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

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

A clandestine startup privately funded by tech billionaires is looking into germline gene editing a human embryo.

CRISPR Therapeutics' CEO said he hoped to have a product on the market in the next four or five years. Read more at ...

A CRISPR-based gene therapy showed improvement in lipids in difficult-to-treat patients, but one study raised flags after a death last month in a trial of an unrelated gene therapy.

In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...

GATC brought together a broad coalition of researchers and executives in cell and gene therapy, gene editing, drug discovery and AI as well as patients and advocates.

A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...

The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it ...

An innovative method of gene therapy to treat hearing and balance disabilities caused by impaired function of the inner ear ...

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

Jayden Wilsey was the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease after federal approval in 2024.