UniQure needs to run another study to prove its gene therapy "actually helps people with Huntington's disease," a senior FDA official said. The official said UniQure is the "latest company to make a ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial results. The drug, called zorevunersen, holds promise, especially for people ...

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate. The agreement, ...

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

In this video, gameplay from Granny Simulator is featured, where Aphmau takes on the role of an evil Granny. The objective is to navigate the challenges posed by her character and attempt to stop her ...

A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...

In 2018, a nervous-looking He Jiankui took the stage at a scientific conference in Hong Kong. A hush settled over the packed auditorium as the soft-spoken Chinese scientist adjusted his microphone and ...

Here’s how extinct DNA could help us in the present—and the future. Yeah, we know—it’s not a dire wolf. In early 2025, the Texas biotech company Colossal Biosciences landed with a splash on the cover ...

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 ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...