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

The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

Two recent studies show the novel therapy works in people with multiple myeloma, but researchers are trying to minimize side ...

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

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 ...

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 ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike to ...

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 ...

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