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.
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.
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.
1 target hypothesis,
Wide net approach
one target at a time,
Speed via multiplexing
- Quantify drug response across entire genome
- Screen entire franchises of root diseases in a single shot
Faster (1-4 years)
More efficient path to disease modifying therapies
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.
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.
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.