AI-powered chemical proteomics to discover drugs for orphan proteins
Official title: AI-powered chemical proteomics for drug discovery targeting orphan proteins
Funded at Northeastern University · NIH-11389526 · Data updated 2026-11-12
A scientist team at Northeastern University is testing new AI-enabled methods to find drug-like compounds that can target hard-to-reach,
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Northeastern University NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | August 15, 2017 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $351,850 |
| NIH project number | 5R01GM122845-09 |
| Project ID | NIH-11389526 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with diseases linked to orphan proteins or those contributing patient samples for research could participate in data sharing or biomarker studies; specific eligibility will be defined by the clinical collaborators.
✕Less likely to help
Some patients may not benefit directly if their condition is not connected to the targeted orphan proteins or if candidate drugs do not advance to clinical testing.
What this research studies
A Northeastern University team led by Lei Xie is developing AI-assisted chemical proteomics to map how small molecules interact with so-called orphan proteins and to connect these interactions to their effects in the body. The project aims to overcome data gaps by integrating genome-wide data, high-throughput experiments, and advanced AI to predict which compounds could become safe, effective drugs for diseases driven by undrugged targets. If successful, this work could accelerate the discovery of new therapies for conditions with few treatment options, by revealing which compounds activate or inhibit specific proteins and how those actions relate to clinical outcomes.
Why it matters
Potential benefit: If successful, patients could gain access to new drug candidates for underserved or rare diseases linked to orphan proteins.
How similar studies have gone: There is growing interest in AI-driven proteomics for drug discovery, but applying it to orphan proteins remains challenging and is relatively novel.
Where this research is happening
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Northeastern University
Boston, United States Active
Researchers
- Principal investigator: Xie, Lei — Northeastern University
- Study coordinator: Xie, Lei
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.