This year, headlines in the cell and gene therapy landscape have so far been dominated by Sarepta. The biotech’s Duchenne muscular dystrophy treatment Elevidys (delandistrogene moxeparvovec) became ...

The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life.

Patients with severe sickle cell disease experienced significant quality of life improvements after receiving exagamglogene autotemcel gene therapy.

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients ...

The first gene therapy treatment for sickle cell disease has been approved in the U.K. , a move that could offer relief to thousands of people with the disease.

“This therapy is intended to eliminate sickle cell disease by applying CRISPR technology that is safer than a standard stem cell transplant from a healthy bone marrow donor,” said Mark Walters, M.D., ...

LONDON (AP) — Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the ...

London — Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the ...

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.

MSK is the first hospital in New York City to offer gene therapy through its pediatric cancer program MSK Kids. Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell ...