Scribe Therapeutics announced that it has been awarded more than $25 million from the California Institute for Regenerative Medicine across two preclinical programs targeting cardiovascular and metabolic disease.
The awards were made through CIRM’s Preclinical Development Program, which supports California-based research projects moving discoveries toward clinical trials.
Scribe is a clinical-stage biotechnology company developing purpose-built in vivo CRISPR technologies designed for disease prevention and durable therapeutic intervention. The new funding will support the advancement of two wholly owned CRISPR-based gene editing therapies toward clinical development.
The awards will support STX-1200 and STX-1400, two programs in Scribe’s cardiometabolic pipeline. Both programs use Scribe’s X-Editor gene editing technology, which is engineered for enhanced activity, specificity, and deliverability.
STX-1200 targets LPA to decrease lipoprotein(a), or Lp(a), with the goal of treating and preventing atherosclerotic cardiovascular disease in patients with genetically elevated Lp(a). Elevated Lp(a) is a major genetic risk factor for heart disease and affects about one in five people worldwide.
Scribe said STX-1200 is designed to reduce Lp(a) and address Lp(a)-driven cardiovascular disease risk through a potentially curative genetic medicine approach.
STX-1400 targets APOC3 to lower triglyceride-rich lipoproteins. The program is designed to address acute pancreatitis in triglyceride-driven diseases such as severe hypertriglyceridemia, familial chylomicronemia syndrome, and multifactorial chylomicronemia syndrome.
Scribe said STX-1400 is intended to durably lower triglycerides through a potentially single-dose treatment, helping reduce the risk of acute pancreatitis and cardiovascular events in underserved patient populations.
The STX-1200 and STX-1400 programs are led by Brett Staahl, Co-Founder and VP of External Innovation at Scribe.
With CIRM’s support, Scribe plans to continue advancing both programs through preclinical development as part of its broader strategy to develop durable, potentially one-time CRISPR-based genetic medicines targeting key lipid drivers of atherosclerotic cardiovascular disease.
The programs are part of Scribe’s broader cardiometabolic strategy, which also includes its clinical-stage lead asset, STX-1150. STX-1150 is a liver-targeted therapy designed to epigenetically silence PCSK9 and reduce LDL-C levels without inducing permanent DNA changes.
Scribe was co-founded by Nobel Prize winner Jennifer Doudna and has formed strategic collaborations with pharmaceutical companies, including Sanofi and Eli Lilly.
KEY QUOTES:
“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. 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.”
Benjamin Oakes, Co-Founder and Chief Executive Officer of Scribe Therapeutics
“CIRM’s preclinical development program is intended to move potential therapies toward clinical trials for patients who have no other options. 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.”
Lisa McGinley, Senior Science Officer for Preclinical Development at CIRM
