Our Roadmap

Monogenic Diseases

Caused by a single gene alteration, monogenic diseases represent an area of high unmet need, and also of great therapeutic opportunity.
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Trafficking Defects

Many monogenic diseases result from a mutation that causes improper trafficking, the failure of the mutated protein to get to the right place inside the cell.
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Patient-Driven Discovery

Stem-Cell technology allows patients to participate in drug discovery programs at their earliest stages.
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