We're enabling a better life for everyone living with a rare disease.
Drug discovery for thousands of genetic disorders in one unified platform.
The Function platform combines two unique data sets: large scale drug screens and genetic screens performed in neurons. The drug screen tests the effects of thousands of small molecules on gene expression. The genetic screen measures changes in expression when disease causing genes are suppressed.
The combined data sets will be used to match potentially effective drugs to rare disorder genes. Our hope is that Function provides many therapeutic candidates that can be rapidly developed for patients living with neurological diseases.
The Function platform discovers drugs that address underlying disease biology, at scale.
“Rarebase is pushing the limits of science and innovating a financial structure that allows patient organizations to directly support drug discovery.”
“Rarebase is stepping outside the traditional drug development pipeline and harnessing new technology and science that patient organizations like ours would never be able to utilize alone.”