First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...
Nature

CRISPR vs cholesterol: can gene editing prevent heart disease?

Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...
Fierce Biotech

Patient dies after receiving Intellia's CRISPR therapy

The patient who was hospitalized after receiving Intellia Therapeutics’ CRISPR therapy nex-z has died. | The patient who was ...
TCTMD

CRISPR-Cas9 Gene-Editing Therapy Shows Early Promise for Dyslipidemia

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

A Gene Editing Therapy Cut Cholesterol Levels by Half

An experimental gene-editing therapy developed by Crispr Therapeutics is showing promise for treating heart disease.
New Atlas

One-off lipid-lowering gene therapy a success in world-first human trial

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

Can a One-Time Gene Therapy Lower Cholesterol?

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

Gene therapy for sickle cell and β-thalassemia works by disrupting three-dimensional genome structure

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...
BioSpace

Patient Dies After Receiving Intellia’s CRISPR Gene Therapy Candidate

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex ...