PlasmaTech Biopharmaceuticals Announces License Agreement for AAV Gene Therapy to Treat Patients With Juvenile Batten Disease Print E-mail
Monday, 08 June 2015 09:13

 New York, NY (June 8, 2015) 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 for the treatment of juvenile Batten disease (JBD) from UNeMed Corporation, the technology transfer and commercialization office for the University of Nebraska Medical Center in Omaha, Nebraska. PlasmaTech is also developing AAV gene therapies for the treatment of Sanfilippo syndromes (MPS IIIA and MPS IIIB).

"This transaction further represents our commitment to our strategy of building a premier gene therapy company focusing on rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "We believe there are significant synergies among Abeona's expertise and programs and the ground breaking work from the scientists at the University Of Nebraska Medical Center, and we are excited about moving their JBD program ahead aggressively."

An inherited genetic disorder, juvenile Batten disease is a lysosomal storage disease that primarily affects the nervous system in children. After a few years of normal development, children with this condition experience progressive vision loss leading to blindness, intellectual and motor disability, speech difficulties, seizures, and premature death. As yet, there are no treatments available that can halt or reverse the symptoms of Batten disease.

"This promising addition to our AAV gene therapy pipeline leverages our experience in treating devastating lysosomal storage diseases with world class research being conducted by Tammy Kielian, Ph.D., and her colleagues at UNMC to accelerate development of a treatment for this deadly disease," said Tim Miller, Ph.D., PlasmaTech Biopharmaceuticals President & CEO.

"There are no treatments available that can halt or reverse the symptoms of children with juvenile Batten disease, but our AAV gene therapy studies have demonstrated significant and promising results in animal models of the disease," said Dr. Kielian, who is professor of pathology and microbiology at UNMC. "Working with PlasmaTech, as well as the dedicated juvenile Batten disease community of patients and their families, will help us accelerate this therapy into clinical trials."

"Juvenile Batten disease is a deadly, progressive neurological disorder that devastates the lives of children. Gene therapy represents a promising approach to treat lysosomal storage disorders like JBD and we are pleased to work with PlasmaTech to accelerate this promising therapy into clinical development," said William Rizzo, M.D., a pediatric geneticist with UNMC's Munroe-Meyer Institute, which treats children with rare genetic diseases and will be involved in the planned clinical trials.

About Juvenile Batten Disease: Juvenile Batten disease (JBD) is a rare, fatal, autosomal recessive (inherited) disorder of the nervous system that typically begins in children between 4 and 8 years of age. Often the first noticeable sign of JBD is vision impairment, which tends to progress rapidly and eventually result in blindness. As the disease progresses, children experience the loss of previously acquired skills (developmental regression). This progression usually begins with the loss of the ability to speak in complete sentences. Children then lose motor skills, such as the ability to walk or sit. They also develop movement abnormalities that include rigidity or stiffness, slow or diminished movements (hypokinesia), and stooped posture. Beginning in mid to late childhood, affected children may have recurrent seizures (epilepsy), heart problems, behavioral problems, and difficulty sleeping. Life expectancy is greatly reduced. Most people with juvenile Batten disease live into their twenties or thirties. As yet, no specific treatment is known that can halt or reverse the symptoms of juvenile Batten disease.

Juvenile Batten disease is the most common form of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs). Collectively, all forms of NCL affect an estimated 2 to 4 in 100,000 live births in the United States. NCLs are more common in Finland, where approximately 1 in 12,500 individuals are affected; as well as Sweden, other parts of northern Europe, and Newfoundland, Canada.

Most cases of juvenile Batten disease are caused by mutations in the CLN3 gene, which is the focus of our AAV-based gene therapy approach. These mutations disrupt the function of cellular structures called lysosomes. Lysosomes are compartments in the cell that normally digest and recycle different types of molecules. Lysosome malfunction leads to a buildup of fatty substances called lipopigments and proteins within these cell structures. These accumulations occur in cells throughout the body, but neurons in the brain seem to be particularly vulnerable to damage. The progressive death of cells, especially in the brain, leads to vision loss, seizures, and intellectual decline in children with juvenile Batten disease.

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. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDFT) Process and Polymer Hydrogel Technology (PHTT), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary alpha-1 protease inhibitor, SDF AlphaT. The company has developed a robust product pipeline that includes two commercial stage products, MuGardĀ® and ProctiGardT. For more information, visit

About University of Nebraska Medical Center: UNMC, with its clinical partner, Nebraska Medicine, is a leading academic medical center, perhaps best known for its treatment, at the request of the U.S. State Department, of three Ebola patients. It also is a world leader in solid organ transplants, in the development of antimalarial vaccines and is the future home of the state-of-the-art Fred & Pamela Buffett Cancer Center. A national leader in primary care education, UNMC is known for its innovative use of the latest experiential technology in training the next generation of health care professionals. With Nebraska Medicine, UNMC is dedicated to leading the world in transforming lives to create a healthy future for all individuals and communities through premier educational programs, innovative research and extraordinary patient care.

About UNeMed Corporation: UNeMed Corporation is the technology transfer and commercialization office (TTO) for the University of Nebraska Medical Center. UNeMed serves all UNMC researchers, faculty and staff who develop new biomedical technology and inventions, and strives to help bring those innovations to the marketplace.

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, those relating to: the Company's acquisition of Abeona, anticipated acceleration in the 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.

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