Drug intelligence / Profile preview

L1 FANA ASO

Development stage
Preclinical
Lead developer
Altos Labs
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intradermal, Intramuscular, Intranasal, Intraperitoneal, Intrathecal, Intravenous, Subcutaneous
01

Overview

L1 FANA ASO is a 2'-deoxy-2'-fluoro-beta-D-arabinonucleic acid (FANA) modified antisense oligonucleotide (ASO) designed to target and knockdown Long Interspersed Nuclear Element-1 (LINE-1 or L1) retrotransposon RNA. Developed by researchers at the Salk Institute for Biological Studies, King Abdullah University of Science and Technology (KAUST), and Altos Labs, this ASO is designed to prevent the accumulation of L1 RNA, which is elevated in progeroid syndromes (such as Hutchinson-Gilford progeria syndrome and Werner syndrome) and during physiological aging. By depleting L1 RNA, the ASO restores heterochromatin homeostasis (specifically preventing the loss of histone marks like H3K9me3 and H3K27me3) and reverses senescent phenotypes. In preclinical models, systemic administration of L1 ASO has been shown to rescue tissue histophysiology and extend lifespan. It is also being investigated in academic research for its role in modulating social behavior and anxiety-like behaviors in neonatal rat models of early life adversity.

Other names
L1 ASOL-1 ASOL 1 ASOL1 antisense oligonucleotideL-1 antisense oligonucleotideL 1 antisense oligonucleotideLINE-1 ASOLINE1 ASOLINE 1 ASOLINE-1 FANA ASOLINE1 FANA ASOLINE 1 FANA ASOLINE-1 antisense oligonucleotideLINE1 antisense oligonucleotideLINE 1 antisense oligonucleotide

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