Intentional drug development.
As a nonprofit pharmaceutical research company, we identify the biological signatures of rare genetic diseases and the drugs that reverse them. Because personalized, targeted therapies should be no accident.
​
What will we cure together today?
Molecular Match Making
Leveraging massive compendia of gene expression data, we identify the molecular signature of genetic mutations. We then cross reference that signature with large compendia of gene expression profiles of drug treatments.
Those drugs that optimally reverse the transcriptomic effects of mutations in the gene of interest are selected for down stream validation.
Novel Markers
In addition to drug discovery, our computational pipeline can also identify novel molecular readouts of disease processes.
This information can shed light on the pathophysiology of disease that can inform therapeutic efforts. In addition, these markers can be used to facilitate high throughput drug screening, or as clinical markers of disease.
​
A Proven Approach
Our computational pipeline has been used to identify a novel therapy for a genetic form of heart failure. The selected drug reversed multiple pathological phenotypes of the mutation, restoring key features of cardiomyocyte physiology at both the electrophysiological and mechanical levels.
Reach out to us today to learn more about the validation of our approach and the exciting results it has produced.
​