AAV-delivered ASC gene therapy targets schwannoma tumors
Official title: AAV-mediated delivery of the inflammasome adapter protein, ASC, as a pro-apoptotic gene therapy for schwannoma
Funded at Merlin Therapeutics INC · NIH-11401143 · Data updated 2026-11-12
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.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Merlin Therapeutics INC NIH-funded |
| Lab location | 1 site (King of Prussia, United States) |
| Project start | August 27, 2025 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,500,000 |
| NIH project number | 5R44NS145814-02 |
| Project ID | NIH-11401143 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with schwannomas, including those with NF2-related schwannomatosis or sporadic schwannomatosis, who are candidates for gene therapy approaches.
✕Less likely to help
Patients without schwannoma or those unlikely to receive effective delivery of the AAV-ASC therapy may not benefit.
What this research studies
This grant describes a gene therapy approach using an AAV vector to deliver the inflammasome adaptor ASC to schwannoma tumors. The therapy aims to selectively activate tumor cell death with a schwannian cell–specific promoter to minimize effects on healthy tissue. The project will build on prior work showing ASC delivery can target schwannomas, and will focus on safety, delivery efficiency, and proof-of-concept in relevant models. If successful, it could provide a targeted, less invasive treatment option for patients who currently rely on surgery. The work is designed to move toward clinical testing by optimizing AAV delivery and ASC expression in schwannoma cells.
Why it matters
Potential benefit: If successful, this approach could reduce tumor growth or shrink schwannomas, potentially lowering symptoms and the need for invasive surgery.
How similar studies have gone: Earlier work has shown that AAV1-delivered ASC under a Schwann-lineage promoter can target schwannomas, supporting continued development.
Where this research is happening
-
Merlin Therapeutics INC
King of Prussia, United States Active
Researchers
- Principal investigator: Yang, Na Nora — Merlin Therapeutics INC
- Study coordinator: Yang, Na Nora
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.