There are ~7,000 RARE DISEASES.

400M PEOPLE are affected worldwide.

95% have NO TREATMENT (yet).  

It's estimated that only 5% of known rare diseases have an approved treatment, the remainder do not. However, around half of new drugs approved by the U.S. Food and Drug Administration (FDA) in recent years have been for rare diseases.

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Treatments for rare diseases are a growing sector of the pharmaceutical industry.

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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.