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

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

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

If a child is born with one defective hemoglobin-beta gene, they may become ... What is the difference between sickle cell disease and thalassemia? Both sickle cell and thalassemia are inherited ...

Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises and transfustion-dependent beta ...

Thalassemia is an inherited disease that ... symptoms thalassemia major or Cooley anemia changes on two beta-globin genes sickle trait and changes in the beta-globin gene coinheritance changes ...

Chow is City of Hope’s first sickle cell patient, while a beta thalassemia patient has been treated at Children’s National. Several authorized centers told WIRED they will begin infusions of ...

"We have about eight patients that are in the insurance approval process," she said, for gene therapy treatments for beta thalassemia and sickle cell disease. "It's something that we do expect to ...

If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or ...