W-0301 / NanoVation Therapeutics - LARVOL DELTA

Development of a JAK2V617F-targeting lipid nanoparticle-siRNA as a novel gene therapy for myeloproliferative neoplasms (ASH 2025) - "Over 50% of patients discontinue ruxolitinib within 3 years due tosevere side-effects...W-0301 induced JAK2V617F-specific gene-silencing, followed by mutation-specific cellkilling of JAK2V617F HSPCs in vitro and in vivo, and amelioration of key disease pathologies. Our studyoffers a new approach for JAK2V617F mutant–selective inhibition through apoptosis-induced eradicationof mutant HSPCs, thereby representing a potential curative strategy for patients with JAK2V617F positiveMPNs. It furthermore demonstrates the potency of our targeting ligand-independent LNP-RNA platformtechnology to create therapeutics for other haematological malignancies with high driver-mutationprevalence."

Gene therapy • Essential Thrombocythemia • Gene Therapies • Hematological Malignancies • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • JAK2 • TFRC