Our mission is to develop life-changing therapies for as many rare diseases as possible for the millions of patients in urgent need.
Medical science has identified approximately 7,000 rare diseases. Individually, these conditions may impact few patients. Together, however, they affect approximately 30 million patients in the US alone, and 300 million worldwide.1-3 Although these diseases affect so many, only about 650 FDA-approved treatments have been developed to target them.4,5
Compared with the vast number of patients suffering from the most common diseases, rare conditions form what we call “the long tail of disease.” Despite how common rare diseases collectively are, the majority go unnoticed, without cure or adequate treatments.5
We see past boundaries to address the “long tail of disease.”
Our portfolio of companies is developing first-in-class antibody therapeutics built on novel biology.
We are a constellation of creative, innovative thinkers and collaborators, commited to exploring new possibilities.
References: 1. Rare Disease FAQ. National Human Genome Research Institute. Genome.gov. Published January 10, 2020. Accessed December 23, 2021. https://www.genome.gov/FAQ/Rare-Diseases 2. US FDA. Rare Diseases at FDA. Published February 20, 2020. Accessed February 4, 2021. https://www.fda.gov/patients/rare-diseases-fda 3. Nguengang Wakap S, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28(2):165-173. doi:10.1038 s41431-019-0508-0 4. Orphan Drug Designations and Approvals. Accessdata.fda.gov. Published 2021. Accessed December 14, 2021. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm 5. Huron J. New study investigates the number of available orphan products, generics and biosimilars. NORD (National Organization for Rare Disorders). Published March 25, 2021. Accessed December 23, 2021. https://rarediseases.org/new-study-investigates-the-number-of-available-orphan-products-generics-and-biosimilars/
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