Beam reports breakthrough results from BEAM-101's early-stage trial for severe sickle cell disease. Read more here.

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in the ...

Two Italian women in their twenties on Tuesday received pioneering gene therapy for rare hereditary blood diseases in Pavia ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

The chance to break free from the cycle of pain, even if it meant trading one set of uncertainties for another, became a compelling reason for Gray to embrace gene therapy. Sickle cell disease, also ...

However, recent advances in gene therapy may eventually lead to a cure for sickle cell disease. Currently, the only treatment is a risky and costly bone marrow transplant. In a half-dozen clinical ...

for sickle cell disease (SCD) and beta-thalassemia. The approval was a landmark in the quest to design gene-editing therapies with curative potential. Casgevy shows exceptional efficacy ...

Sickle cell disease is a genetic, lifelong condition that affects red blood cells, causing them to become sickle-shaped and block blood flow to other parts of the body. This disease is most seen ...

becoming the first New Yorker cured of sickle cell anemia. According to a PIX11 news report, Sebastien, who's dealt with the disease since infancy, now lives without pain. "Since gene therapy, I ...