Date: 2015-06-15
Type of information: Licensing agreement
Compound: ABO-301 (AAV-FANCC) - AAV gene therapy for the treatment of Fanconi anemia
Company: PlasmaTech Biopharmaceuticals, now Abeona Therapeutics (USA - NY) University of Minnesota (USA - MN)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: Fanconi anemia
Details: * On June 15, 2015, PlasmaTech Biopharmaceuticals announced that it has added another adeno-associated virus (AAV) gene therapy program to its product pipeline. The company has licensed an AAV gene therapy and IP from the University of Minnesota to treat patients with Fanconi anemia disorder and other rare blood diseases. "Aside from blood and marrow transplantation that carries a risk of significant side effects, there are no treatments available that can halt or reverse the symptoms of children with Fanconi anemia disorder. Using the CRISPR-Cas9 gene-editing system to repair the FANCC gene in human fibroblasts from a Fanconi anemia patient, our AAV-based gene therapy studies have demonstrated significant and promising results," said Jakub Tolar, M.D., Ph.D., who is Professor, Department of Pediatric Blood & Marrow Transplantation, Tulloch Chair in Stem Cell Biology, Genetics and Genomics; and Director, Stem Cell Institute at the University of Minnesota. "Working with PlasmaTech, as well as the dedicated Fanconi anemia disorder community of patients and their families, will help us accelerate this therapy into clinical trials." PlasmaTech recently acquired Abeona Therapeutics, which developed AAV gene therapies for the treatment of Sanfilippo syndromes (MPS IIIA and MPS IIIB), and licensed an AAV gene therapy program in juvenile Batten disease from the University of Nebraska Medical Center.
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