Press Release

CRISPR Therapeutics to Present Late-Breaking Data at the American Heart Association (AHA) Scientific Sessions 2025

ZUG, Switzerland and BOSTON, Sept. 09, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that a late-breaking oral presentation highlighting the Company's Phase 1 clinical data of its investigational CRISPR/Cas9 in vivo gene editing therapy, CTX310™, targeting angiopoietin-related protein 3 (ANGPTL3) for cardiovascular and cardiometabolic disease, will be presented at the American Heart Association (AHA) Scientific Sessions 2025, taking place November 7 – 10, 2025, in New Orleans, Louisiana. Additionally, the Company will present a poster presentation on CTX340™, its in vivo preclinical program targeting angiotensinogen (AGT) for the treatment of refractory hypertension.

Abstract Title: First-in-Human Phase 1 Clinical Trial of a CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3
Abstract Number: 4392851
Session Type: Late-Breaking Science (Oral Presentation)
Session Title: LBS.01 Groundbreaking Trials in Cardiometabolic Therapeutics
Session Date and Time: Saturday, November 8, 2025, 8:30 a.m. CST

Abstract Title: In vivo gene editing of Angiotensinogen in hepatocytes safely and potently reduces blood pressure in preclinical models
Abstract Number: 4391815
Session Type: Late-Breaking Basic Science (Poster Presentation)
Session Title: LBBS.APS.01 Precision Interventions for the Failing Heart: Genetic, Metabolic, and Immune Frontiers
Session Date and Time: Saturday, November 8, 2025, 2:30 p.m. CST

The accepted abstract titles are available online on the AHA website. The data in the abstracts and presentations are embargoed until the date and time of presentation. A copy of each presentation will be available at www.crisprtx.com once the presentation concludes.

About In Vivo Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular and cardiometabolic disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively.

About CRISPR Therapeutics
Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY^® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.

CRISPR THERAPEUTICS^® standard character mark and design logo CTX310™ and CTX340™ are trademarks and registered trademarks of CRISPR Therapeutics AG.

CRISPR Therapeutics Forward-Looking Statement
Statements contained in this press release 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. Such statements include, but are not limited to, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in the above-described oral presentation and above-described abstracts and any associated poster, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; and (iii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9, as well as other technologies. 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 CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics 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. We disclaim 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.

Investor Contact:
+1-617-307-7503
ir@crisprtx.com

Media Contact:
+1-617-315-4493
media@crisprtx.com

Source: CRISPR Therapeutics AG