Beacon Therapeutics Ltd. and Abeona Therapeutics Inc. have entered into a non-exclusive licensing agreement for Beacon to assess Abeona’s patented adeno-associated virus (AAV)-based capsid for the potential development and commercialization of ophthalmic disease-targeted gene therapies.
First, let’s discuss these players.
Beacon Therapeutics is an ocular gene therapy company launched in June 2023 by Syncona, a life science investments healthcare company.
- Its focus: Using AAV-centered gene therapy to perform clinical research on three investigational candidates being studied for restoring and improving vision in patients with prevalent and rare blinding retinal diseases:
- X-linked retinitis pigmentosa (XLRP)
- Dry age-related macular degeneration (AMD)
- Cone-rod dystrophy (CRD)
Abeona Therapeutics, a clinical-stage biopharmaceutical company launched in 2015, is also developing AAV-based gene therapies for ophthalmic diseases with high unmet needs (although the ophthalmology sector isn’t the pipeline’s only focus).
Sticking with Abeona… talk about the basis for these gene therapies.
The foundation for the company’s next-generation AAV capsids is its in-license AIM capsid library.
What it does: Uses AAV biology to selectively target delivery of genetic payloads (AIM vectors)—with multiple routes of delivery—to areas such as the eye, central nervous system (including the retina), lungs, muscle, and liver.
- Note: Per Abeona, these AIM vectors are not only non-replicating, but have also “shown the potential to evade the immune responses generated by exposure to naturally occurring AAV vectors.”
- The intended result: Potential for redosing patients previously treated with AAV-based gene therapies
Now this AAV capsid.
AAV204 is a novel AAV capsid from the AIM capsid library licensed by Albeona from the University of North Carolina (UNC) at Chapel Hill.
- How it works: The capsid has been clinically studied in facilitating the transduction of both the inner and outer retina following administration via a modified intravitreal injection (i.e., para-retinal injection) in mice and non-human primates (animals).
Note: AAV204 is covered by the U.S. Patent Nos. 10,532,110 B2 and 10,561,743 B2.
Back up: Explain this type of administration.
Unlike subretinal administration, intravitreal injections are designed to be potentially both less complex and traumatic to ocular tissues—however, it does provide limited retinal transduction, research notes.
With AAV vectors, their large size (vs most intraocular therapeutics) often means an injection is unable to reach the target location within the anterior to mid-vitreous.
Enter: Para-retinal administration, a form of intravitreal administration capable of injecting deep into the vitreous without requiring localized retinal detachment of the photoreceptors from the retinal pigment epithelium (RPE).
Gotcha. Now back to AAV204’s clinical data.
Reported during the 2022 Association for Research and Vision in Ophthalmology (ARVO) annual meeting, the study involved four non-human primate eyes that were para-retinally administered either AAV204 green fluorescent protein (GFP) or AAV8 GFP.
- The details: GFP expression was monitored via scanning laser ophthalmoscope (SLO) and was followed by immunohistochemistry analysis (conducted at 28 days post-injection).
Alrighty, so what will Beacon do with this capsid?
The company will conduct a 12-month evaluation of AAV204 for potential use in specific retinal disease indications, according to the agreement. Other terms enable Beacon to:
- Have the option to use a global, non-exclusive license for AAV204 in connection to up to five gene/disease targets
- Use AAV204 for up to four additional gene or disease targets (pending certain conditions)
And in return?
Per the agreement, Abeona will receive an upfront payment once Beacon chooses to exercise its AAV204 licensing option—as well as additional payments following certain development, regulatory, and sales milestone achievements.
- Plus: The company will also be granted tiered royalties on global net sales for licensed products that incorporate AAV204.
About those licensed products…
Before you ask: Beacon will be responsible for both the development and commercialization of all these licensed products.
- As for indications: Beacon noted that it would receive the rights for indications that are “distinct from those currently in development at Abeona.”
Any input from the companies?
Abeona Chief Commercial Officer (COO) and Head of Business Development Madhav Vasanthavada, PhD, MBA, stated that the agreement “underscores the potential of AAV204 to enable efficient targeting in the eye of novel AAV-based gene therapies for patients with rare and prevalent ophthalmic diseases with high unmet medical need.”