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

NEW YORK, NY and CLEVELAND, OH--(Marketwired - June 15, 2015) - PlasmaTech Biopharmaceuticals, Inc. (NASDAQ: PTBI)

  • CRISPR-Cas9 Gene-Editing System Demonstrated Promising Efficacy in Repairing the FANCC Gene in Human Fibroblasts
  • Preclinical Studies Have Demonstrated Promising Efficacy for Treating Fanconi Anemia Disorder, a Rare, Fatal Autosomal Recessive Disease
  • Establishes Technology Platform for Additional Rare Blood Disease Pipeline Products

PlasmaTech Biopharmaceuticals, Inc. (NASDAQ: PTBI), 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."

About Fanconi anemia (FA) disorder: 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.

The major function of bone marrow is to produce new blood cells. In FA, a DNA mutation renders the FANCC 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 (AML) 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 (BMT) there are no specific treatments known that can halt or reverse the symptoms of FA. Repairing fibroblast cells in FA patients with a functional FANCC gene is the focus of our AAV-based gene therapy approach.

About CRISPR-Cas9: The CRISPR (clustered, regularly interspaced short palindromic repeats)-Cas9 (CRISPR associated protein 9) system, the newest genome editing approach, uses a protein-RNA complex composed of an enzyme known as Cas9 bound to a guide RNA molecule that has been designed to recognize a particular DNA sequence. The RNA molecules guide the Cas9 complex to the location in the genome that requires repair. CRISPR-Cas9 uniquely enables surgically efficient knock-out, knock-down or selective editing of defective genes in the context of their natural promoters, unlocking the potential to treat both recessive and dominant forms of genetic diseases. Most importantly, this approach has the potential to allow safer, more precise gene modification.

About University of Minnesota: Founded in 1851, the University of Minnesota is ranked among the nation's top public research universities. As a land-grant institution, the U of M is committed to engaging Minnesota, national and global communities to advance interdisciplinary knowledge; enhance students' academic, civic, career, social and personal development; and apply intellectual and human capital to serve the public good.

About PlasmaTech Biopharmaceuticals: PlasmaTech Biopharmaceuticals is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech's lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. Clinical trials for Sanfilippo types A and B are anticipated to begin in 2015. The Company recently licensed a third gene therapy program in juvenile Batten disease from University of Nebraska Medical Center. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary alpha-1 protease inhibitor, SDF Alpha™. For more information, visit and

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, development and internationalization of clinical programs, information regarding the future performance of the combined company, the outlook on medical needs, future pipeline expectations, management plans for the Company, the anticipated closing of the transaction, and general business outlook. These statements are subject to numerous risks and uncertainties, including but not limited the satisfaction of closing conditions for the transaction, the parties' ability to successfully integrate and operate the new company, and achieve expected synergies and other benefits; the impact of competition; the ability to develop products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

Contact Information

Company and Media Contact:
Andre'a Lucca
Director of Communications
PlasmaTech Biopharmaceuticals, Inc.