Decoding why gene therapies can be risky and how to make them safer and more effective
Official title: Decoding the Toxicity-Efficacy Paradox to Engineer Safer, More Efficacious Gene Therapies
Funded at Arizona State University-Tempe Campus · NIH-11431272 · Data updated 2026-11-12
A five-year NIH grant at a major university aims to understand why gene therapies sometimes cause safety problems and to find ways to deliver them that are both safer and more effective for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Arizona State University-Tempe Campus NIH-funded |
| Lab location | 1 site (Scottsdale, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $378,995 |
| NIH project number | 1R35GM166587-01 |
| Project ID | NIH-11431272 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients who may ultimately receive gene therapies could benefit from safer delivery approaches developed by this program.
✕Less likely to help
Some patients might not directly benefit in the short term until safer therapies are available, depending on disease and trial readiness.
What this research studies
This grant supports a mechanism-driven research program to map how endosomal escape, inflammation, and immune responses limit the safe dosing of gene therapies. The team will build an integrated atlas of the toxic pathways and test ideas to reduce harmful side effects while preserving therapeutic impact. The work combines studies of delivery biology with analyses of how cells and the immune system react to genetic cargo, using a flexible, iterative approach. If successful, the findings could guide safer dosing strategies and new design principles for future therapies. The project is planned over five years at Arizona State University with broad potential relevance to patients who may benefit from gene-based treatments.
Why it matters
Potential benefit: Could lead to safer, more effective gene therapies for patients by identifying ways to minimize toxicity while preserving therapeutic benefits.
How similar studies have gone: There is a history of ongoing efforts to address gene therapy safety, but detailed mechanisms of escape-induced toxicity are still not fully defined, making this a novel and potentially impactful direction.
Where this research is happening
-
Arizona State University-Tempe Campus
Scottsdale, United States Active
Researchers
- Principal investigator: D'arcy, Richard — Arizona State University-Tempe Campus
- Study coordinator: D'arcy, Richard
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.