A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five ...

The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

A breakthrough therapy is driving aggressive leukaemia into remission. Here’s what the headlines don’t tell you about life ...

The three-hit model, developed from more than a decade of systems biology research, proposes that autism develops when three ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

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Researchers explored this question at ASH and shared data suggesting genomic instability in tumors with high TMB likely ...

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...

Initial safety results for the full cohort of 19 indicated that the treatment was well tolerated. The only notable adverse ...