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Autigen siHES1#7 (Otologic Pharmaceutics, Inc.)

Development stage
Preclinical
Lead developer
Otologic Pharmaceutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratympanic, Intracochlear
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Overview

Autigen siHES1#7 is a preclinical-stage small interfering RNA (siRNA) therapeutic candidate developed by Otologic Pharmaceutics for the treatment of sensorineural hearing loss (SNHL). It is a specific variant within the Autigen program designed to promote the regeneration of sensory hair cells in the inner ear. The mechanism of action involves the siRNA-mediated knockdown of Hairy and enhancer of split 1 (HES1), a transcription factor that normally inhibits the differentiation of supporting cells into hair cells. By suppressing HES1 expression, the drug aims to trigger the transdifferentiation of these supporting cells into new sensory hair cells, potentially restoring hearing function lost due to hair cell damage.

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Targets

HES1 (Hairy and Enhancer of Split 1 Protein)

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