A path forward,
for one or for many.

rare disease patient. Hurlers Syndrome. genetic condition
Rarebase is a public benefit precision medicine company that leverages cutting-edge technology and biology to discover and develop treatments for the millions of people worldwide living with a rare disease.
Meet the Function™️ platform

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.

Learn More

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

1
Prediction
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.
2
3
Validation
We test therapeutic candidates in patient cell models for therapeutic effects, such as making a functional gene copy compensate for a dysfunctional one.
Learning
We use validation data from the disease models to improve the Function platform’s predictions.
4

“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.

25+
partnerships
Rarebase collaborates with a growing number of rare disease focused patient organizations to apply the latest in drug discovery to genetic neurological diseases.
100%
by recommendation
All of our patient organization relationships come from recommendations from current partners or connections from within the rare disease community.
110+
participants
Over 110 patients and their families affected by rare disease are actively participating in our research studies.
1
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, PhD
Chief Scientific Officer
Hayley Brooks
Chief of Staff
Elizabeth Iorns, PhD
Scientific Founder & Advisor
Nicole Perfito, PhD
Director of Program Management
Lynsey Chediak
Head of Partnerships
Natalie Downs, MS, CGC
Research Project Manager
Amina Ann Qutub, PhD
VP of Computational Biology
Sabine Topka, PhD
Director of Discovery Research
Carrie Rich
Partnerships Advisor
Riley Parsons
Senior Software Engineer
Clayton Mellina
Director of Software Engineering
Arun Mahadevan, PhD
Quantitative Neuroscientist
Joe Bellucci, PhD
Director, Scientific Partnerships
Kayla Capper, PhD
Director of Product Science
Brittany Anderton, PhD
Scientific Communications Lead
Devin Cavero
Community Engagement Coordinator
Mo Rahman
Scientific Advisor (Bioinformatics)
Michael Genin, PhD
Scientific Advisor (Drug Development)
Meg Wood
Strategic Advisor (Partnerships)
Matt Deardorff, MD PhD
Scientific Advisor (Clinical Genetics)
Matt Might, PhD
Scientific Advisor
Matt De Silva
Advisor
Carolina Garcia Rizo, PhD
Advisor

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

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Photography by: Ceridwen Hughes
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