Abeona Therapeutics Inc., a New York City and Cleveland-based clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, announced the exclusive worldwide license of a next generation gene therapy AAV capsid portfolio from University of North Carolina at Chapel Hill. The AIM™ vector system is a next generation platform of AAV capsids capable of widespread central nervous system gene transfer and can be used to confer high transduction efficiency for various therapeutic indications. Studies indicate that AIM vectors can efficiently and broadly target CNS tissue, and may provide a treatment for patients that have inhibitory antibodies to natural AAV serotypes. Importantly, the AIM vector system may provide second-generation treatment approaches for patients that have received a previous AAV injection.
“As we continue to build out our orphan and rare disease drug portfolio and move additional programs into the clinic, this agreement with UNC continues the execution of our strategy to combine our expertise in advancing gene therapy programs with the development of a next-generation proprietary AAV vector platform,” stated Steven H. Rouhandeh, Executive Chairman. “We look forward to harnessing the clinical utility and therapeutic potential of the AIM vector system technology platform to address a broad range of rare genetic diseases.”
In addition to the AAV capsid library, the license also adds ABO-202, an AAV-based CLN1 program, to Abeona’s Batten pipeline. ABO-202, developed at UNC by Steven Gray, Ph.D. with the support of The Saoirse Foundation, Taylor’s Tale, Hayden’s Batten Disease Foundation, and the Batten Disease Support and Research Association, is anticipated to enter clinical trials in 2017 for patients with infantile neuronal ceroid lipofuscinosis (INCL, infantile Batten disease), an inherited fatal genetic disease that primarily affects the nervous system.
“ABO-202 has shown promising preclinical efficacy in INCL mice after delivery of a functioning copy of the CLN1 gene to cells of the central nervous system, by extending survival and preserving strength when administered early in the disease course,” noted Steven J. Gray, Ph. D, Assistant Professor, Department of Ophthalmology, Gene Therapy Center, University of North Carolina at Chapel Hill. “Our work in developing these novel, next generation AAV gene therapy vectors have the potential to further advance the field of AAV-based technologies by efficiently and specifically targeting the CNS, with a likelihood of avoiding antibodies endogenously generated by natural AAV serotypes.”
“The AIM vector system is a next generation AAV-based gene therapy technology platform that represents a transformational opportunity for Abeona. The AIM platform will allow us to leverage our current pipeline into second generation products for CNS and other tissue-specific delivery, and help provide an answer for patients that have existing inhibitory antibodies,” stated Timothy J. Miller, Ph.D., President & CEO. “In addition, we add another AAV-based product ABO-202 (AAV-CLN1) for treatment of patients with infantile neuronal ceroid lipofuscinosis (INCL), which builds on our expertise in developing treatments for patients with forms of Batten disease.”
