New gene therapy for treating Neurofibromatosis Type 2
Official title: Development of a Novel AAV9 Gene Replacement Therapy for Neurofibromatosis Type 2
Funded at Merlin Therapeutics INC · NIH-10818151 · Data updated 2026-11-12
This study is testing a new gene therapy that aims to help people with Neurofibromatosis Type 2 (NF2) by delivering a healthy version of a gene that can prevent tumor growth, with the hope of easing symptoms and improving treatment options for those who currently have no approved therapies.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Merlin Therapeutics INC NIH-funded |
| Lab location | 1 site (King of Prussia, United States) |
| Project start | August 1, 2024 |
| Project end | December 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $699,559 |
| NIH project number | 1R41NS135883-01 |
| Project ID | NIH-10818151 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Neurofibromatosis Type 2 who are experiencing symptoms related to tumor growth in the nervous system.
✕Less likely to help
Patients with other forms of neurofibromatosis or those without a confirmed diagnosis of Neurofibromatosis Type 2 may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy using an adeno-associated viral vector (AAV9) to deliver a healthy copy of the NF2 gene, which is crucial for preventing tumor growth in patients with Neurofibromatosis Type 2 (NF2). The therapy aims to express the Merlin protein in all affected cell types, potentially reversing the debilitating symptoms caused by tumor growth in the nervous system. By utilizing advanced vector technology, the researchers hope to provide a more effective treatment option for patients who currently have no FDA-approved therapies available. The approach has shown promise in preliminary studies, indicating its potential efficacy in addressing the underlying genetic cause of NF2.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that alleviates the debilitating symptoms of Neurofibromatosis Type 2 and improves patients' quality of life.
How similar studies have gone: While gene therapy approaches for other conditions have shown success, this specific application for Neurofibromatosis Type 2 is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Merlin Therapeutics INC
King of Prussia, United States Active
Researchers
- Principal investigator: Henwood, Nicole — Merlin Therapeutics INC
- Study coordinator: Henwood, Nicole
Related research
Other NIH-funded projects on the same topics.
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AAV-delivered ASC gene therapy targets schwannoma tumors
A tiny virus (AAV) would deliver a gene called ASC directly into schwannoma tumors to trigger tumor cell death and help people with NF2-related schwannomatosis or schwannomatosis.
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.