Coming soon: First-in-human study of gene-modifying drug for hypercholesterolemia
Scribe Therapeutics said it plans to start a first-in-human Phase 1 study in mid-2026 for its STX-1150 drug to treat hypercholesterolemia. This novel drug is a CRISPR-based genetic modification agent that targets the liver to silence PCSK9 and reduce low-density lipoprotein cholesterol (LDL-C) without permanent DNA changes.
CRISPR-based gene editing has shown potential to treat a variety of diseases, and STX-1150 will be among only a few in cardiovascular medicine to begin human trials.
According to Scribe, preclinical studies showed durability of the medication with a LDL-C drop of more than 50% in non-human primates maintained for about 18 months after a single dose of less than 1 mg/kg.
STX-1150 was created using the company’s proprietary Epigenetic Long-Term X-Repressor (ELXR) technology. It is designed to turn off gene expression of multiple genetic targets without cutting DNA, the company said. Scribe also announced the online publication of a manuscript describing the engineering behind this technology.
“Entering the clinic with STX-1150 represents a defining moment for Scribe and the wider genetic medicine field,” Benjamin Oakes, PhD, co-founder and CEO of Scribe, said in a statement. "We designed STX-1150 to overcome many of the limitations of today’s lipid-lowering therapies through powerful epigenetic silencing, and to meaningfully change how cardiovascular risk is managed for millions of patients.”
The company said STX-1150 is designed to address the gap between efficacy in controlled settings and real-world outcomes for chronic lipid-lowering therapies. These limitations, including the use of repeat doses, can limit patient adherence and treatment effectiveness.
Gene-editing drugs have been a big research topic at cardiology conferences in recent years and hold promise for new directions in pharmacology. One such drug made it into the late-breaking presentations at the 2025 American Heart Association (AHA) meeting. In that first-in-human study, a single dose of a new intravenously administered CRISPR-Cas9 gene-editing therapy CTX310 from CRISPR Therapeutics significantly reduced LDL cholesterol and triglyceride levels in high-risk heart patients.
Another CRISPR agent, VERVE-101 from Verve Therapeutics, had its first-in-human study results presented at AHA 2023. The results showed a significant reduction in LDL that could potentially last for decades.
Dave Fornell has covered healthcare for more than 17 years, with a focus in cardiology and radiology. Fornell is a 5-time winner of a Jesse H. Neal Award, the most prestigious editorial honors in the field of specialized journalism. The wins included best technical content, best use of social media and best COVID-19 coverage. Fornell was also a three-time Neal finalist for best range of work by a single author. He produces more than 100 editorial videos each year, most of them interviews with key opinion leaders in medicine. He also writes technical articles, covers key trends, conducts video hospital site visits, and is very involved with social media. E-mail: [email protected]