W-0301

**W-0301** is a preclinical, intravenously administered lipid nanoparticle small interfering RNA therapeutic designed to selectively silence the **JAK2 V617F** mutant transcript while sparing wild-type JAK2. It encapsulates the mutation-selective siRNA cargo W-01 in a ligand-independent bone-marrow-targeting lipid nanoparticle formulation designated 0.03. In JAK2 V617F-positive myeloproliferative neoplasm models, W-0301 produced dose-dependent mutant-selective knockdown, apoptosis of mutant hematopoietic stem and progenitor cells, and improvement of polycythemia vera-like erythroid disease features in mice. ([ashpublications.org](https://ashpublications.org/blood/article/146/Supplement%201/383/554977/Development-of-a-JAK2V617F-targeting-lipid))

Other names

LNP-siJAK2V617FLNP-siJAK-2V617FLNP-siJAK 2V617F