Pathway Convergence Enables Matching
Prediction
Rare diseases sharing >50% pathway overlap with common diseases will respond to drugs approved for those common diseases.
Rare Disease
7,000+ rare diseases. 300 million patients. 95% have no approved treatment. But pathway convergence means drugs approved for common diseases may work for rare ones. These six hypotheses address systematic drug repurposing.
6
Hypotheses
7,000+
Diseases
95%
Without Treatment
Hypotheses
Autoinflammatory Disease as Model
Prediction
For any autoinflammatory disease with elevated IL-1/IL-6/TNF, the corresponding approved inhibitor will show clinical benefit regardless of the specific genetic cause.
Interferonopathy Treatment via JAK Inhibition
Prediction
JAK inhibitors will reduce interferon signatures and improve outcomes in all Type I interferonopathies, regardless of upstream genetic cause (STING, TREX1, ADAR, etc.).
mTOR Pathway Diseases
Prediction
mTOR pathway activation status will predict response to mTOR inhibitors across rare diseases, regardless of specific genetic cause.
Complement-Mediated Diseases
Prediction
Complement activation biomarkers will predict response to C5 inhibition across rare diseases beyond current indications.
Regulatory Pathway Innovation
Prediction
Systematic repurposing dossiers will accelerate FDA acceptance of expanded access requests and orphan indications.