Scribe Therapeutics, Inc. (Scribe), a clinical-stage biotechnology company engineering purpose-built in vivo CRISPR technologies designed to extend healthy lifespan through disease prevention and durable therapeutic intervention, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded the company more than $25 million across two preclinical programs targeting cardiovascular and metabolic disease. The awards were made through CIRM’s Preclinical Development Program (PDEV), which supports California-based research projects moving discoveries toward clinical trials. The two awards will support the advancement of Scribe’s wholly-owned CRISPR-based gene editing therapies toward clinical development.
The research awards support two novel therapies in Scribe’s comprehensive cardiometabolic pipeline: STX-1200, which targets LPA to decrease lipoprotein(a) [Lp(a)] to treat and prevent atherosclerotic cardiovascular disease (ASCVD) in patients with genetically elevated Lp(a); and STX-1400, targeting APOC3 to lower triglyceride-rich lipoproteins to address acute pancreatitis in triglyceride-driven diseases such as severe hypertriglyceridemia (SHTG), in particular familial chylomicronemia syndrome (FCS) and multifactorial chylomicronemia syndrome (MCS). Both programs leverage Scribe’s X-Editor (XE) gene editing technology, a platform highly engineered for enhanced activity, specificity, and deliverability. Scribe’s STX-1200 and STX-1400 programs are led by principal investigator Brett Staahl, Ph.D., co-founder and VP of External Innovation, and were selected as part of a cohort of awards supporting promising gene therapy and stem cell research.
“We are proud to share that two of Scribe’s CRISPR-based genetic medicine approaches have been recognized by CIRM for their strong therapeutic potential to address cardiovascular disease for millions of patients,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe. “These grants support our efforts to advance potentially curative gene editing therapies to provide genetic solutions for the lifelong genetic diseases that underpin many cardiovascular events. Patients deserve a more practical and effective standard of care, and we aim to deliver a suite of fit-for-purpose approaches for each patient. We are honored to be supported by the CIRM vision for accelerating biomedical research and delivering cutting-edge technology to patients in our home state of California.”
“CIRM’s preclinical development program is intended to move potential therapies toward clinical trials for patients who have no other options,” said Lisa McGinley, Ph.D., Senior Science Officer for Preclinical Development at CIRM. “Genetic medicines such as those being developed by Scribe could be life-changing for the millions of people in California and the world who have genetic predisposition to forms of heart disease.”
Elevated Lp(a) is a major genetic cause of heart disease, affecting about one in five people worldwide. People with elevated Lp(a) are two to four times more likely to have a premature heart attack or a heart valve condition known as aortic stenosis. Because Lp(a) levels are primarily determined by a person’s genes and passed down in families, elevated Lp(a) represents one of the largest genetically driven risks for heart disease and is well-suited for targeting by a genetic medicine approach. Despite its impact, elevated Lp(a) remains underdiagnosed, and there are currently no approved therapies that directly and durably lower Lp(a). STX-1200 is designed to reduce Lp(a) and eliminate Lp(a)-driven cardiovascular disease risk by providing a potentially curative genetic medicine approach for this genetic disease.
Elevated triglycerides and triglyceride-rich lipoproteins, which are fat-like particles in the blood, can increase the risk of heart disease and serious conditions such as acute pancreatitis in diseases such as familial chylomicronemia syndrome (FCS), multifactorial chylomicronemia syndrome (MCS), and severe hypertriglyceridemia (SHTG). SHTG affects approximately 8 million adults in the U.S. and Europe, while in FCS, up to 9 out of 10 patients experience acute pancreatitis requiring intensive care. STX-1400 targets the APOC3 gene, with the goal of lowering triglycerides durably through a potentially single-dose treatment. By targeting APOC3, a genetic regulator of triglyceride levels, STX-1400 is designed to provide a genetic solution that may help reduce the risk of acute pancreatitis and cardiovascular events, especially for patients who remain underserved by current drugs and restrictive diet regimens.
