Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a ...

Scientists at the MRC Laboratory of Medical Sciences (LMS) have discovered a DNA-based “dimmer switch” that regulates the activity of a critical developmental gene, Cdx2. This work could pave the way ...

The Breakthrough Prize Foundation awarded researchers involved in developing gene therapies for blindness and sickle cell ...

For years, researchers have been trying to figure out how to treat inherited blood disorders like sickle cell disease without causing new health problems in the process. Now, a team of scientists from ...

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

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 ...

For the second time, Astellas is walking away from Taysha Gene Therapies, this time choosing not to license the biotech’s adeno-associated-virus-based therapy designed to treat a rare ...

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