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

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

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

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...

The status could support staged transitions to new manufacturing processes, potentially mitigating some risks of high-stakes ...

A 3-year-old boy with Hunter syndrome has shown clear improvement in his speech, cognitive, and motor skills nine months after becoming the world's first patient to receive gene therapy for the ...

Elana Thieme and Mark Mendoza, fourth-year graduate students in the Molecular and Cell Biology (MCB) PhD Program jointly ...

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

This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question ...

Oliver Chu has shown “dramatic improvements” following the groundbreaking treatment, his father Ricky Chu said ...