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LTX-004

Development stage: Preclinical
Lead developer: Leal Therapeutics
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intrathecal

Overview

LTX-004 is an antisense oligonucleotide (ASO) drug candidate developed by Leal Therapeutics. It is in preclinical development for the treatment of neurodegenerative diseases, specifically Alzheimer’s disease and dementia with Lewy bodies. The drug targets lipid trafficking pathways implicated in these disorders, although its precise molecular target has not been explicitly disclosed in public sources. The therapeutic rationale involves modulating lipid metabolism to address pathological processes underlying neurodegeneration[1][2][3].

Targets

LDLR (Low-density lipoprotein receptor)

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