We're enabling a better life for everyone living with a rare disease.

400 million people worldwide live with a rare disease.

95% of people with a rare disease don't have effective treatment.
We're on a mission to change that.

Through our FUNCTIONTM product engine, 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.

Our Revolutionary

Rarebase is an innovative public benefit ​​biotech company that is revolutionizing drug discovery at unprecedented speed and scale.

​Rarebase combines proven science and innovative high-throughput technology to discover disease-modifying therapies for hundreds of diseases at a time. We are building the technology and partnerships that can dramatically accelerate the way the world does drug discovery.

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Traditional drug discovery is not enough.

We’re using proven science together with innovation to challenge ​​the status quo. While traditional methods can take 10+ years and >$20 million to generate one clinical candidate, Rarebase can screen thousands of drugs across thousands of root-cause genes 10-100x more cost-effectively and in a fraction of the time.​​​​​​

Drug Discovery
Target &
Target & Scope
1 disease,
1 target hypothesis,
10,000+ compounds

Wide net approach

100s of diseases, 1,000s of genes, 1,000s of compounds
Target agnostic cell-based screening - able to cast a wide net to identify best targets that modulate root cause gene expression
Single-tracked -
one target at a time,
many years

Speed via multiplexing

Our technology can:
  • Quantify drug response across entire genome
  • Screen entire franchises of root diseases in a single shot
Time to
Time to Clinic
Slower (8-10 years)
Nascent chemical starting point

Faster (1-4 years)

Mature starting point of high quality, well annotated drugs
Complex disease

More efficient path to disease modifying therapies

Focus on monogenic disorders with validated known root cause genes


Our Revolutionary Product Engine
High Throughput
Lab Screen
Clinical Candidate

Many genetic rare diseases could be treated directly at the root cause if the right drug is identified to modulate gene expression. Our FUNCTION product engine can screen thousands of small molecule compounds and map their effect on gene expression at near genome scale. FUNCTION also integrates intelligence applications that allow Rarebase scientists to pull data insights with greater efficiency. It is a synergistic partner to the scalable experiments performed in our laboratory and identifies many possible indications for high-potential drug candidates. This approach quickly aligns a lead compound with diseases that may benefit most from its mechanism of action, driving research forward across an entire landscape of rare diseases.

Our Progress

25+ Partnerships

Rarebase collaborates with a growing number of rare disease focused patient organizations to apply the latest in drug discovery to genetic rare diseases. Collaborating with patients is a critical piece to accelerating drug discovery for the entire community.

12 Months

In neurons, Rarebase screened 4,000 drugs across 20,000 genes in just 12 months. With unrivaled speed and scale, our proprietary process has been able to assess thousands of drugs across thousands of root-cause genes in a fraction of the time of traditional drug discovery methods.

Creating Value For Partners - Exponentially

For patient organizations and biotech companies, we accelerate the drug discovery process. We can create targeted drug portfolios with high capital efficiency. Each cell-type drug screen has the potential to open hundreds of new opportunities – but we need partners at all levels to pursue them all. We believe in exploring every viable partnership path on our mission to address the needs of the rare disease community.

Rarebase leadership

Bringing together world-class science, engineering, and communities to deliver immense value and positive impact.
Chris Moxham, PhD
CEO and Chief Scientific Officer
Nicole Perfito, PhD
VP of Program Management
Clayton Mellina
VP of Software Engineering
Deanna Wong
VP of People & Operations

Partner with us,
let’s talk.