Drug intelligence / Profile preview

LErafAON

Development stage
Phase 1
Lead developer
Neopharm
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

LErafAON is a liposome-encapsulated antisense oligodeoxynucleotide designed to target the mRNA of human c-Raf (RAF1), a serine/threonine-protein kinase that acts as a regulatory link between Ras GTPases and the MAPK/ERK signaling cascade. By binding to the translation initiation site of c-Raf mRNA, LErafAON blocks its expression and production, thereby inhibiting Raf-mediated cell proliferation and survival pathways. This mechanism is intended to enhance the cytotoxic effects of radiation and chemotherapy in various solid tumors, including those resistant to standard treatments. Developed by NeoPharm, clinical development reached Phase 1/2 for advanced cancers but was ultimately discontinued[1][2][4][5][7].

Other names
liposomal c-raf antisense oligonucleotide
02

Targets

RAF1 (c-Raf-1 (Y340D/Y341D))

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