PlasmaTech Biopharmaceuticals Expands Rare Disease Pipeline With License Agreement for Gene Therapy in Fanconi Anemia & CRISPR/Cas9-Based Technology Platform for Rare Blood Disorders

PlasmaTech Biopharmaceuticals, Inc.

, a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, announced today 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 (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 juvenile Batten disease from the University of Nebraska Medical Center. "This licensing transaction expands our commitment to building a leadership position in the gene therapy space, with a focus on developing therapies for patients with rare diseases," stated Steven Rouhandeh, Executive Chairman. "We plan to leverage the unique capabilities of the CRISPR-Cas9 gene editing platform technology to build a robust product pipeline to address the unmet needs of patients that may have one of a variety of blood diseases." "This promising addition to our AAV gene therapy pipeline leverages our experience in developing products for severe and life threatening rare diseases. There are a significant number of blood disorders that could potentially be corrected using this licensed technology including Beta-thalassemia, Fabry disease, as well as inherited forms of neutropenia, thrombocytopenia, and anemias," noted Tim Miller, Ph.D., President & CEO. "We're very pleased and honored to be collaborating with the world class research being conducted by Dr. Jakub Tolar and his colleagues at the University of Minnesota, as well as patient groups and regulators, to accelerate the development of a treatment for this devastating disease." Fanconi anemia is a rare (1 in 160,000) pediatric, autosomal recessive (inherited) disease characterized by multiple physical abnormalities, organ defects, bone marrow failure, and a higher than normal risk of cancer. The average lifespan for people with FA is 20 to 30 years. As yet, no specific treatment is known that can halt or reverse the symptoms of FA. "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."