#gene editing

An early clinical trial suggests a single infusion of VERVE-102 can sharply lower LDL cholesterol, pointing to a possible alternative to lifelong medication.

Eli Lilly says a high dose of its VERVE-102 gene-editing therapy reduced cholesterol levels by 62% in participants in an early clinical study.

A first-in-human Nature Medicine trial reports that a CRISPR-edited transplant approach paired with targeted maintenance therapy achieved rapid engraftment in adults with high-risk AML and MDS.

Intellia has reported what the source material describes as the first Phase 3 success for an in vivo CRISPR therapy, a milestone that could move gene editing inside the body closer to regulatory approval.

Intellia Therapeutics says a gene-editing treatment for hereditary angioedema succeeded in Phase 3, marking what the company describes as the first Phase 3 CRISPR readout and a consequential step for the field.

A Rockefeller University-led study describes a gene-editing approach that reprograms a small number of stem cells so the immune system can produce long-lasting therapeutic proteins, including protective antibodies.

Researchers report an enhanced CRISPR system that could support targeted delivery inside the body, with the source text citing editing efficiencies of up to 90%.