STX1400 / Scribe Therapeutics - LARVOL DELTA

Single dose of a novel CRISPR-CasX editor potently reduces triglycerides in preclinical models and efficiently edits APOC3 in non-human primates (AHA 2025) - "Our findings demonstrate that the STX-1400 prototype potently and specifically edits APOC3 across human cells, mouse models, and non-human primates. This study is the first demonstration that a CRISPR-based therapy has achieved saturated in vivo editing of the APOC3 locus in NHP liver. These results provide strong preclinical evidence supporting the potential of STX-1400 as a first-in class genome editing therapy for hypertriglyceridemia in patients with SHTG and FCS."

Preclinical • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Chylomicronemia Syndrome • Hypertriglyceridemia • Pancreatitis • Severe Hypertriglyceridemia • APOC3