into real treatments for patients who need them.
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 | February 28, 2026 |
| Funding year | 2024 |
| Award (this year) | $296,964 |
| NIH project number | 1OT2NS138339-01 |
| Project ID | NIH-10953878 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ultra-rare neurological disorders, particularly those with a genetic basis.
✕Less likely to help
Patients with common neurological disorders or those without a genetic component may not benefit from this research.
What this research studies
This research aims to establish a Gene Therapy Consortium that will provide expert guidance on clinical trials for gene therapies targeting ultra-rare neurological disorders in both adults and children. The consortium will collaborate with various stakeholders, including government and private organizations, to streamline the development and implementation of these therapies. By focusing on innovative trial designs and supporting researchers from the planning phase through to analysis and reporting, the consortium seeks to enhance the translation of scientific advances into effective treatments for patients with limited options.
Why it matters
Potential benefit: If successful, this research could lead to the development of new gene therapies for patients suffering from ultra-rare neurological disorders, potentially offering them effective treatment options.
How similar studies have gone: Previous research has shown promise in gene therapy approaches for rare diseases, indicating that this consortium's efforts could build on existing successes.
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
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.