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

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

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

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

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor ...

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

Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The toddler has now exceeded doctors' expectations after becoming the first ...

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

The New York Blood Center (NYBC) opened its first-ever donor center based in Queens, marking a milestone for the organization ...

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 ...