Company Update (NASDAQ:PTBI): Plasmatech Biopharmaceuticals Inc Announces License Agreement For AAV Gene Therapy To Treat Juvenile Batten Disease

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 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. (Original Source)

Shares of Plasmatech Biopharmaceuticals opened today at $7.98 and are currently trading down at $7.7634. PTBI has a 1-year high of $8.20 and a 1-year low of $2.88. The stock’s 50-day moving average is $6.61 and its 200-day moving average is $4.63.

On the ratings front, H.C. Wainwright analyst Andrew Fein initiated coverage with a Buy rating on PTBI and a price target of $15, in a report issued on May 18. The current price target implies an upside of 93.3% from current levels.

According to, Fein has a total average return of 26.7%, a 66.2% success rate, and is ranked #109 out of 3612 analysts.

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