Accelerating drug discovery for one or for many.

rare disease patient. Hurlers Syndrome. genetic condition
Rarebase is a public benefit biotech led by scientists, engineers, patients and advocates. We leverage innovative technologies to build platforms that help us discover and develop treatments for the millions of people worldwide affected by the long tail of rare genetic disorders.
Meet Function

Drug discovery for thousands of genetic disorders in one unified platform.

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. Our hope is that Function provides many therapeutic candidates that can be rapidly developed for patients living with neurological diseases.

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Function discovers drugs that address underlying disease biology, at scale.

We match genetic disorders with thousands of therapeutic candidates using our functional genomics screening platform.
Disease models
We create cell-based disease models directly from patients’ samples.
We test therapeutic candidates in patient cell models for therapeutic effects, such as making a functional gene copy compensate for a dysfunctional one.
We use validation data from the disease models to improve Function’s predictions.

“Rarebase is pushing the limits of science and innovating a financial structure that allows patient organizations to directly support drug discovery.”

- Mike Graglia, Founder and Managing Director, SynGAP Research Fund

“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.”

- Sandra Sermone, Founder and President, ADNP Kids Research Foundation

We're in this together.

Rarebase collaborates with a growing number of rare disease focused patient organizations to apply the latest in drug discovery to genetic neurological diseases.
by recommendation
All of our patient organization relationships come from recommendations from current partners or connections from within the rare disease community.
Over 110 patients and their families affected by rare disease are actively participating in our research studies.
novel candidate in development
Rarebase is already developing its first novel neurodegeneration candidate in partnership with a patient organization. This target is conserved across multiple neurodegenerative diseases.

For us, it’s personal.

We're a passionate team focused on pushing the boundaries of translational medicine to help develop new therapies for medically underserved patient communities.
Onno Faber
Co-founder and CEO
Omid Karkouti
Co-founder and COO
Chris Moxham
Chief Scientific Officer
Hayley Brooks
Chief of Staff
Elizabeth Iorns, PhD
Scientific Founder & Advisor
Nicole Perfito, PhD
Director of Research Operations
Lynsey Chediak
Head of Partnerships
Meghan Edgar
Director of Preclinical Development
Amina Ann Qutub, PhD
Scientific Advisor (Computational Biology)
Mo Rahman
Scientific Advisor (Bioinformatics)
Michael Genin, PhD
Scientific Advisor (Drug Development)
Daniel McCoy, PhD
Scientific Advisor (Gene Therapy)
Meg Wood
Strategic Advisor (Partnerships)
Donald Apanovitch, PhD
Scientific Advisor (Functional Genomics)
Matt Deardorff, MD PhD
Scientific Advisor (Clinical Genetics)
Matt Might, PhD
Scientific Advisor
Matt De Silva

We’d love to hear from you. Let’s talk.

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Photography by: Ceridwen Hughes