Project CargoTek

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Adeno-associated virus (AAV)

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

 

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

 

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

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