For gene therapy to achieve its potential, biotech companies need to diversify the range of tissues that can be targeted by these treatments. Capsida Biotherapeutics is developing technology that could enable delivery of a gene therapy to cells of the central nervous system. Now Eli Lilly is paying $55 million to kick off a multi-drug R&D alliance, making it the latest pharmaceutical industry partner to sign on with the startup.
The agreement announced Wednesday is with Lilly’s gene therapy subsidiary, Prevail Therapeutics. In addition to the $55 million upfront payment, Prevail agrees to participate in the next financing round of Thousand Oaks, California-based Capsida. Prevail’s payments for research and development as well as milestones could bring the startup up to $685 million more.
Gene therapies are mainly delivered to their cellular destinations carried aboard adeno-associated viruses (AAV). These engineered viruses preferentially target liver cells. Capsida works with capsids, the protein shells that envelop a gene therapy. Engineering capsids in certain ways is intended to optimize their ability to target particular tissues.
Capsida, based on technology from the California Institute of Technology, uses machine learning to screen engineered capsids in order to find the ones that can address desired tissue types, such as those of the central nervous system (CNS). The biotech claims it can engineer capsids that target a single organ or multiple organs simultaneously while also limiting exposure to organs that are not targets of the therapy. That capability could improve the safety of AAV-delivered gene therapies. Delivery to non-target cells can trigger adverse effects.
Specific disease targets covered by the pact were not disclosed. But the deal calls for Prevail to use Capsida’s technology to identify and develop capsids that are paired with genetic cargo from Prevail. That resulting gene therapies will be directed to targets that are known to cause CNS disorders.
The deal gives Capsida the option to participate in the development and commercialization of one of the partnered programs. For all of the programs covered under the alliance, Capsida will take the lead on capsid discovery. Prevail is responsible for preclinical research and studies of the gene therapy candidates with the therapeutic payloads.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“Prevail’s expertise in neuroscience, gene therapy R&D, and access to Lilly’s world-class commercialization capabilities complements Capsida’s fully integrated approach, including our next-generation AAV engineering platform,” Capsida CEO Peter Anastasiou said in a prepared statement. “Prevail and Capsida are committed to developing highly effective and safe gene therapies that have the potential to be transformative for patients living with serious CNS diseases.”
The agreement with Prevail is similar to a deal Capsida has with AbbVie. That alliance unveiled two years ago covers the development of gene therapies for three undisclosed CNS targets. AbbVie paid Capsida $80 million up front and made a $10 million equity investment in the startup. Amyotrophic lateral sclerosis and Friedreich’s ataxia are the disease targets for Capsida’s alliance with CRISPR Therapeutics.
Lilly has become a more active dealmaker in its effort to beef up its gene therapy prospects. Prevail joined Lilly two years ago via an $880 million acquisition. A Parkinson’s disease gene therapy is Prevail’s most advanced program. Late last year, Indianapolis-based Lilly paid $487 million to acquire Akouos, a biotech developing a gene therapy for an inherited form of hearing loss.
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