RARECast: Addressing Life-Threatening Rare Diseases with Gene Therapy


The emergence of gene therapy is giving hope of new treatments for rare diseases. Abeona Therapeutics is one of the burgeoning generation of therapeutics companies working to address life-threatening rare diseases with this new therapeutic approach. We spoke to Michelle Berg, Vice President of Patient Advocacy for Abeona, about the company, its pipeline, and the future gene therapy holds for addressing rare diseases.

Abeona Therapeutics, Inc. is a proud sponsor of this years Tribute to Champions of Hope Gala. Abeona is focused on developing and delivering 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™. For more information, visit www.abeonatherapeutics.com.

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