Beacon Will Evaluate Abeona’s AAV204 Capsid for Select Ophthalmic Indications

Beacon Therapeutics will evaluate Abeona Therapeutics’ patented AAV204 capsid for potential gene therapies for select ophthalmic indications, the companies announced July 11.

AAV204, a novel AAV capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has been shown to achieve high levels of genetic material transfer, or transduction, in the macula and optic nerve after para-retinal administration. It has also been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates.

No financial details for the partnership were disclosed. Under the agreement, Beacon will have the right to evaluate, for a year, AAV204 for potential use in certain ophthalmic indications, with an option to take a worldwide, non-exclusive license to use AAV204 in connection with up to five gene or disease targets.

Beacon will also have the right to use AAV204 for up to four additional nominated gene or disease targets, subject to certain conditions, the companies said. Abeona will receive an upfront payment upon Beacon’s exercise of its option, 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.

Beacon will be responsible for the development and commercialization of all licensed products. The targets for which Beacon would receive rights are distinct from those in development at Abeona, the companies said.

Beacon, with offices in London and the US, is developing AGTC-501, a subretinal gene therapy candidate for X-linked retinitis pigmentosa.

In March 2023, Abeona unveiled a preclinical gene therapy pipeline targeting Stargardt, X-linked retinoschisis, and autosomal dominant optic atrophy. It is based in Cleveland, Ohio.