A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine One of the more exciting opportunities in medical ...

Advances delivered what may feel like medical miracles, including the first bladder transplant, a lifesaving personalized ...

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 ...

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 new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

A new gene-editing therapy called Casgevy, which is designed to help treat patients suffering from sickle cell disease, has been endorsed by the National Catholic Bioethics Center and its president, ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...

Gene therapy may have the potential to cure Duchenne muscular dystrophy, but early results from clinical trials have not been as promising as researchers hoped. Research is ongoing. Duchenne muscular ...

Rocket Pharmaceuticals’ flight path to approval for Kresladi has been delayed again after the FDA issued a complete response letter (CRL) requesting additional manufacturing information. The biotech ...

A new gene-editing therapy called Casgevy, which is designed to help treat patients suffering from sickle cell disease, has been endorsed by the National Catholic Bioethics Center and its president, ...