Abeona Therapeutics (ABEO) Reports Publication of Preclinical Data Supporting Clinical Trials for ABO-201

October 7, 2016 9:28 AM EDT
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Abeona Therapeutics Inc. (NASDAQ: ABEO):

  • Preclinical efficacy data demonstrated corrected motor deficits, attenuated neuroinflammation and reduced lysosomal throughout the central nervous system (CNS) and peripheral organs
  • A single, intravenous injection demonstrated dose-dependent benefits in the central nervous system that remained over one year post-injection
  • Results from this study are the first demonstration of successful systemic administration to restore CLN3 in vivo using scAAV9

Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing products for life-threatening rare genetic diseases, announced today that preclinical data supporting clinical trials for ABO-201 (AAV-CLN3), the AAV-based single intravenous gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL), were published in the September issue of the Journal of Neuroscience (doi: 10.1523/JNEUROSCI.1635-16.2016).

"This is the first demonstration of a systemic delivery route to restore CLN3 function, and highlights the importance of selecting the right vector, promoter and delivery route to treat the symptoms of this devastating disease," said Tammy Kielian, Ph.D., lead scientific investigator and Professor of the Department of Pathology and Microbiology, University of Nebraska Medical Center.

Researchers concluded that a single intravenous injection "led to widespread virus biodistribution in the brain, spinal cord, and eye" that was capable of "improving motor function, attenuating microglial and astrocyte activation, and reducing lysosomal pathology, all hallmarks of JNCL" at an age when significant lysosomal pathology had already manifested.

"The data support the clinical translation of ABO-201 for patients with juvenile Batten disease, and demonstrates AAV delivery to target tissues in the central nervous system as well as peripheral organs led to resolution of the underlying disease pathology," stated Timothy J. Miller, Ph.D., President & CEO. "Using a single, intravenous injection to treat the underlying lysosomal storage disease pathology mirrors our clinical trial approach for the treatment of patients with Sanfilippo syndromes type A and B."

The publication article can be accessed by clicking on the following link: (http://www.jneurosci.org/content/36/37/9669.short).



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