RARE Daily

AbbVie and Capsida Expand Collaboration to Develop Genetic Medicines for Eye Diseases

February 23, 2023

Rare Daily Staff

AbbVie and Capsida Biotherapeutics said they expanded their strategic collaboration in neurodegenerative diseases to develop genetic medicines for eye diseases with high unmet need.

AbbVie’s extensive capabilities will be paired with Capsida’s novel adeno-associated virus (AAV) engineering platform and manufacturing capability to identify and advance three programs. The collaboration builds upon the neurodegenerative disease partnership announced in 2021.

“Combining AbbVie’s expertise in eye disease drug development and commercialization with Capsida’s fully integrated next-generation AAV engineering platform and manufacturing capabilities offers the potential to provide novel therapies enabling unprecedented benefit to patients with serious eye diseases,” said Peter Anastasiou, CEO of Capsida.

Under the terms of the expanded agreement, Capsida will receive $70 million, consisting of upfront payments and a potential equity investment. For the three programs, Capsida may be eligible to receive up to $595 million in option fees and research and development milestones, with potential for further commercial milestones.

Capsida is also eligible to receive mid-to-high single-digit royalty payments on future product sales. Capsida will lead capsid discovery efforts for all programs using its high throughput AAV engineering platform and will be responsible for process development and early clinical manufacturing. AbbVie will lead innovative therapeutic cargo approaches and be responsible for development and commercialization.

The company’s AAV engineering platform is designed to generate capsids optimized to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease, allowing for improved efficacy and safety. The platform uses machine learning, structural biology, non-human primate models, and human tissue models to screen billions of engineered capsids for the ability to target desired tissue types precisely.

Capsida says its engineered capsids have demonstrated markedly enhanced tissue tropism for neurons versus astrocytes, glia, and other CNS cell types, thus demonstrating potential to unlock treatments for disorders requiring neuronal transduction that exceeds the performance of first-generation AAV9-based therapies.

Photo: Peter Anastasiou, CEO of Capsida

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