Before the therapy, the affected children were legally blind, only able to distinguish between light and dark. However, after ...

Intellia Therapeutics’ stock faces risks from market uncertainty and funding needs. Read why NTLA stock is downgraded to HOLD ...

A woman with a rare muscle-weakening disorder has become one of the first patients in the world to take part in a trial of a ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

Artificial intelligence is making waves across industries, but its impact is higher in some sectors than others. Medicine and other sciences stand to gain much from this technology, thanks to their ...

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw improvements ...

On the very day Noa was diagnosed, an experimental gene therapy and clinical trial were posted to treat Canavan disease. Noa was only the third patient in the world to be dosed with the gene therapy, ...

The Novato-based maker of biopharmaceutical treatments for rare diseases plans to release this year a new gene therapy for ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...

A new study led by Profs. Fan Kelong and Yan Xiyun from the Institute of Biophysics of the Chinese Academy of Sciences ...

Columnist Jennifer Lynne assesses the effects of Pfizer's decision to halt production of its hemophilia B gene therapy, Beqvez.

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...