Tools to increase fetal hemoglobin by targeting BCL11A
Chemical tools for modulating the fetal hemoglobin inducer BCL11A
Developing new chemical drugs to increase protective fetal hemoglobin for people with sickle cell disease and beta-thalassemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-11324633 on NIH RePORTER |
What this research studies
Researchers are creating small molecules that can bind to and remove BCL11A, a protein that turns off fetal hemoglobin in adults. They will design compounds such as PROTACs and molecular glues and test them in lab-grown human cells and animal models. The team focuses on finding drug-like molecules that can work against a protein that has been hard to target with traditional medicines. Promising compounds could later move toward safety testing and clinical trials for patients.
Who could benefit from this research
Good fit: Adults with sickle cell disease or beta-thalassemia would be the likely future candidates for therapies that come from this work.
Not a fit: People with unrelated causes of anemia or those needing immediate clinical treatment are unlikely to receive direct benefit from this early laboratory-focused research.
Why it matters
Potential benefit: Could raise fetal hemoglobin levels and reduce anemia, pain, and other complications for people with sickle cell disease and beta-thalassemia.
How similar studies have performed: Gene-editing and other approaches that lower BCL11A have helped some patients, but using small-molecule degraders like PROTACs against BCL11A is a new and largely untested strategy.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Dassama, Laura — Stanford University
- Study coordinator: Dassama, Laura
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.