On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Doctors in the UK have developed a groundbreaking gene therapy using base editing to treat aggressive blood cancers once ...
Zevaskyn, the first gene therapy for recessive dystrophic epidermolysis bullosa, showed significant wound healing in phase 3 ...
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 ...
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
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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' ...
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...
On November 18, 2025, ToolGen, Inc. (“ToolGen”) filed a complaint (“Complaint”) in the District of Massachusetts alleging infringement of its ...
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