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.

Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...

21-year-old Sebastien Beauzile from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell anemia using the innovative Lyfgenia gene therapy. This ...

Though Beauzile is the first in New York to successfully undergo sickle cell anemia-curing gene therapy, he's preceded by Kendric Cromer, a 12-year-old boy from Washington, DC who received ...

But instead, he had become so weak from sickle cell anemia that he had to be carried ... after undergoing Lyfgenia, a new type of gene therapy that uses stem cell technology to transform his ...

“Since gene therapy, I haven’t had any pain ... who currently suffer from sickle cell anemia.