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ABO-301

Development stage: Preclinical
Lead developer: Abeona Therapeutics
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intravenous

Overview

ABO-301 is an adeno-associated virus (AAV) gene therapy developed by Abeona Therapeutics, licensed from the University of Minnesota. It is designed to treat Fanconi anemia and other rare blood diseases. The technology utilizes the CRISPR/Cas9 platform, suggesting a gene editing approach, likely targeting the FANCC gene, to address the underlying genetic defect in Fanconi anemia.

Targets

FANCC (Fanconi anemia group C protein)

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