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 ...
Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
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.
A pioneering treatment that edits healthy immune cells to fight cancer has shown promise for adults and children with a rare ...
New three-year follow-up results from the TRANSCEND FL trial show that patients with relapsed or refractory follicular ...
A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Bispecific antibodies bring multiple tumor-cell-killing mechanisms into play simultaneously. When a T cell is activated and ...
Data presented at ASH suggest that the CRISPR therapy could provide a one-time functional cure in children as young as 5 ...
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