AbbVie and Capsida Biotherapeutics partner on eye disease gene therapies

By Jane Byrne

- Last updated on GMT

© GettyImages/Zorica Nastasic
© GettyImages/Zorica Nastasic

Related tags Gene therapy AAV AbbVie Capsida Biotherapeutics Eli lilly

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

The deal builds upon the neurodegenerative disease partnership that the two entitites announced in 2021. This extension sees Capsida moving into the ophthalmology space.

AbbVie outlined how its capabilities will be paired with Capsida's adeno-associated virus (AAV) engineering platform and manufacturing capabilities to identify and advance three programs. The companies did not go into the specifics of those gene therapy targets.

Under the terms of the agreement, Capsida will receive $70m, consisting of upfront payments and a potential equity investment. In addition, the developer may be eligible to receive up to $595m in option fees and R&D milestones. It is also entitled to receive mid-to-high single-digit royalty payments on future product sales.

The gene therapy startup is to lead capsid discovery efforts for all programs using its high throughput AAV engineering platform and it will be responsible for process development and early clinical manufacturing. AbbVie will lead innovative therapeutic cargo approaches and be responsible for development and commercialization. 

"We are excited to expand our collaboration into ophthalmology with the world leader in this therapeutic area," said Peter Anastasiou, CEO of Capsida. "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,” he added.

Additional partnerships

The deal follows the announcement last month by Capsida that it had entered into a multi-year pact with Eli Lilly subsidiary, Prevail Therapeutics, to develop transformative genetic medicines for serious diseases. As part of the tie-up, Prevail will leverage Capsida’s AAV engineering platform to identify and advance clinically translatable capsids paired with Prevail’s cargo to develop IV-administered gene therapies directed to specified targets known to cause serious diseases that affect the central nervous system (CNS).

Delivering AAV gene therapy systemically to target the CNS, while limiting exposure to non-target organs such as the liver has been a significant challenge in the gene therapy field. Looking to tackle such challenges, Capsida says it has developed a high throughput platform to biologically screen and identify engineered AAV capsids that target specific tissues, such as the brain, and limit transduction of tissues and cell types that are not relevant to a given disease. “This platform has the potential to improve the efficacy and safety of systemically administered AAV gene therapies and deliver on the promise of gene therapy for patients that are living with diseases that are difficult to treat with existing approaches.”

Capsida also established a neurological disorder focused alliance with CRISPR Therapeutics, back in June 2021. That partnership is focused on the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.

Under their agreement, CRISPR Therapeutics will lead R&D of the Friedreich’s ataxia program and perform gene-editing activities for both programs, and Capsida will lead R&D of the ALS program while conducting capsid engineering for both projects.

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