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ABO-302

Development stage: Preclinical
Lead developer: Abeona Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Not Specified (likely Intravenous Or Systemic Gene Therapy Routes Based On Platform, But Not Explicitly Stated)

Overview

ABO-302 is an **AAV9-delivered gene therapy** being developed by Abeona Therapeutics for treating genetic inborn diseases, specifically congenital disorders. The therapeutic approach uses an adeno-associated virus serotype 9 (AAV9) vector to deliver corrective genetic material to target cells, thereby facilitating **gene transference** as its principal mechanism of action. ABO-302 is currently in the preclinical stage of development and has not yet entered human clinical trials. Its primary therapeutic area is genetic diseases with congenital or inborn errors[3].

Targets

FANCC (Fanconi anemia group C protein)

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