Date: 2015-10-06
Type of information: Licensing agreement
Compound: AAV (adeno-associated virus) delivery vector
Company: Abeona Therapeutics (USA - NY) Stanford University (USA - CA)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: Fanconi anemia
Details: * On October 6, 2015, Abeona Therapeutics announced a license agreement with Stanford University for an AAV (adeno-associated virus) delivery vector for the treatment of Fanconi anemia and rare blood disease platform. This license augments a previously announced license agreement with the University of Minnesota for ABO-301 (AAV-FANCC) to treat patients with Fanconi anemia disorder and other rare blood diseases. In Fanconi anemia, a mutation renders the FANCC (Fanconi Anemia Complementation Group C) gene nonfunctional. Loss of FANCC causes patient skeletal abnormalities and leads to bone marrow failure. Fanconi anemia patients also have much higher rates of hematological diseases, such as acute myeloid leukemia or tumors of the head, neck, skin, gastrointestinal system, or genital tract. The likelihood of developing one of these cancers in people with Fanconi anemia is between 10 and 30 percent. Aside from bone marrow transplantation there are no specific treatments known that can halt or reverse the symptoms of Fanconi anemia. Repairing fibroblast cells in Fanconi anemia patients with a functional FANCC gene is the focus of this AAV-based gene therapy approach.
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