We're enabling a better life for everyone living with a rare disease.
400 million people worldwide live with a rare disease.

.webp)
Rarebase is tackling this overwhelming need using proven science and innovative high-throughput technology to achieve drug discovery at unprecedented speed and scale. Through our FUNCTIONTM platform, we are creating a portfolio of potential therapeutics for hundreds of diseases at once, at a fraction of the cost and time of traditional methods.















.webp)


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.
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
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.
The latest from Rarebase
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

Elizabeth Iorns, PhD
Scientific Founder & Advisor

Nicole Perfito, PhD
VP of Research Operations

Natalie Downs, MS, CGC
Senior Project Manager

Sabine Topka, PhD
Director of Discovery Biology

Carrie Rich
Chief Partnerships Officer

Riley Parsons
Senior Software Engineer

Clayton Mellina
Director of Software Engineering

Arun Mahadevan, PhD
Senior Scientist I, Phenotypic Screening

Joe Bellucci, PhD
Director of High Throughput Biology

Alex Guin, PhD
Bioinformatics Engineer

Ursula Leone Haditsch, PhD
Director of iPSC Cell Modeling

Amanda Faubel
Project Manager

Gabriel Rivera Del Toro
Research Associate I, Discovery Biology

Kristine Hashimoto
Partnerships Associate

Deanna Wong
Senior Operations Manager

Christian Markopoulos
Senior Scientist II, Phenotypic Screening

Long McFarlin
Senior Scientist I, iPSC Cell Modeling

Eric Yeager
Associate Director, Facilities

Jackie Van Siclen
Research Associate II, iPSC Cell Modeling

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.
Oops! Something went wrong while submitting the form.
Photography by: Ceridwen Hughes