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AVB-203

Development stage: Preclinical
Lead developer: AviadoBio
Modality: Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

AVB-203 is a preclinical gene therapy candidate being developed by AviadoBio for the treatment of Amyotrophic Lateral Sclerosis (ALS) associated with mutations in the SOD1 gene. It utilizes AviadoBio's proprietary vMiX RNA silencing platform, which is designed to suppress the expression of the toxic SOD1 protein. The therapy is delivered intravenously and aims to provide a one-time treatment to slow or halt disease progression in patients with SOD1-ALS.

Targets

SOD (Manganese Superoxide Dismutase)

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