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

CRISPR Therapeutics recently drew attention as updates highlighted progress in its gene-editing pipeline, including early-stage studies of CTX310, an in vivo therapy designed to lower LDL cholesterol ...

The global cell and gene therapy market size is valued at USD 27.02 billion in 2025 and is predicted to hit around USD 232.22 ...

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

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

Almost two thirds of patients with T-cell acute lymphoblastic leukaemia involved in a clinical trial of the treatment are now ...

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

Of the 11 patients treated with BE-CAR7 T cells, seven are in remission, including one patient who has been in remission for three years.

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...