With support from CIRM, Scribe will continue advancing STX-1200 and STX-1400 through preclinical development as part of its broader strategy to develop durable, potentially one-time CRISPR-based genetic medicines to target key lipid drivers of ASCVD. Together with Scribe’s clinical-stage lead asset, STX-1150, these programs reflect the company’s commitment to applying its CRISPR by Design™ approach to expand the potential of genetic medicine for prevalent cardiometabolic diseases.
About Atherosclerotic Cardiovascular Disease (ASCVD)
Cardiovascular disease remains the leading cause of death worldwide and impacts over 120 million individuals in the United States alone. Every 40 seconds, someone in the United States suffers a heart attack.
Elevated levels of low-density lipoprotein cholesterol (LDL-C), lipoprotein(a) [Lp(a)], and triglycerides carried by triglyceride-rich lipoproteins (TRLs) are key lipid drivers of atherosclerosis and ASCVD. Hypercholesterolemia, an excess of LDL-C in the bloodstream, promotes plaque formation in the arterial walls, restricting blood flow and increasing the risk of heart attacks and strokes. Similarly, elevated atherogenic Lp(a) and TRL particles drive increased risk of ASCVD independent of controlled LDL-C levels.
Despite major advances in the development of new classes of lipid-lowering therapies, today’s standard of care for ASCVD treatment and prevention is insufficient. Existing treatments struggle to demonstrate broad impact as they suffer from significant lack of durability, diminishing levels of adherence-adjusted efficacy, and onerous treatment burdens including well-documented side effects. All of this leads to poor uptake, low adherence, and limited real-world effectiveness. Moreover, treatment is often initiated only after decades of substantial silent cumulative arterial injury or following an acute cardiovascular event. These limitations underscore the importance of developing durable therapies that can be administered safely earlier in the course of disease. Scribe’s goal is to develop CRISPR-based therapeutics that are safe, effective, durable, and scalable enough to preemptively reduce lifetime cardiovascular risk, with the aim of reducing the global burden of ASCVD.
About Scribe’s X-Editor (XE)
XE is Scribe’s novel CRISPR-based gene editing technology designed for precise and versatile genetic modification. It uses a staggered DNA cleavage mechanism to achieve high on-target activity, specificity, and flexibility across a range of applications, including gene knock-out, knock-down, knock-in, exon skipping, genetic excision, and other targeted modifications. Scribe has engineered its novel CasX enzyme, XE, with improved nuclease stability, DNA binding, cleavage activity, and specificity, resulting in greater than 100-fold higher editing than naturally occurring CasX in cell-based assays while maintaining high specificity across target sites. XE’s compact size and engineered PAM recognition together provide a differentiated combination of potency, specificity, delivery, flexibility, and a broad therapeutic window, supporting its potential application in durable, one-time treatments for genetically defined diseases.
About Scribe Therapeutics
Scribe Therapeutics is a clinical-stage biotechnology company engineering CRISPR-based technologies into purpose-built in vivo genetic medicines designed to become standard of care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. Leveraging its CRISPR by Design™ approach and nature’s blueprint for improved cardiovascular health, Scribe’s initial programs focus on addressing the key lipid drivers of ASCVD such as elevated LDL-C, lipoprotein(a), and triglycerides. The company’s lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence the PCSK9 gene and reduce LDL-C levels without inducing permanent DNA changes. 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 advancing scalable, transformative, and preventative genetic medicines with the goal of improving outcomes and democratizing access to the protective effects of beneficial human genetics. To learn more, visit www.scribetx.com.
About the California Institute for Regenerative Medicine (CIRM)
The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative treatments. Today, CIRM is pioneering new models of therapy development and accelerating medical breakthroughs that change lives — in California and around the world. For more information, visit www.cirm.ca.gov.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260618696563/en/
Media gallery
