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.

Drexel faculty researchers working on the cutting edge of cell and gene therapy shared insights and information about their groundbreaking work at the Cell & Gene Therapy Symposium held in May 2023.

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

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., ...

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

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 ...

Mice treated with the gene therapy remained cancer-free for at least 100 days and resisted cancer resurgence in a lab test.