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(Incorporated in Delaware)
We are a Phase 1 clinical biotechnology company focused on developing CRISPR therapies to prevent cardiovascular and metabolic diseases.
Our initial programs address the key drivers of atherosclerotic cardiovascular disease, or ASCVD.
Every 40 seconds, someone in the United States suffers a heart attack, and each year, heart disease costs the nation more than $400 billion, according to the American Heart Association. Despite major advances in our understanding of the pathology of heart disease and ASCVD, and the development of new classes of pharmaceuticals, we believe today’s standard of care for ASCVD is insufficient.
We have engineered two proprietary technologies based on a novel CRISPR-CasX enzyme: the Epigenetic Long-Term X-Repressor, or ELXR, designed for precise, durable epigenetic silencing without altering the underlying DNA sequence, and the X-Editor, or XE, designed for specific and efficient gene editing.
We are advancing three in vivo product candidates targeting three key lipid drivers of ASCVD: (1) elevated low-density lipoprotein cholesterol, or LDL-C; (2) elevated lipoprotein(a), or Lp(a), and (3) elevated triglycerides carried by triglyceride-rich lipoproteins, or TRLs.
Our lead product candidate, STX-1150, uses ELXR, our highly engineered epigenetic silencing technology, and is designed to deliver persistent and potent LDL-C reductions without permanent genetic changes. STX-1150 is a demonstration of moving towards our goal to transform practical therapeutic adherence and real-world medical outcomes for patients in the multibillion-dollar LDL-C lowering landscape. We have secured regulatory clearance from the Australian Therapeutic Goods Administration, or TGA, for and initiated a first-in-human clinical trial of STX-1150 in Australia in up to 64 adults with elevated LDL-C and increased risk of ASCVD. We anticipate reporting initial data from this trial in the first half of 2027.
Our lead product candidate, STX-1150, is designed to durably lower LDL-C by repressing the expression of PCSK9, a genetically and clinically validated target. Inhibition of PCSK9 is among the most effective known mechanisms to reduce LDL-C, complementing or outperforming other existing therapies. Unlike CRISPR gene editing, base editing, or prime editing approaches, STX-1150 is designed to achieve long-lasting therapeutic benefit without permanently altering underlying DNA sequence. STX-1150 aims to improve on the real-world efficacy of small-molecule, antibody, and siRNA therapies by eliminating the need for years to decades of chronic medication.
Our next programs, STX-1200 and STX-1400, target two additional lipid drivers of ASCVD, elevated Lp(a) and severely high triglycerides, by editing the LPA and APOC3 genes, respectively. Elevated Lp(a) and high triglycerides represent key risk factors for ASCVD affecting large populations. Both programs are based on our XE technology and aim to provide curative solutions for genetic diseases associated with the modification of these targets.
In mouse models of disease, prototype versions of both STX-1200 and STX-1400 programs have achieved greater than 90% reduction in target gene expression with durable effect consistent with a one-time treatment profile. In non-human primates (NHPs), surrogates demonstrated greater than 95% Lp(a) reduction and greater than 75% APOC3 on-target editing. In an off-target analysis of primary human hepatocyte donor cells, no detectable off-target editing was observed even at supersaturating doses.
We have been awarded grant funding from the California Institute for Regenerative Medicine, or CIRM, of up to about $25.7 million to support the development and manufacturing activities for our STX-1200 and STX-1400 programs. With CIRM funding support, we anticipate continuing to advance both the STX-1200 and STX-1400 programs through preclinical development to enable the initiation of a Phase 1 clinical trial for one of these programs as early as 2027 and one in 2028.
Note: Net loss and collaboration revenue are for the 12 months that ended on March 31, 2026.
(Note: Scribe Therapeutics, Inc. filed its S-1 on July 2, 2026, for its IPO, without disclosing the terms. Estimated proceeds are $75 million, a placeholder figure.)
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