Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Zevaskyn, the first gene therapy for recessive dystrophic epidermolysis bullosa, showed significant wound healing in phase 3 ...
Doctors in the UK have developed a groundbreaking gene therapy using base editing to treat aggressive blood cancers once ...
The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
Dec 6 (Reuters) - Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with ...
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
On November 18, 2025, ToolGen, Inc. (“ToolGen”) filed a complaint (“Complaint”) in the District of Massachusetts alleging infringement of its ...
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