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Patient Avatars

Phase I of III
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Representative Animals

Patient Avatars are animals that are most closely representative of human patients with age-related diseases, and have often acquired the disease naturally.

Why doesn’t everyone use representative animals?

Because traditional drug testing requires dozens of animals to test a single therapeutic, drug developers typically use small, young animals (usually mice) that are plentiful and cost-effective to house over time. Disease is usually artificially induced in an attempt to mimic the disease phenotype in humans.

Because Mosaic Screening tests thousands of therapies in a single sick animal, Gordian can use animal models with physiology and biology significantly more similar to the human patients we eventually want to treat, delivering answers about what’s likely to be effective clinical trials.

Animals have natural biological variations, which are typically addressed by using statistical methods that average over these variations. However, human patients are also diverse. By conducting screens within individual animals, Gordian assesses therapeutic effects on an animal-by-animal basis, allowing their relevance to specific patient populations to be predicted.

This results in the discovery of powerful information about treatments that reveal what’s most likely to be effective in clinical trials.

While the Gordian platform allows the use of more advanced animal models, these models still aren’t human. We mitigate this in two ways: Identifying the most human-like animal models for the disease of interest and finding the subset of humans most likely to respond to the therapies discovered in that animal model. By leveraging the same gene therapy, single-cell sequencing, and machine-learning technologies used for our screens, we explore the similarity of animal models to the human system. We also examine the effects of screen hits in human systems ex vivo. Together, these provide a direct human link to all therapeutics we advance into development.

Silvia Domcke Head of Human Genomics
A portrait of Silvia Domcke.