Abeona Therapeutics Inc., a company specializing in cell and gene therapy using adeno-associated virus (AAV) technology, and Beacon Therapeutics, a Syncona-backed firm focusing on ophthalmic gene therapies, have announced a collaboration. This partnership involves Beacon evaluating Abeona's patented AAV204 capsid for the development and potential commercialization of gene therapies targeting specific eye conditions.

Dr. Madhav Vasanthavada, Abeona's Chief Commercial Officer, emphasized AAV204's potential to efficiently target novel gene therapies for both rare and common ophthalmic diseases. AAV204, sourced from the AIM™ capsid library licensed from the University of North Carolina at Chapel Hill, has demonstrated significant transduction capabilities in the macular and optic nerve regions through para-retinal administration. It has also shown promise in transducing both the inner and outer layers of the retina through intravitreal administration in animal models.

Beacon Therapeutics, expressed excitement about evaluating AAV204's ability to target various layers of the retina, aiming to develop therapies for multiple retinal diseases with substantial unmet medical needs.

Under the agreement, Beacon has a 12-month evaluation period for AAV204, with an option to obtain a worldwide, non-exclusive license for up to five gene or disease targets. Beacon also has the option to pursue up to four additional nominated gene or disease targets under specified conditions. Abeona will receive an upfront payment upon Beacon exercising its licensing option for AAV204, along with milestone payments linked to development, regulatory approvals, and sales, as well as royalties on global net sales of licensed products featuring AAV204. Beacon will oversee the development and commercialization of all licensed products, focusing on targets distinct from Abeona's current pipeline.

The AIM™ capsid library includes various novel AAV serotypes designed to deliver genetic payloads to critical tissues implicated in severe genetic disorders, such as the central nervous system, lungs, eyes, muscles, and liver, potentially offering improved tropism profiles. 

 

Source: globenewswire.com

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