Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

Acute liver injury has been associated with the death of two non-ambulatory DMD patients treated with Elevidys.

Intellia Therapeutics temporarily paused dosing and screening for its phase 3 Magnitude and Magnitude-2 trials of Nex-z following a patient hospitalization, according to an Oct. 27 news release. The ...

Milestone Pharmaceuticals won FDA approval for a new nasal-spray medicine to treat episodes of paroxysmal supraventricular ...

Intellia Therapeutics Inc. shares dropped after the company said a patient died after suffering liver damage in a clinical trial for the company’s gene-editing treatment. The patient, who was in his ...

After the deaths of three patients, the maker of an investigational gene therapy stopped shipments of the drug after it refused a request from the U.S. Food and Drug Administration (FDA) to pull the ...

After spending over 300 days at the Children's Hospital of Philadelphia (CHOP), 9-month-old KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene editing therapy.

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

Etranacogene dezaparvovec meets the benchmarks for success, researcher says ...