John Maraganore, the former CEO of Alnylam Pharmaceuticals, launched City Therapeutics, a new startup with $135 million from ...

Upstream Bio has swollen its IPO to $255 million as the company joins CAMP4 Therapeutics this morning in becoming the latest ...

A Bluebird Bio gene therapy has helped children with a devastating neurological disorder, but seven have developed blood ...

Researchers will develop therapies for urea cycle disorders and create a platform to rapidly develop treatments for other rare genetic disorders.

A new technology enables the control of specific brain circuits non-invasively with magnetic fields, according to a ...

A new gene drive can copy and paste itself into the genomes of herpes simplex viruses in mice. The end goal is a version that disables the virus in humans.

Since the FDA cleared Sarepta Thereapeutics' gene therapy for nearly all people with Duchenne, its stock price has tanked.

Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new ...

Booster Therapeutics has launched with $15 million in seed financing and a mission to take on the current crop of targeted ...

Despite previous attempts to treat these diseases through gene therapy, success has been limited due to immune responses to current therapies. Prime editing could change this by enabling permanent ...