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PlasmaTech Biopharmaceuticals Expands Rare Disease Pipeline

License agreement for gene therapy in Fanconi anemia & CRISPR/Cas9-based technology platform for rare blood disorders

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By: Tim Wright

Editor-in-Chief, Contract Pharma

PlasmaTech Biopharmaceuticals, Inc. 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 (FA) disorder and other rare blood diseases. 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 juvenil...

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