Drug intelligence / Profile preview

LNP-dsP21

Development stage
Preclinical
Lead developer
University of California, San Francisco
Modality
Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravesical
01

Overview

LNP-dsP21 is a lipid nanoparticle-formulated small activating RNA (saRNA) designed to induce the expression of the p21WAF1/CIP1 (p21) gene through a mechanism known as RNA activation (RNAa). Developed through a collaboration between the University of California San Francisco and Alnylam Pharmaceuticals, the drug targets the promoter region of the p21 gene, which encodes a key negative regulator of the cell cycle often downregulated in various cancers. By restoring p21 expression, LNP-dsP21 inhibits cell proliferation, induces cell cycle arrest, and promotes apoptosis in tumor cells. In preclinical studies, intravesical delivery of LNP-dsP21 demonstrated significant tumor regression and improved survival in orthotopic bladder cancer models, while maintaining stability in urine and exhibiting minimal immunostimulatory effects due to 2′-Fluoro chemical modifications.

Other names
LNP-formulated dsP21p21 activating dsRNAp-21 activating dsRNAp 21 activating dsRNA
02

Targets

CDKN1A (Cyclin-dependent kinase inhibitor 1A)AGO2 (Argonaute RISC catalytic component 2)

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