Drug intelligence / Profile preview

EN-06

Development stage
Preclinical
Lead developer
Immunicom
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
Intracerebroventricular
01

Overview

EN-06 is an experimental **ENA-modified antisense oligonucleotide** designed to correct abnormal tau isoform splicing by lowering the 4R-tau to 3R-tau ratio while preserving overall **MAPT** expression. Preclinical reports describe it as a long-acting splice-switching ASO identified in vitro as the most effective ENA candidate and subsequently shown in mouse models of 4R tauopathy to reduce phosphorylated tau pathology, improve behavioral abnormalities, and produce durable effects after intracerebroventricular administration. The program appears to be an academic-stage therapeutic effort associated with investigators at Shiga University of Medical Science and collaborators, with relevance to **4R tauopathies** such as progressive supranuclear palsy, corticobasal degeneration, and frontotemporal lobar degeneration.

Other names
ENA-ASO EN-06

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