CRISPR Therapeutics reports promising data for cholesterol drug CTX310

Published 26/06/2025, 12:06
CRISPR Therapeutics reports promising data for cholesterol drug CTX310

ZUG, Switzerland and BOSTON - CRISPR Therapeutics (NASDAQ:CRSP), a $4.4 billion gene editing company whose stock has surged over 21% year-to-date according to InvestingPro data, reported new Phase 1 clinical data for its experimental cholesterol drug CTX310, showing dose-dependent reductions in triglycerides and LDL cholesterol levels, according to a company statement released Thursday.

The data showed peak reductions of up to 82% in triglycerides and up to 86% in LDL cholesterol at the highest tested dose, with what the company described as a well-tolerated safety profile.

CTX310 targets the ANGPTL3 gene, which regulates LDL and triglyceride levels - both recognized risk factors for atherosclerotic heart disease. The drug is being tested in patients with various forms of high cholesterol and triglycerides, including homozygous familial hypercholesterolemia and severe hypertriglyceridemia.

The company plans to present complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025.

CRISPR Therapeutics also announced that data for CTX320, which targets the LPA gene for elevated lipoprotein(a), is now expected in the first half of 2026 rather than earlier. The company cited a "strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape" for the delay.

Additionally, the company reported that its preclinical cardiovascular program CTX340, targeting the AGT gene for refractory hypertension, is advancing toward regulatory filings.

CRISPR Therapeutics is developing these therapies using its proprietary lipid nanoparticle platform for delivering CRISPR/Cas9 gene editing components to the liver.

The company estimates that more than 40 million patients in the U.S. alone are affected by elevated LDL cholesterol, severely elevated triglycerides, or both conditions. With analyst price targets ranging from $32 to $268, investors can access detailed financial analysis and 12 additional exclusive ProTips through InvestingPro’s comprehensive research reports.

In other recent news, CRISPR Therapeutics has made significant strides in its financial and strategic endeavors. The company reported first-quarter 2025 results that met analyst expectations, prompting Clear Street to upgrade its stock rating from Hold to Buy, with a new price target of $45.00. Shareholders at CRISPR Therapeutics’ 2025 Annual General Meeting approved key proposals, including amendments to the Articles of Association and the election of board members. Additionally, CRISPR Therapeutics has formed a strategic partnership with Sirius Therapeutics to develop SRSD107, a novel siRNA therapy targeting thromboembolic disorders, with promising Phase 1 trial results. Citizens JMP has reiterated its Market Outperform rating and $86.00 price target, acknowledging the company’s progress in its in-vivo therapeutic programs. The firm highlighted the potential for CRISPR Therapeutics to achieve growth beyond its initial focus areas. The company’s CTX310 program has shown positive results in reducing ANGPTL3 protein levels, and there is anticipation for upcoming data from its CTX320 program targeting Lp(a). These developments underscore CRISPR Therapeutics’ ongoing efforts to expand its portfolio and enhance its position in the biotechnology sector.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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