Beacon Therapeutics, an ophthalmic gene therapy company and Syncona portfolio company, has exercised its option to license Abeona Therapeutics' patented AAV204 capsid technology for developing gene therapies targeting a range of prevalent and rare retinal diseases that result in blindness. The worldwide, non-exclusive license follows a year-long evaluation period that began with an agreement announced in July 2024.
Novel AAV Capsid Shows Superior Retinal Targeting
AAV204 represents a breakthrough in gene therapy delivery systems for ophthalmology applications. The novel AAV capsid, sourced from the AIM™ capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has demonstrated exceptional targeting capabilities in preclinical studies. Research has shown that AAV204 achieves high macular and optic nerve transduction levels after para-retinal administration and facilitates transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates.
"Beacon's option exercise further validates AAV204's potential to enable targeted delivery of gene therapies in rare and prevalent ophthalmic diseases," said Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development at Abeona Therapeutics. "Our non-exclusive agreement with Beacon enables us to fully explore the therapeutic value of AAV204 in additional ophthalmic diseases."
Licensing Terms and Commercial Potential
The license agreement grants Beacon the right to use AAV204 in connection with up to five gene or ophthalmology disease targets. Under the terms of the agreement, Abeona will receive an undisclosed upfront license payment 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.
The exercise of this license option concludes Beacon's initial evaluation of the AAV204 capsid for development and commercialization of gene therapies, marking a significant milestone in the translation of this technology from research to potential clinical applications.
Advanced Gene Therapy Platform Technology
The AIM™ capsid library represents a collection of novel AAV serotypes designed to target delivery of genetic payloads to key tissues implicated in devastating genetic diseases, including the central nervous system, retina, lungs, eye, muscle, liver and other tissues, with potentially improved tropism profiles. Notably, AIM™ vectors have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors in preclinical studies.
AAV204 is protected by U.S. Patent Nos. 10,532,110 and 10,561,743, providing intellectual property coverage for this innovative gene therapy delivery platform.
Abeona's Expanding Gene Therapy Portfolio
Abeona Therapeutics is a commercial-stage biopharmaceutical company with an established track record in cell and gene therapies. The company's ZEVASKYN™ (prademagene zamikeracel) represents the first and only autologous cell-based gene therapy for treating wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).
The company operates a fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio, which serves as the manufacturing site for ZEVASKYN commercial production. Abeona's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need, with novel, next-generation AAV capsids being evaluated to improve tropism profiles for various devastating diseases.
