A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Scientists have successfully transplanted gene-edited insulin-producing cells into a man with type 1 diabetes—allowing him to ...

Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...

Rocket Pharmaceuticals (NASDAQ:RCKT) advances gene-therapy innovation and appears in broader discussions involving the nasdaq ...

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 ...

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

This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

NEW ORLEANS — New data from a first-in-human trial of a CRISPR-based gene therapy for cholesterol disorders are giving lipids experts reason for optimism about the approach for treating cardiovascular ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...

Opal Sandy underwent a gene therapy injection to her right ear in 2023. A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...