Products and Pipeline

For more than two decades, we have leveraged expertise in genetics and genomics to create new medicines for people with serious medical conditions. Learn how we’re now applying our proven development capabilities to bring forward a pipeline of medicines aimed at helping larger groups of people with unmet medical needs.

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Our eight approved therapies have been transformational treatment options for patients.

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Our robust pipeline includes a number of clinical-stage and preclinical programs.

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Expanded Access

We’re committed to compliantly providing safe and effective therapies to as many people who may benefit from them as possible.

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