RARE Daily

Rarebase Launches a Neuroscience Drug Discovery Platform Collaborating with 15 Rare Disease Patient Groups

September 15, 2021

Rarebase, a public benefit biotechnology company, launched its tech-enabled drug discovery platform called Function.

Function 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. The hope is that Function will provide many therapeutic candidates that can be rapidly developed for patients living with neurological diseases.

“We see the potential of applying our technology at scale to the discovery of drugs and targets for thousands of diseases that currently have no treatments,” said Onno Faber, co-founder and CEO of Rarebase.

Function’s 15 patient organization collaborators represent genes that cause epilepsy, autism, developmental delay and neurodegeneration. Function platform collaborators include: ADNP Kids Research Foundation, SynGAP Research Fund, FOXG1 Research Foundation, STXBP1 Research Foundation, Hereditary Neuropathy Foundation, Hope4Harper, Lightning and Love Foundation, Cure ATRX, Charlotte and Gwenyth Gray Foundation, CureMito Foundation, the SHANK2 Foundation, and multiple funds associated with the Rare Village and Rare Crossroads networks.

“There are many common elements shared between neurological diseases. Instead of each of us working in silos, partnering on specific projects is critical,” said Nasha Fitter, the co-founder and CEO of the FOXG1 Research Foundation. “With Rarebase, we can screen more drugs, we can share costs, and we have more opportunity to learn from one another.”

Photo: Onno Faber, co-founder and CEO of Rarebase.

Author: Rare Daily Staff

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