A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and getting ready to spend Christmas at home after being hospitalized last ...

A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene editing treatment at CHOP.

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

The boy was born with the rare disease that would gradually take away his cognitive and motor function without treatment.

At ASH 2025, investigators reported early results from the first-in-human phase 1 inMMyCAR study evaluating KLN-1010, an ...

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 ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

Started in 2004 by a Duke University offensive lineman, Teamworks today says its software is used by more than 6,500 sports ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

A long-term, trust-based CDMO partnership can support cell and gene therapy developers from early process development through GMP manufacturing and commercial supply.

AMD Phase 3 timing, Otsuka funding to 2028, and mixed 4D-710 data with financial risk. See why FDMT stock is a hold.