CTX320 / CRISPR Therap - LARVOL DELTA

Novel Lipoprotein (a) Therapies: A Comprehensive Review. (PubMed, Curr Atheroscler Rep) - "Pelacarsen, a GalNAc-conjugated antisense oligonucleotide (ASO), reduces Lp(a) by about 80% via hepatic apo(a) mRNA reduction and is the subject of the phase 3 Lp(a) HORIZON trial, with results due in late 2026. Three small interfering RNA (siRNA) drugs, olpasiran (> 95% reduction, OCEAN(a)-Outcomes), lepodisiran (93.9% reduction lasting over 12 months after one dose, ACCLAIM-Lp(a)), and zerlasiran (96.4% reduction), allow quarterly or possibly yearly dosing. Muvalaplin, the first oral small-molecule Lp(a) inhibitor, interferes with apo(a)-ApoB particle formation and reduces intact Lp(a) by up to 85.8% in phase 2 KRAKEN trials; its phase 3 CVOT (MOVE-Lp(a)) is underway. Additionally, CRISPR/Cas9-based liver gene editing targeting the LPA gene (CTX320) has entered phase 1 studies...Positive outcomes from these CVOTs could lead to regulatory approvals and guideline updates affecting hundreds of millions of high-risk patients globally. Recent guidelines now recommend..."

Journal • Review • Atherosclerosis • Cardiovascular • APOB

CTX320, targeting LPA (GlobeNewswire) - "The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) landscape."

P1 data • Trial status • Cardiovascular

CTX320: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces Lipoprotein (a) Levels in Non-Human Primates After a Single Dose (AHA 2023) - "CTX320 was well-tolerated in NHPs and resulted in high levels of editing in the liver that led to long-lasting reduction in plasma Lp(a). This data suggests CTX320 could be used to reduce plasma Lp(a) levels in humans."

Preclinical • Atherosclerosis • Cardiovascular • Dyslipidemia