brain x-ray image

x-ray image of the brain

For gene therapy to achieve its potential, biotech companies must diversify the set of tissues that can be targeted for these treatments. Capsida Biotherapeutics is developing technology that could enable the delivery of gene therapy to cells of the central nervous system. Eli Lilly is now paying $55 million to start a a research and development alliance for various drugsmaking it the latest pharmaceutical industry partner to join the startup.

The agreement announced Wednesday is with Lilly’s gene therapy subsidiary, Prevail Therapeutics. In addition to the $55 million upfront payment, Prevail has agreed to participate in Thousand Oaks, California-based Capsida’s next round of financing. Prevail’s R&D and milestone payments could bring the startup up to $685 million more.

Gene therapies are mainly delivered to their cellular destinations carried aboard adeno-associated viruses (AAVs). These engineered viruses primarily target liver cells. Capsida works with capsids, the protein shells that envelop gene therapy. Designing capsids in certain ways aims to optimize their ability to target specific tissues.

Capsida, based on technology from Caltech, uses machine learning to screen engineered capsids to find those that can target desired tissue types, such as those of the central nervous system (CNS). The biotech claims it can design capsids that target a single organ or multiple organs simultaneously, while limiting exposure to organs that are not the therapy’s targets. This ability may improve the safety of AAV-delivered gene therapies. Delivery to non-target cells can cause adverse effects.

The specific disease targets covered by the pact were not disclosed. But the deal requires Prevail to use Capsida’s technology to identify and develop capsids that are paired with genetic cargo from Prevail. The resulting gene therapies will target targets known to cause CNS disorders.

The deal enables Capsida to participate in the development and commercialization of one of the partner programs. For all programs covered by the alliance, Capsida will take the lead in capsid discovery. Prevail is responsible for preclinical research and studies of gene therapy candidates with therapeutic payloads.

“Prevail’s neuroscience, gene therapy and development expertise and access to Lilly’s world-class commercialization capabilities complement Capsida’s fully integrated approach, including our next-generation AAV engineering platform,” said Capsida CEO Peter Anastasiou in 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. This alliance, revealed 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 targets of Capsida disease alliance with CRISPR Therapeutics.

Lilly has become a more active marketer in its efforts to improve its prospects for gene therapy. Prevail joined Lilly two years ago through an Acquisition for $880 million. Parkinson’s disease gene therapy is Prevail’s most advanced program. Late last year, based in Indianapolis Lilly paid $487 million to acquire Akouos, a biotech developing gene therapy for an inherited form of hearing loss.

Photo: Jolygon, Getty Images

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