Developing new treatments to increase fetal hemoglobin for sickle cell disease
Targeted protein degraders for the treatment of b-hemoglobinopathies
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 ID | NIH-11019948 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Sickle Cell Disease, particularly those who have not responded well to current therapies.
Not a fit: Patients with other forms of hemoglobinopathies or those who do not have Sickle Cell Disease may not benefit from this research.
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 performed: 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
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Crews, Craig M — Yale University
- Study coordinator: Crews, Craig M
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 below.