Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Capsida Biotherapeutics has paused an early-stage rare disease trial evaluating its lead gene therapy candidate, CAP-002, after the death of the first patient dosed in the study. The death comes just ...

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 ...

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 ...

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 ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) ...

When the FDA reworked the prescribing information for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy Elevidys earlier this month, the company touted a plan to study a regimen | ...

Capsida Biotherapeutics has paused a gene therapy trial after the first patient died. The biotech, which began the phase 1/2 study in July, took the action to give it time to determine the “root cause ...

The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday. The company, Capsida ...

The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday. The company, Capsida ...