The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits ...

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a win for a biotech that has struggled in recent months under the weight of a ...

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

Scientists studying axolotls, zebrafish, and mice have uncovered a shared set of genes that may one day help humans regrow ...

CRISPR-edited tumor-infiltrating lymphocytes are emerging as a promising alternative to CAR T-cell therapy for solid cancers.

Saffie Sandford was born with Leber's Congenital Amaurosis and "missed out on a lot" due to difficulties seeing in any ...

Researchers at the Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah, and the University of Utah School of Medicine, have demonstrated that a gene therapy can ...

As other global companies race to find new gene therapies, Regeneron plans to make the new drug free for those in the US ...