Capsida teams up with Lilly subsidiary for gene therapies

Capsida Biotherapeutics, a gene therapy start-up, will team up with Eli Lilly and Company subsidiary Prevail Therapeutics to develop gene therapies for diseases affecting the central nervous system (CNS). Prevail will pay Capsida $55 million in the form of an up-front payment and a commitment to participate in Capsida’s next financing round, and Capsida may get up to $685 million in milestones as well as royalties.

Under the agreement, Capsida will head efforts to engineer suitable adeno-associated virus (AAV) capsids, or protein shells, that will ferry Prevail’s therapeutic cargo to targets within the body.

A challenge for treating CNS conditions with gene therapy is getting enough of the treatment across the blood-brain barrier, says Capsida’s CEO Peter Anastasiou. Almost all CNS-directed gene therapies are delivered through invasive procedures such as injection into the cerebrospinal fluid. And high doses that try to get more drug to the brain can end up in the wrong place, like the liver, he says. Capsida is engineering its capsids to tackle several problems at once; to cross the blood-brain barrier with intravenous delivery, while limiting crossing into unintended organs, he says.

The company employs high-throughput screening to find capsid variants with the desired specificity and that are able to be administered to the body through the intended route. It also engineers its capsids primarily in nonhuman primates, which translates to humans more readily than engineering in mice does, Anastasiou says.

Finding or engineering better viral vectors is a focus of companies in the gene therapy field. For instance, Massachusetts-based Affinia Therapeutics is also engineering AAV capsids and struck a deal with Vertex Pharmaceuticals in 2020 to make capsids for a variety of treatments. Companies like Carbon Biosciences and Kelonia Therapeutics, which launched last year, have moved beyond AAVs to other types of viral vectors. Upon its launch in 2021, Capsida also began a partnership with Abbvie to develop gene therapies for three CNS disease targets.