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ABO-204

Development stage: Unknown
Lead developer: Arbor Biotechnologies
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Not Specified (likely Parenteral Or Intrathecal Based On Typical AAV-based Cns Therapies)

Overview

ABO-204 is an investigational gene therapy developed by Arbor Biotechnologies. It is an adeno-associated virus (AAV)-based gene therapy designed to inhibit superoxide dismutase 1 (SOD1), a target implicated in certain neurodegenerative and metabolic diseases. The primary mechanism of action involves SOD1 inhibition, which may be relevant for conditions such as amyotrophic lateral sclerosis (ALS). As of the latest updates, the drug remains in the discovery phase and has not yet entered clinical trials[2]. Arbor Biotechnologies specializes in developing next-generation genetic medicines using proprietary genome editing technologies[6].

Targets

Misfolded SOD1

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