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

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

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

Victoria Gray is the first person in the world to receive CRISPR,  a gene-editing therapy for sickle cell disease created by ...

Victoria Gray, the first person cured of sickle cell using CRISPR gene editing therapies, spoke in Fargo about her journey ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

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

Oregon Health Plan (OHP) members will see increased access to lower-cost, lifesaving cell and gene therapies. The state is ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

A renowned Harvard hematologist, Maureen Okam Achebe, has called for the institutionalisation of continuous newborn screening ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the ...

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