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What we do
We are accelerating access to treatments for rare disease patients by developing promising new drug therapies.
Our pipeline
Our drug therapy pipeline
We breathe new life into assets that have not been fully unlocked. We acquire drug candidates that demonstrate, based on existing human safety and efficacy data, the potential for approval in rare disease indications where there's no current treatment.
We are building a broad, late-stage portfolio that is designed to deliver regulatory approvals in a shorter time and with higher probability than more traditional early-stage biotechnology business models.
Explore our acquisition-based rare disease pipeline
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