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Ultragenyx Pharmaceutical Announces UX701 AAV9 Gene Therapy To Treat Wilson Disease Failed To Meet Criteria, Being Terminated

Benzinga·11/03/2026 21:46:02
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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced it is terminating UX701 for Wilson disease based on not achieving study goals.