Date: 2016-09-21
Type of information: Licensing agreement
Compound: AIM ™ vector system
Company: Abeona Therapeutics (USA - NY) The University of North Carolina (USA - NC)
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases - Neurodegenerative diseases
Type agreement: licensing
Action mechanism: gene therapy. 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.
Disease: Batten disease
Details: * On September 21, 2016, Abeona Therapeutics announced the exclusive worldwide license of a next generation gene therapy AAV capsid portfolio from University of North Carolina at Chapel Hill. 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 (AAV-CLN1) , 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).
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