Roundtable Sponsor
Name
Amgen
Description
Amgen is a global biopharmaceutical company focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Our portfolio, along with our pipeline, is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We are relentless in pursuing breakthroughs that boldly change the lives of the communities we serve. Discover more about our medicines in rheumatology, including the first and only biologic treatment for uncontrolled gout, and research for new therapeutic targets in gout and systemic sclerosis at www.amgen.com.
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Sponsors
Name
Alexion
Description
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
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Name
Crystalys
Description
Crystalys Therapeutics is a clinical-stage biopharmaceutical company transforming the treatment of gout. Headquartered in San Diego, California, and co-founded by Catalys Pacific and Novo Holdings, Crystalys brings together a world-class team with deep expertise in gout drug development, dedicated to delivering more effective options for people living with gout. The company’s lead candidate, dotinurad, is a next-generation, once daily oral, URAT1 inhibitor in clinical development as a second-line therapy aimed to reduce uric acid, gout flares and tophi. Dotinurad was invented by Fuji Yakuhin and has obtained regulatory approval in Japan, China, Philippines, Taiwan and Thailand. With best-in-class potential for both safety and efficacy, dotinurad is supported by clinical data from multiple Asian markets where it is approved with over 2.2 million having received dotinurad in Japan alone in the post-marketing setting . Crystalys is advancing dotinurad in 3 Phase 3 trials (RUBY, TOPAZ and OPAL) and 2 Phase 2 trials (AMATHEYST and TOURMALINE) toward regulatory approval and commercial launch. For more information, visit www.crystalystx.com/ and follow us on X and LinkedIn.
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Name
Sobi - Medical Affairs
Description
At Sobi, we are transforming the lives of people affected by rare diseases. As a specialized biopharmaceutical company, we provide sustainable medicines to innovative therapies in the areas of hematology and immunology. We bring something rare to rare diseases – a rare expertise and a strength in access that allows us to be a partner in care for those with high unmet needs. Sobi headquarters are located in Stockholm, Sweden.
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