Breakthrough Science
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
Our new Chief R&D Officer shares what drew him to medicine, how he found a home in biopharma and what about 澳门正规博彩十大排行平台’s science excites him the most.
Rebeca Costa is using her voice to help make Rio de Janeiro’s world-famous event more accessible.
This September for Newborn Screening Awareness Month, we spoke with Denise Queally, President of New England Connection for PKU and Allied Disorders, to learn more about why early diagnosis has been so meaningful for her family.
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
We listen to and learn from patient communities to collaborate on solutions with the potential to help improve care.
Our talented and dedicated people represent the best the biotech industry has to offer and have helped shape a culture focused on working together to deliver for the patients we serve.
Since our founding in 1997, we’ve applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Our approved medicines treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases. We’re also advancing an industry-leading pipeline of investigational medicines that are in various stages of clinical and preclinical development.
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We're proud to announce that our Chief Commercial Officer Cristin Hubbard has been appointed to the Biotechnology Innovation Organization (BIO) Board of Directors. BIO is the world’s largest biotech advocacy association focused on advancing the industry, fostering innovation and expanding access to medicines. In her new role, Cristin will be a member of the organization’s Health Section Governing Board. Congrats, Cristin!
Meet F8: a gene that plays a key role in blood clotting by telling cells how to make a protein called factor VIII. Changes in a person’s F8 gene can cause hemophilia A, a genetic condition that can lead to health complications, including uncontrolled bleeding and irreversible joint damage. Our scientists have led research into F8 with the goal of targeting the underlying cause of hemophilia A.
Come work with us. Search our global career opportunities to learn how you can make your mark.