Oral protein-targeting drugs to raise fetal hemoglobin for sickle cell disease
Official title: Targeted protein degraders for the treatment of b-hemoglobinopathies
Funded at Yale University · NIH-11252565 · Data updated 2026-11-12
Testing new oral medicines that remove proteins blocking fetal hemoglobin to help adults with sickle cell disease reduce red blood cell sickling.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project start | January 1, 2025 |
| Project end | November 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $729,938 |
| NIH project number | 5R01HL176850-02 |
| Project ID | NIH-11252565 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults aged 21 and older with sickle cell disease who still experience sickling or painful crises despite current treatments are the likely candidates.
✕Less likely to help
People under 21, those with other types of hemoglobin disorders not driven by fetal hemoglobin suppression, or patients already cured by transplant or gene therapy are unlikely to benefit from this specific work.
What this research studies
This project aims to create oral small-molecule drugs (protein degraders) that target two proteins that normally keep fetal hemoglobin switched off. Researchers will test these compounds in lab-grown human blood cells and in animal models to see whether fetal hemoglobin (HbF) levels go up and sickling goes down. The teams will optimize the compounds for oral availability and study safety, bioavailability, and dosing in preclinical assays. If promising, they plan steps to move the best candidates toward clinical testing in people.
Why it matters
Potential benefit: If successful, these oral drugs could raise fetal hemoglobin to reduce red blood cell sickling and painful crises, offering a less invasive and more affordable option than gene therapy.
How similar studies have gone: Protein degraders are a new class of drugs with early successes in other diseases, but using them to boost fetal hemoglobin for sickle cell disease is largely novel and currently supported mainly by preclinical data.
Where this research is happening
-
Yale University
New Haven, United States Active
Researchers
- Principal investigator: Crews, Craig M — Yale University
- Study coordinator: Crews, Craig M
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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.