Project CargoTek

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Adeno-associated virus (AAV)

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Project CargoTek aims to reduce immunogenicity associated with biologics, particularly AAV and cell therapies. 

 

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Avoiding cytokine release syndrome and adaptive responses can greatly improve these therapies for many patients.

 

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High-dose steroids and other immune suppressives are not completely effective at preventing innate and adaptive immune responses that hinder gene/cell therapies. 

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We believe there is a solution to reducing adverse events associated with gene/cell therapies, and it will be found outside of traditional immune suppression.

Demonstrated efficacy in human cells and mouse models

  • Demonstrated ability to re-dose certain AAV8 and AAV9 therapies

  • Reduced lymphocyte reactions to AAV upon re-dosing

  • Demonstrated tolerance phenotypes in human cells

Current Challenges

  • High-dose AAV (1e14-1e15 vector genomes) remains highly immunogenic

  • Antibody titers remain high post-second dose AAV.

  • Models that better capture the cellular immune responses observed in humans.

What's next?

  • Assess combinations of new immune modulators

  • Test with new serotypes of AAV

  • Test with new, more potent cell therapies