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Autigen siHES1+GSK3b
- Development stage
- Preclinical
- Lead developer
- Otologic Pharmaceutics
- Modality
- Small Molecules, 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
01
Overview
Autigen siHES1+GSK3b is a combination therapeutic candidate developed by Autigen Therapeutics in collaboration with Boehringer Ingelheim for the treatment of sensorineural hearing loss. The therapy utilizes a regenerative medicine approach to restore hearing by converting inner ear supporting cells into functional sensory hair cells. It consists of a small interfering RNA (siRNA) designed to silence the Hairy and enhancer of split 1 (HES1) gene, a transcriptional repressor that inhibits hair cell differentiation, and a small molecule inhibitor of Glycogen Synthase Kinase 3 beta (GSK3β). By simultaneously downregulating HES1 and inhibiting GSK3β (which activates the Wnt signaling pathway), the treatment aims to stimulate the proliferation of progenitor cells and their subsequent differentiation into new sensory hair cells within the cochlea.
- Other names
- Autigen siHES1+GSK3bAutigen HES1/GSK3b programsiHES1 + GSK3b inhibitor
02
Targets
HEY1 (Hairy/enhancer-of-split related with YRPW motif protein 1)GSK3 (Glycogen synthase kinase 3 beta)
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