Scribe Therapeutics Highlights Latest Data on Lp(a)-Lowering Therapy and Novel AI Platform for Next-Generation CRISPR-Based Therapeutics at Two Premier Conferences

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ALAMEDA, Calif.--(BUSINESS WIRE)--Sep 9, 2025--

Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, recently presented new breakthrough data from two of its core platforms. At the European Society of Cardiology (ESC) Congress, Scribe presented results from STX-1200, its next-generation CasXE-based therapy, showing strong potency and precision in lowering lipoprotein(a) (Lp(a)), a major and under-addressed driver of cardiovascular disease. At the Cold Spring Harbor Laboratory (CSHL) Genome Engineering meeting, the company unveiled DeepXE, its proprietary AI-powered CRISPR design platform that accurately predicts gRNA editing efficiency, outperforming existing Cas9-based predictive models, thereby enabling the more rapid development and deployment of precise and effective genome-editing treatments.

“STX-1200 expands our cardiometabolic portfolio with a direct approach to a major unsolved ASCVD risk factor, elevated Lp(a). Our CRISPR therapy is designed to deliver profound and durable Lp(a) lowering for patients worldwide. Beyond potency, Scribe’s XE platform is the only CRISPR approach to demonstrate Lp(a) gene editing without off-target effects, even at supersaturating doses,” said Benjamin Oakes, Ph.D., co-founder and CEO of Scribe. “For the first time, we are also sharing elements of our machine learning toolkit, including ‘DeepXE,’ which leverages AI to accelerate the design of potent, precise CRISPR-based therapies. These advances further enhance the speed and accuracy with which Scribe can create the next generation of genetic medicines that are effective and safe enough for broad use.”

CRISPR-Based Gene Editor for Durable Lp(a) Lowering

At the ESC Congress 2025, Scribe reported late-breaking data from its development and evaluation of STX-1200 in preclinical models for editing potency and functional effects on the reduction of Lp(a). Elevated Lp(a) is a prevalent, genetically driven risk factor for cardiovascular disease, and no FDA-approved therapies exist to date. Affecting approximately 20% of the global population, elevated Lp(a) is considered a significant emerging priority in cardiovascular disease management.

The reported findings validate the company’s CasXE-based approach to selective LPA gene editing, leading to substantial permanent reduction of the apolipoprotein(a) (Apo(a)) protein, the key component of the Lp(a) particle. Scribe has designed STX-1200 as part of its pipeline of therapies to address the key unmet need in the treatment of cardiovascular disease: lack of effective, long-term therapeutic adherence. Highlights from the presentation include:

• A single very low dose of STX-1200 achieved highly efficient hepatic LPA editing and >90% Apo(a) knockdown in a preclinical, pharmacologically relevant transgenic LPA mouse model
• Comprehensive off-target assessment confirmed no detectable off-target editing, even at a supersaturating dose of 10X EC90, highlighting that STX-1200 has an unmatched specificity profile and is designed for safe and targeted LPA editing
• The study represents the first preclinical proof-of-concept of an exceptionally safe and effective CRISPR-based genetic medicine for elevated Lp(a), potentially transforming treatment for millions of patients

AI-Enabled CRISPR gRNA Design to Accelerate Next-Generation CRISPR Genetic Medicines

At the 11th Genome Engineering: CRISPR Frontiers meeting hosted by Cold Spring Harbor Laboratory, Scribe introduced DeepXE, its proprietary AI-enabled CRISPR design platform that accurately predicts editing efficiency for its X-Editor (CasXE)-based therapeutics. By enabling more precise discovery of highly potent molecules, DeepXE fundamentally changes the speed and efficiency with which next-generation CasXE-based therapeutics can be developed to support Scribe’s growing pipeline. Highlights from the presentation include:

• DeepXE is the first machine learning model capable of accurately predicting on-target editing efficiency for XE, a highly engineered variant of wild-type CRISPR-CasX, addressing a critical gap for next-generation XE-based genome editing therapies
• The model demonstrates robust predictive performance and high predictive accuracy, delivering a 2x higher hit rate with <10% false negatives
• With a sensitivity exceeding 90%, DeepXE also significantly outperforms Cas9-based conventional machine learning models
• Assessed on multiple engineered XE variants, target genes, and assay conditions, DeepXE exhibited strong generalizability, highlighting its broad applicability for different therapeutic programs
• By enabling faster and more cost-effective design of potent molecules (achieved >50% reduction in screening size), DeepXE streamlines efforts for developing more precise and effective treatments

About Scribe Therapeutics

Scribe Therapeutics is revolutionizing medicine by developing optimized in vivo CRISPR-based genetic medicines designed to become standard of care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. The company is on a mission to build the first CRISPR-based therapeutics that are effective and safe enough to transform everyone’s lifetime risk for disease. Scribe’s CRISPR by Design™ approach engineers bacterial immune systems into a premier suite of genome and epigenome editing tools built for unique molecular advantages in activity, specificity, and deliverability, enabling the creation of therapies with a broader therapeutic window and safe for use as a preventative treatment. The company’s lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence the PCSK9 gene, resulting in significant and durable reduction of LDL-C levels. To broaden and accelerate the impact of its engineered CRISPR technologies for patients, Scribe has formed strategic collaborations with world-leading pharmaceutical companies including Sanofi and Eli Lilly. Co-founded by Nobel Prize winner Jennifer Doudna and backed by leading life sciences investors, Scribe is engineering the future of genetic medicine. To learn more, visit www.scribetx.com.

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KEYWORD: CALIFORNIA UNITED STATES NORTH AMERICA

INDUSTRY KEYWORD: CARDIOLOGY BIOTECHNOLOGY TECHNOLOGY HEALTH PHARMACEUTICAL HEALTH TECHNOLOGY RESEARCH ARTIFICIAL INTELLIGENCE GENETICS SCIENCE CLINICAL TRIALS

SOURCE: Scribe Therapeutics Inc.

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PUB: 09/09/2025 09:00 AM/DISC: 09/09/2025 08:59 AM

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