Drug intelligence / Profile preview

siHES1 + GSK3b

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
Otic
01

Overview

siHES1 + GSK3b (also known as Autigen siHES1+GSK3b) is a preclinical-stage siRNA combination therapy being developed by Otologic Pharmaceutics for the treatment of Sensorineural Hearing Loss (SNHL). The therapeutic employs a dual mechanism of action by combining two small interfering RNAs (siRNAs): one targeting HES1 mRNA and another targeting GSK3b (Glycogen Synthase Kinase 3 Beta) mRNA. HES1 is a transcriptional repressor in the Notch signaling pathway, which is involved in the immune response and sensory hair cell loss following acoustic trauma. By simultaneously knocking down HES1 and GSK3b, the therapy aims to stimulate the regeneration of inner ear hair cells or protect existing ones from damage. The program is currently available for partnering or out-licensing.

Other names
Autigen siHES1+GSK3b
02

Targets

HES1 (Hairy and Enhancer of Split 1 Protein)GSK3 (Glycogen synthase kinase 3 beta)

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