Encore: speeding up finding new uses for existing drugs to help kids with rare diseases
Official title: Transforming therapeutic discovery for rare diseases with Encore
Funded at Broad Institute, INC. · NIH-11405200 · Data updated 2026-11-12
A big NIH project will test thousands of existing medicines to see if any can help treat rare genetic diseases in children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | September 15, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $1,910,368 |
| NIH project number | 1R01HL193392-01 |
| Project ID | NIH-11405200 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young people with rare genetic diseases who currently have limited or no approved treatments.
✕Less likely to help
Some diseases or drug candidates may not respond or be suitable for repurposing, so clinicians may not find a benefit for every condition.
What this research studies
This project will create a high-throughput system that screens over 1.6 million drug/disease combinations to find potential new therapies for many rare diseases. It will also use patient-relevant genetic information to virtually screen about 29 million drug/variant combinations. The goal is to create a practical, faster way to repurpose medicines that already exist, potentially benefiting children and families affected by rare conditions. The work combines advanced algorithms, computer vision-inspired analysis, and robust phenotyping to identify promising drug candidates for further testing. If successful, it could streamline bringing effective treatments to patients who currently have few or no options.
Why it matters
Potential benefit: Could dramatically expand treatment options for children with rare diseases by repurposing existing drugs.
How similar studies have gone: This approach builds on prior work in drug repurposing and high-throughput screening, with a planned massively parallel implementation that is novel in scale.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Neal, James Thomas — Broad Institute, INC.
- Study coordinator: Neal, James Thomas
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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.