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 ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

BEAM-101, a genetically modified cell therapy for sickle cell, is designed to turn on HbF to counteract mutated adult ...

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 ...