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LTX-003

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, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intrathecal, Intravitreal

Overview

LTX-003 is an antisense oligonucleotide (ASO) drug candidate developed by Leal Therapeutics. It is in preclinical development for the treatment of neurodegenerative and ophthalmic diseases, specifically Alzheimer's disease, frontotemporal dementia (FTD), and age-related macular degeneration (AMD). The drug targets the complement system, aiming to modulate complement activity implicated in these disorders. As an ASO, LTX-003 works by binding to specific RNA sequences to alter gene expression related to its target pathway.

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