Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, describe how optogenetic control of brain cells and circuits is already steering ...

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 ...

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

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 ...

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.

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 ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

Eye drug maker Ocuphire Pharma is acquiring gene therapy developer Opus Genetics in an all-stock transaction that will see the commercial-stage company adopt the biotech’s identity. The resulting ...