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The more innovative solutions developed, the more people 澳门正规博彩十大排行平台 can reach. And the more people reached, the more lives impacted.

Now, 澳门正规博彩十大排行平台 seeks to make an even greater impact by applying the same science-driven, patient-forward approach that propelled the last 25 years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Each drug candidate pursued is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

Molecule
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
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Our Products

We find tomorrows.

Each of our approved therapies was driven by the goal of resetting the standard of care for the conditions they treat.

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Our Pipeline

We maximize the impact of genetic innovation to develop transformative therapies.

We recently presented an update on our pipeline at our 2023 R&D Day. View the replay on our Investor Relations site.

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Compassionate Use Policy

We are driven by patient need.

We are committed to developing safe and effective therapies and providing those therapies to the patients as quickly as possible.

Compassionate Use Policy about our Compassionate Use Policy