In gene therapy, a gene (a piece of DNA) is introduced into cells. This process uses a virus that has been stripped of its disease-causing properties—referred to as a viral vector—as the delivery ...

KJ Muldoon, the first infant to undergo gene-editing therapy, has accomplished another first as a 1-year-old — his first steps KJ is walking at home ahead of Christmas after being hospitalized last ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

Though the therapy, Kresladi, isn’t seen as a big seller, its clearance is a step forward for a company that’s lost most of ...

The regenerative medicine market is projected to reach $578 billion by 2033. Cell therapy alone surpassed $8.2 billion this year. And yet the central challenge of the entire sector remains unresolved: ...

Recent advances in gene therapy offer significant promise for patients with genetic diseases," said University of Virginia School of Medicine researcher Manoj Patel, PhD. "Instead of addressing only ...

Ocugen OCGN is pushing three retinal gene therapy programs toward late-stage milestones, with multiple data readouts and regulatory steps clustered into 2026 and early 2027. The s ...

A groundbreaking gene therapy trial has successfully restored hearing in individuals with a rare form of congenital deafness.

The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF.