Capsida Biotherapeutics is stepping beyond neurodegenerative conditions, diving into the world of eye disease with an AbbVie expansion pact for three gene medicine targets.
The partnership comes one month after the gene therapy platform company signed a deal worth up to $685 million biobucks with Eli Lilly. The newest deal also builds on a 2021 neurodegenerative disease partnership with AbbVie in which the Big Pharma put down $90 million for three CNS disease targets.
This time, the pair aims to develop three targeted genetic medicines for eye diseases with high unmet need. The partnership marks Capsida’s move into ophthalmology; up until this point, the California company had been exclusively focused on neurodegenerative conditions.
“We're a platform company, so we're not limited to just the CNS space,” Capsida CEO Peter Anastasiou told Fierce Biotech. “We've always wanted to go into a new therapeutic area.”
Under the deal extension, Capsida will combine its adeno-associated virus (AAV) engineering platform with the Big Pharma’s extensive capabilities.
(Capsida Biotherapeutics)
“It's a win-win, because rather than getting into the ophthalmology space by ourselves we're doing it with the world's leader,” Anastasiou said, referencing AbbVie’s Allergan arm. “That's a great way to enter into a new therapeutic area.”
Capsida will lead capsid discovery efforts and will be responsible for process development and early clinical manufacturing, while AbbVie will lead innovative therapeutic cargo approaches, development and commercialization.
Under the terms of the deal, Capsida is set to receive $70 million in upfront payments and a potential equity investment. For the three programs, Capsida could make up to $595 million in option fees and R&D milestones, not to mention commercial milestone payments and royalties.
The company, which debuted in 2021 with a $50 million series A alongside the $90 million from the original AbbVie deal, aims to create customized therapies that target specific organ systems while limiting exposure to non-targeted organs.
Lilly is the other pharma to have come on board, committing $55 million upfront and $685 million in milestones in January for access to the biotech's AAV platform. The pact adds capabilities to Prevail Therapeutics, an AAV9-focused gene therapy business that Lilly snapped up in 2020.
AAV9 is the current vector of choice for gene therapies that cross the blood-brain barrier, but there is room to improve on the delivery vehicle. Ideally, a vector will zero in on specific tissues, maximizing therapeutic impact while minimizing off-target effects. But such a vehicle has proved difficult to find.
Capsida has shown evidence that its engineered capsids can improve on the performance of first-generation AAV9-based therapies and CEO Anastasiou believes the company has the ability to fully actualize gene therapy’s potential.
Anastasiou, who spent the first 15 years of his career at Lilly and Bristol Myers Squibb, also believes that Capsida’s development and manufacturing abilities are part of the reason other companies have been drawn to the biotech.
“If we wanted to partner more, we could’ve—we had a lot of others expressing interest,” he said. “We have enough on our plate for now and will focus on delivering on the partnerships we have.”
The company, which employees around 130 staff, is growing its team to support the new partnerships and execute its own programs.