SplicingModulators to Target Hard-to-Drug Genes in Cancer and Neurodegeneration
Official title: Development of splicing modulators and their combinations to target undruggable genes
Funded at University of Chicago · NIH-11439248 · Data updated 2026-11-12
A small set of compounds that adjust RNA splicing are being studied to turn off disease-driving genes that are hard to target with traditional drugs.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Chicago NIH-funded |
| Lab location | 1 site (Chicago, United States) |
| Project start | August 1, 2026 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $451,000 |
| NIH project number | 1R21TR006398-01 |
| Project ID | NIH-11439248 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients whose diseases involve undruggable transcription factors or disordered proteins, including certain leukemias and neurodegenerative conditions, may benefit from future applications of these modulators.
✕Less likely to help
If a condition does not involve cryptic splice sites or relies on different disease mechanisms, the modulators may not provide benefit.
What this research studies
This project tests tiny molecules that change how RNA is processed in human cells, aiming to turn off harmful genes by triggering their molecular decay. Researchers will explore two types of modulators: one that directly stabilizes RNA processing at weak splice sites and another that tweaks other splicing factors to enhance effects. Early results show promise in downregulating disease-causing genes like PRNP and transcription factors linked to cancer and neurodegenerative diseases. The work includes creating lead compounds and testing their combinations to see if they can safely reduce problematic gene activity. If successful, this could open new avenues for treating conditions where current therapies are limited.
Why it matters
Potential benefit: If the approach works, patients with diseases driven by challenging genes could gain new therapeutic options.
How similar studies have gone: Preliminary data show that combining driver and helper modulators can downregulate disease-driving factors, with early proof-of-concept results.
Where this research is happening
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University of Chicago
Chicago, United States Active
Researchers
- Principal investigator: Wang, Jingxin — University of Chicago
- Study coordinator: Wang, Jingxin
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.