Meet Abeona Therapeutics: A Biotech Champion for Sanfilippo, Juvenile Batten and Fanconi Anemia

August 5, 2015

Patients are looking towards Abeona Therapeutics for leading the search for answers on Sanfilippo Syndrome A & B, Juvenile Batten Disease,and  Fanconi Anemia. The company, who recently changed names from PLASMATECH, is focused on developing gene therapy and plasma-based products for severe and life threatening rare diseases.

Abeona’s lead program is an adeno-associated virus (AAV)-based 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 an AAV-based gene therapy program in juvenile Batten disease (JBD) from the University of Nebraska Medical Center; and licensed an AAV-based gene therapy program for Fanconi anemia (FA) disorder and other rare blood diseases using CRISPR/Cas9 gene editing from the University of Minnesota. 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™.

Want to learn more about what’s coming from Abeona? For more information, visit

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