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ABO-203
- Development stage
- Preclinical
- Lead developer
- Arbor Biotechnologies
- Modality
- Programmable Nucleases → Gene Editing → Gene Therapies, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
01
Overview
ABO-203 is an AAV-based gene therapy developed by Arbor Biotechnologies. It utilizes nuclease excision technology for delivery and is designed to target specific regions of the brain to address the underlying genetic drivers of Amyotrophic Lateral Sclerosis (ALS). This approach aims to provide long-lasting therapeutic effects by modifying genetic material.
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