STAT
Child dies after receiving new experimental gene therapy
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 ...
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Child dies in Phase I Capsida gene therapy trial
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 ...
FiercePharma
FDA restricts Sarepta's gene therapy Elevidys after Duchenne patient deaths
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 ...
Fierce Pharma
Sarepta gets FDA's go-ahead to study immunosuppressive Elevidys regimen in bid to reverse label restriction
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 | ...
Heartland
FDA Halts, Then Resumes Gene Therapy After Three Deaths
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 ...
HealthDay on MSN
FDA Limits Duchenne Gene Therapy After Two Teens Die of Liver Failure
Key Takeaways The FDA now limits Elevidys to walking boys ages 4 and up after two teens died from liver failureThe therapy will now carry a boxed warning for serious liver injury, liver failure and ...
Medscape
The Gene Therapy Poised to Rewrite Childhood Deafness
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...
The American Journal of Managed Care
FDA Approves Gene Therapy Onasemnogene Abeparvovec for Broader SMA Population
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 ...
The Boston Globe
Intellia tumbles after patient dies following Crispr treatment
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 ...
FierceBiotech
Capsida pauses phase 1 gene therapy trial after child dies
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 ...
Benzinga.com
Child dies after receiving new experimental gene therapy
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 ...
Benzinga.com
STAT+: Child dies after receiving new experimental gene therapy
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 ...