CRISPR Therapeutics' Investigational Gene Therapy Shows Promising Lipid-Lowering Effects In Early Trial

On Saturday, CRISPR Therapeutics AG (NASDAQ:CRSP) shared Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3.

ANGPTL3, or Angiopoietin-Like 3, is a protein primarily produced in the liver that plays a major role in regulating blood lipid (fat) levels, particularly triglycerides and cholesterol.

A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean decrease from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose.

Also Read: CRISPR Therapeutics’ Data Shows Promise, But RFK Jr. Appointee Could Be Negative: Analyst

These data demonstrate the potential of CTX310 to deliver meaningful and sustained lipid lowering following a single-course intravenous infusion.

The data, presented at the American Heart Association Scientific Sessions, was published simultaneously in The New England Journal of Medicine.

CTX310 was generally well tolerated, with no dose-limiting toxicities or serious treatment-related adverse events observed. Adverse events were generally mild to moderate.

Analysts’ Take

William Blair on Monday wrote, “We believe the CTX310 data represents a significant positive development for the company as it demonstrated robust, dose-dependent reductions in circulating ANGPTL3 and atherogenic lipoproteins, positioning CTX310 as a potentially transformative one-time treatment for dyslipidemias.”

Analyst Sami Corwin writes that CTX310 clinical data appear competitive with the LDL-C reductions reported with Arrowhead Pharmaceuticals Inc.’s (NASDAQ:ARWR) AROANG3 and Regeneron Pharmaceuticals Inc.’s (NASDAQ:REGN) Evkeeza, and the TG reductions potentially set a new bar in the space.

Corwin highlights that the data more broadly support CRISPR's in vivo cardiovascular gene-editing platform and de-risk the safety of its other assets, including CTX320.

Price Action: CRSP stock is up 0.86% at $55.05 during the premarket session at the last check on Tuesday.

Read Next:

• Celsius Holdings Stock Bounces Back From Steep Sell-Off

Photo: Shutterstock