Abeona and Beacon Announce Agreement to Evaluate AAV204 Capsid for Eye Treatments

CLEVELAND and LONDON, July 11, 2024 – Abeona Therapeutics Inc. (Nasdaq: ABEO), a company specializing in cell and gene therapies, and Beacon Therapeutics, an ophthalmic gene therapy firm within the Syncona portfolio, have announced a collaboration. This partnership will see Beacon evaluate Abeona's proprietary AAV204 capsid for potential gene therapies aimed at specific ophthalmology conditions.

Dr. Madhav Vasanthavada, Chief Commercial Officer at Abeona, highlighted the potential of AAV204 in targeting eye diseases with considerable unmet medical needs. He expressed optimism about the collaboration with Beacon, which aims to deliver innovative AAV-based therapies for such conditions.

The AAV204 capsid, part of the AIM™ capsid library that Abeona licensed from the University of North Carolina at Chapel Hill, has shown promising results in animal studies. It has demonstrated high transduction levels in the macula and optic nerve following para-retinal administration. Additionally, it has successfully transduced both inner and outer retinal layers after intravitreal administration in both mice and non-human primates.

Abraham Scaria, Chief Scientific Officer at Beacon Therapeutics, also emphasized the importance of this partnership. He noted that they are eager to explore AAV204's capability to transduce different retinal layers, which could lead to the development of therapies for various retinal diseases with significant unmet needs.

According to the agreement, Beacon will have a 12-month period to evaluate the AAV204 capsid for its applications in certain ophthalmology indications. They hold the option to secure a worldwide, non-exclusive license to use AAV204 for up to five gene or disease targets. Additionally, Beacon can nominate up to four extra gene or disease targets under specific conditions. Abeona will receive an upfront payment if Beacon exercises its option to license AAV204, followed by milestone payments based on development, regulatory, and sales achievements, and tiered royalties on global net sales of products incorporating AAV204. Beacon will handle the development and commercialization of all licensed products while ensuring that the targets under this agreement do not overlap with those currently being pursued by Abeona.

The AIM™ capsid library, which includes novel AAV serotypes, targets the delivery of genetic payloads to crucial tissues affected by severe genetic diseases, such as the central nervous system, lungs, eye, muscle, liver, and other tissues. These vectors have shown a potential advantage in evading immune responses generated by natural AAV exposure in preclinical studies. AAV204 is protected by U.S. Patent Nos. 10,532,110 and 10,561,743.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company that develops cell and gene therapies for severe diseases. One of its key projects includes prademagene zamikeracel (pz-cel), designed for treating recessive dystrophic epidermolysis bullosa. This investigational therapy, developed in Abeona’s cell and gene therapy manufacturing facility, is poised for commercial production upon FDA approval. Abeona's pipeline also includes AAV-based gene therapies targeting eye diseases with high unmet needs, utilizing novel capsids to enhance disease-specific targeting.

Beacon Therapeutics, founded in 2023, focuses on ophthalmic gene therapy with the mission to prevent and restore vision loss caused by various retinal diseases. Beacon's portfolio includes a late-stage candidate for X-linked retinitis pigmentosa and two preclinical programs targeting dry age-related macular degeneration and cone-rod dystrophy. The company is backed by Syncona, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences, and other investors.