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AV-016

Development stage: Preclinical
Lead developer: Avicenna Biosciences
Modality: Small Molecules

Overview

AV-016 is a small molecule inhibitor of Rho-associated protein kinase (ROCK) currently in the discovery stage for the treatment of Charcot-Marie-Tooth disease type 2A (CMT2A). Developed by Avicenna Therapeutics in partnership with the Charcot-Marie-Tooth Research Foundation (CMTRF), the drug aims to address the underlying pathology of CMT2A, which is characterized by mutations in the mitofusin 2 (MFN2) gene leading to mitochondrial dysfunction and axonal degeneration. ROCK inhibition is explored as a therapeutic strategy to improve axonal health and transport in peripheral nerves.

Targets

ROCK1 (Rho-associated coiled-coil containing protein kinase 1)

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