Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons that can correct multiple disease-causing mutations at once. Unlike ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could provide hope for ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

This year marked a pivotal moment in the quest to treat Huntington’s disease, a rare but devastating form of dementia. Scientists found that an experimental gene therapy slowed the condition’s ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

PHILADELPHIA (WPVI) -- A miracle baby who was treated with a pioneering gene-therapy at Children's Hospital of Philadelphia has reached a major milestone as he took his first steps. KJ Muldoon was the ...

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of his ...

The FDA has approved Fondazione Telethon ETS’s etuvetidigene autotemcel (Waskyra) for Wiskott–Aldrich syndrome (WAS). Etuvetidigene autotemcel is the first gene therapy to be approved for this rare ...

Fox News Digital had the most engaged news story of 2025 and tallied nine other stories in the top 100, according to a data roundup from Chartbeat. Chartbeat, a content analytics platform that ...

Pfizer Inc. (NYSE: PFE) is scheduled to provide its full-year 2026 financial guidance on Tuesday, with Bank of America Securities expecting the company's growth profile to closely resemble conditions ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...