Abeona Therapeutics Inc., a cell and gene therapy company with proprietary adeno-associated virus (AAV)-based capsids, and Beacon Therapeutics, an ophthalmic gene therapy company and Syncona portfolio company, announced an agreement by which Beacon will evaluate Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology indications.
“This agreement with Beacon underscores the potential of AAV204 to enable efficient targeting in the eye of novel AAV-based gene therapies for patients with rare and prevalent ophthalmic diseases with high unmet medical need, and we are looking forward to a fruitful collaboration,” said Dr. Madhav Vasanthavada, chief commercial officer and head of Business Development at Abeona Therapeutics.
AAV204, a novel AAV capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has been shown to achieve high macular and optic nerve transduction levels after para-retinal administration and has also been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates.
Abraham Scaria, chief scientific officer of Beacon Therapeutics, said, “We are looking forward to evaluating AAV204 for its ability to transduce various layers of the retina in order to develop therapies for multiple retinal diseases with high unmet need.”
Under the terms of the agreement, Beacon will have the right to evaluate, for a 12-month period, the AAV204 capsid for potential use in certain ophthalmology indications, with an option to take a worldwide, non-exclusive license to use AAV204 in connection with up to five gene or disease targets.
Beacon will also have the right to use AAV204 for up to four additional nominated gene or disease targets subject to certain conditions.
Under the agreement, Abeona will receive an upfront payment upon Beacon’s exercise of its option to license AAV204, with additional payments upon the achievement of certain development, regulatory, and sales milestones, along with tiered royalties on worldwide net sales for licensed products incorporating AAV204. Beacon will be responsible for the development and commercialization of all licensed products.
The targets for which Beacon would receive rights under its non-exclusive license agreement are distinct from those currently in development at Abeona.
About the AIM capsid library The AIM capsid library is a collection of novel AAV serotypes that target delivery of genetic payloads to key tissues implicated in devastating genetic diseases, including the central nervous system (including the retina), lungs, eye, muscle, liver and other tissues, with potentially improved tropism profiles.
AIM vectors have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors in preclinical studies. AAV204 is covered by U.S. Patent Nos. 10,532,110 and 10,561,743.