Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.
- FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.
- This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts.
- Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene.
- Fulcrum is also advancing FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.