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

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Gene therapy ‘switch’ may offer non-addictive pain relief. New approach targets pain signals while leaving the rest of the brain untouched.

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in ...

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 ...

As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch ...

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

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 ...

An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...

The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14. The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino ...