Developing new treatments to increase fetal hemoglobin for sickle cell disease
Official title: Targeted protein degraders for the treatment of b-hemoglobinopathies
Funded at Yale University · NIH-11019948 · Data updated 2026-11-12
This study is working on new medications that can help increase a type of hemoglobin in your blood to better manage Sickle Cell Disease, aiming to provide a more reliable and safer treatment option than what’s currently available.
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 | 2025 |
| Award (this year) | $765,622 |
| NIH project number | 1R01HL176850-01 |
| Project ID | NIH-11019948 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Sickle Cell Disease, particularly those who have not responded well to current therapies.
✕Less likely to help
Patients with other forms of hemoglobinopathies or those who do not have Sickle Cell Disease may not benefit from this research.
What this research studies
This research focuses on creating new small molecule drugs that can increase levels of fetal hemoglobin (HbF) to help treat Sickle Cell Disease (SCD). Current treatments like hydroxyurea can be inconsistent in their effectiveness, so this project aims to develop targeted protein degraders that can more reliably elevate HbF levels. By using innovative approaches, including the degradation of specific gene repressors, the research seeks to provide a safer and more effective alternative to existing therapies. Patients may benefit from improved management of their condition and reduced complications associated with SCD.
Why it matters
Potential benefit: If successful, this research could lead to more effective and accessible treatments for patients with Sickle Cell Disease.
How similar studies have gone: Other research has shown promise in using targeted therapies to increase fetal hemoglobin levels, indicating that this approach may be viable.
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
Related research
Other NIH-funded projects on the same topics.
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In utero gene editing for sickle cell disease and alpha-thalassemia using lipid nanoparticles
A prenatal therapy using specially designed lipid nanoparticles to edit genes in fetal blood-forming cells could prevent sickle cell disease and alpha-thalassemia before symptoms start.
-
Targeting blood stem cells with tiny fat bubbles to treat inherited blood diseases
A new approach uses targeted lipid nanoparticles to temporarily reduce certain blood-forming stem cells, paving the way for safer stem cell transplants for conditions like sickle cell and thalassemia.
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In vivo gene therapy for blood diseases using multiplexed virus-like particles
A new approach aims to repair patients’ own blood stem cells inside their bodies to treat genetic blood diseases.
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Oral protein-targeting drugs to raise fetal hemoglobin for sickle cell disease
Testing new oral medicines that remove proteins blocking fetal hemoglobin to help adults with sickle cell disease reduce red blood cell sickling.
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Using nurses to provide genetic counseling for sickle cell disease in primary care.
This study is looking at how training nurses to provide genetic counseling and carrier screening for sickle cell disease can help families in Sierra Leone make informed choices about having children, especially since there aren't many genetic counselors available in the area.
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HOPE Biobank: connecting blood disease patients through stem cell transplant and gene therapy samples
This study will collect blood and other biospecimens along with clinical data from people undergoing stem cell transplant or gene therapy for non-cancer blood diseases to build a large biobank for future research.
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.