The investigational therapy aims to restore normal RBC function through increasing levels of hemoglobin F (HbF) by increasing γ-globin protein levels (Fig. 1). γ-globin is a subunit of fetal ...

New data from a Phase 1b clinical trial testing Fulcrum's pociredir for sickle cell disease are expected by the middle and ...

MaxCyte Inc. (NASDAQ: MXCT) is a global developer of next-generation cell therapies. They provide crucial technology for cell ...

The trial is testing base editing—the most precise form of gene therapy yet developed—as a way to boost fetal hemoglobin production. In contrast to conventional gene editing strategies that ...

YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the ...

Fetal Hemoglobin Bart's Disease is a severe form of alpha thalassemia that can lead to significant complications during pregnancy, including fetal anemia and hypoxia. This condition arises from ...

Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the company will encore data from the BEACON Phase 1/2 ...

Ten days of subcutaneous Dacogen resulted in responses among a third of patients with myelofibrosis. Among patients with ...