To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...

This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

A new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond ...

Despite a challenging CASGEVY launch, high cash burn, and an almost 10% YTD share count growth, CRISPR’s pipeline breadth and ...