Beacon Therapeutics exercises option to license Abeona Therapeutics’ AAV204 capsid

(image Credit: AdobeStock/dikushin)

Beacon Therapeutics has exercised its option agreement to license Abeona Therapeutics’ patented AAV204 capsid for use in potential gene therapies for a range of prevalent and rare retinal diseases that result in blindness.

This worldwide, non-exclusive license is pursuant to the agreement between Abeona and Beacon that was announced in July 2024 to evaluate the therapeutic potential of AAV204.

Under the terms of that agreement, Beacon was given the right to evaluate the AAV204 capsid for potential use in certain ophthalmology indications for a 12-month period. Additionally, 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.

Madhav Vasanthavada, PhD, chief commercial officer and head of business development at Abeona Therapeutics, commented on the agreement, saying, “Beacon’s option exercise further validates AAV204’s potential to enable targeted delivery of gene therapies in rare and prevalent ophthalmic diseases. Our non-exclusive agreement with Beacon enables us to fully explore the therapeutic value of AAV204 in additional ophthalmic diseases.”

AAV204 is a novel adeno-associated virus (AAV) capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill. AAV204 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.¹

The AIM capsid library is a collection of novel AAV serotypes that target the delivery of genetic payloads to key tissues implicated in devastating genetic diseases

References:

1. Abeona Therapeutics announces option exercise by Beacon Therapeutics for novel AAV204 capsid for ophthalmology gene therapy. Published July 1, 2025. Accessed July 1, 2025. https://www.globenewswire.com/news-release/2025/07/01/3108229/0/en/Abeona-Therapeutics-Announces-Option-Exercise-by-Beacon-Therapeutics-for-Novel-AAV204-Capsid-for-Ophthalmology-Gene-Therapy.html