Item 8.01 Other Events.
On June 26, 2025, CRISPR Therapeutics AG (the “Company”) announced updates across its in vivo cardiovascular disease programs. These include new data for CTX310™, targeting ANGPTL3, as well as continued progress on CTX320™, targeting the LPA gene, and CTX340™, targeting the AGT gene.
CTX310
CTX310 is in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia, or HoFH, severe hypertriglyceridemia, heterozygous familial hypercholesterolemia, or mixed dyslipidemias. Eligible participants have levels of TG >300 mg/dL and/or LDL-C >100 mg/dL (or >70 mg/dL for subjects with atherosclerotic heart disease). Both low-density lipoprotein, or LDL, and triglyceride, or TG, are validated surrogate endpoints accepted by regulatory agencies.
CTX310 continues to demonstrate dose-dependent reductions in TG and LDL. These new results build upon previously disclosed top-line data from the first 10 patients across the first four cohorts (lean body weight-based doses of dose level 1 [0.1 mg/kg], dose level 2 [0.3 mg/kg], dose level 3 [0.6 mg/kg] and dose level 4 [0.8 mg/kg] with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at dose level 4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings.
CTX320
CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events. Elevated Lp(a) levels affect up to 20% of the global population.
The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) landscape.
CTX340
The Company is also advancing its preclinical in vivo cardiovascular program CTX340, targeting angiotensinogen for the treatment of refractory hypertension. CTX340 is currently progressing through IND/CTA-enabling studies.
Forward-Looking Statements
Statements contained in this Current Report on Form 8-K regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in the Company's most recent annual report on Form 10-K and in any other subsequent filings made by the Company with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. The Company disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This Current Report on Form 8-K also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information.