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 opened an investigation into Takeda’s recombinant protein med Adzynma following the reported death of a pediatric patient who received the drug. | The FDA has opened an investigation into ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died.

Intellia Therapeutics has acknowledged the death of the elderly patient hospitalized last week in its Phase III trial of assessing nexiguran ziclumeran (nex-z) in transthyretin amyloidosis with ...

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.

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 ...

The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and changed the indication of the drug after reports of fatal liver injury emerged ...

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 | ...

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 ...

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 ...