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Abeona Therapeutics® Announces Option Exercise by Beacon Therapeutics for Novel AAV204 Capsid for Ophthalmology Gene Therapy

Core Insights - Abeona Therapeutics has announced that Beacon Therapeutics has exercised its option to license Abeona's patented AAV204 capsid for potential gene therapies targeting various retinal diseases, which could lead to significant financial benefits for Abeona through license payments and royalties [1][3]. Group 1: Licensing Agreement - The licensing agreement with Beacon Therapeutics is worldwide and non-exclusive, allowing Beacon to develop gene therapies for up to five ophthalmic disease targets using AAV204 [1][3]. - Abeona will receive an undisclosed upfront license payment, along with additional payments tied to development, regulatory, and sales milestones, plus tiered royalties on worldwide net sales of products incorporating AAV204 [3]. Group 2: AAV204 Capsid Details - AAV204 is a novel AAV capsid from the AIM™ capsid library, which has demonstrated high transduction levels in the macula and optic nerve after administration in preclinical studies [2][4]. - The AIM™ capsid library includes various AAV serotypes designed to improve delivery to tissues affected by genetic diseases, potentially evading immune responses [4]. Group 3: Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including its autologous cell-based gene therapy ZEVASKYN™ for treating recessive dystrophic epidermolysis bullosa [5]. - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which supports the commercial production of ZEVASKYN [5].