Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
More uniform response in Cohort 3 with two confirmed responders and one potential responder New female responder in Cohort 2 for a total of three confirmed female responders across all cohorts Up to six responders across all nine patients dosed in study Prophylactic steroid cohort to begin in first half 2020; data expected in second half 2020 Ultragenyx to host conference call today at 4:30 p.m. Eastern Time NOVATO, Calif., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. In Cohort 3 (n=3), there were two confirmed female responders as well a third potential male respo
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