Capsida Biotherapeutics, a gene therapy startup that has quietly amassed a network of Big Pharma partnerships, announced in January that its partner AbbVie will exercise its option on their first neurodegenerative disease program, triggering a $40 million license payment. The five-year-old company, which has built its war chest primarily through pharma collaborations rather than traditional venture funding, has secured major deals with AbbVie, Eli Lilly, and CRISPR Therapeutics. “We’ve raised money almost exclusively through pharma partnering… unusual for a five-year-old biotech,” says Peter Anastasiou, CEO of Capsida Biotherapeutics. The AbbVie decision follows successful primate studies using Capsida’s engineered AAV capsids, which demonstrated broad neuronal expression while avoiding liver and dorsal root ganglia targeting.
Capsida’s collaboration with AbbVie, forged in 2021 and focused on neurodegenerative diseases, laid the foundation for this new milestone. “We delivered on what they expected, and because we delivered on it, they’re taking the program forward,” Anastasiou explains, adding that “it’s great validation of the work we do.”
Capsida Biotherapeutics’ initial partnership with AbbVie included an $80 million upfront payment and a $10 million equity investment. This deal later expanded into ophthalmology programs in 2023 with an additional $70 million. In January 2023, Capsida also secured a pact with Eli Lilly’s subsidiary, Prevail Therapeutics, which provided $55 million upfront and the potential for up to $685 million in milestones. To date, Capsida has raised more than $265 million in total.
Despite the broader ongoing biotech volatility, Capsida is rooted in an unconventional yet promising blueprint. The company’s engineered capsids target the central nervous system with unprecedented precision. “We’re not just using naturally occurring viruses like AAV9 that have a lot of challenges,” Anastasiou says. “We’re engineering the virus—the capsids—to address those challenges.”
Anastasiou says the company has “a clear set of tasks ahead.” It must “deliver on our INDs, get our clinical trials set up and executed, and meet our partners’ expectations.” While acknowledging continued uncertainty in biotech, he notes if the company hits its objectives, “we’ll be fine regardless of the market environment.”
Anastasiou, who joined after leadership stints at Lundbeck, BMS, and Lilly, felt drawn to Capsida’s vision of potentially disease-modifying therapies rather than incremental improvements. “Toward the last few years [at my previous company], the natural life cycle of drug development hits you,” he recalls. Drawn to entrepreneurship, he said: “I love and am driven by building and creating. At this stage, I wanted to do something transformative,” and Capsida “definitely has transformative potential.”
In addition to bolstering its partnerships with AbbVie and Lilly, Capsida is advancing a pipeline of CNS programs—from a gene therapy for STXBP1 developmental epileptic encephalopathy to another aiming to slow the progression of GBA-linked Parkinson’s disease. With multiple INDs on the horizon, the company aims to prove that its tailored capsid technology, combined with strong pharma alliances, can reshape CNS treatment where conventional therapies have struggled to gain ground.
Capsida’s origins trace back to Caltech in 2019, when Nick Flytzanis, Ph.D. and Dr. Nick Goeden, Ph.D. unveiled an approach to engineer custom AAV capsids capable of crossing the blood-brain barrier with precision. Now, armed with a $50 million Series A from Versant Ventures and Westlake Village BioPartners, the fledgling company drew Peter Anastasiou—formerly of Lundbeck, BMS, and Lilly—who was motivated by the promise of “something transformative” for CNS diseases. “The brain has been kind of like the last organ… we still haven’t fully figured it out,” Anastasiou explained. But there is clearly promise in unravelling its mysteries.
Filed Under: Neurological Disease
