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Abeona Therapeutics, Inc.

Drug Names(s): Recombinant AAV9 expressing LK19 FANCC

Description: ABO-301 is an adeno-associated virus (AAV) gene therapy program expressing LK19 FANCC for the treatment of Fanconi anemia. Overall, mutations in the FANCC (Fanconi anemia, complementation group C) gene account for ~15% of cases of Fanconi Anemia.

Loss of FANCC causes skeletal abnormalities and bone marrow failure. Fanconi Anemia patients also have much higher rates of hematological disorders, such as acute myeloid leukemia (AML) or tumors of the head, neck, skin, gastrointestinal system, or genital tract. Reparing fibroblast cells in FA patients with a functional FANCC gene is the focus of the company's AAV-based gene therapy approach.

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