On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
A new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond ...
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Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders
Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...
Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
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 ...
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
Encoded Therapeutics has linked its Dravet syndrome gene therapy ETX101 to a 78% reduction in seizures, keeping the biotech ...
Vertex Pharmaceuticals presents clinical data demonstrating benefits of Casgevy in SCD and TDT patients aged 5+ at ASH ...
Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
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