Drug intelligence / Profile preview

ARVN601

Development stage: Preclinical
Lead developer: Arctic Vision
Modality: Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Intravitreal, Ophthalmic

Overview

ARVN601 (also known as ARVN-601) is a preclinical-stage, microRNA-based neuroprotective gene therapy being co-developed by Arctic Vision and the University of Hong Kong (HKU) for the treatment of glaucoma. Unlike conventional glaucoma therapies that focus on lowering intraocular pressure (IOP), ARVN601 targets non-IOP-related pathways to prevent or delay glaucomatous neurodegeneration. Delivered via an adeno-associated virus (AAV) vector, the therapy is designed to protect the optic nerve by modulating mitochondrial activity, reprogramming cellular pathways for neuroprotection, and promoting neuro-regeneration in retinal ganglion cells (RGCs).

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