Panel C shows naturally occurring variants that cause hereditary persistence of fetal hemoglobin (HPFH). Single-base mutations that lead to increased γ-globin gene expression are indicated.

"Sickle cell disease is a devasting disorder that affects approximately 100,000 people in the U.S., leading to anemia, severe ...

leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal ...

Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to ...

leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal ...

This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for ...

This innovative approach aims to increase fetal hemoglobin levels as a treatment for sickle cell disease and beta thalassemia. In studies with humanized mice and non-human primates, the tLNP ...

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that ...