Ultragenyx (NASDAQ:RARE) announces positive results from cohort 3 and longer-term data from cohort 2 in an ongoing Phase 1/2 clinical trial evaluating gene therapy DTX301 in patients with ornithine transcarbamylase deficiency.
In cohort 3 (n=3), there were two confirmed female responders and one potential male responder (longer follow-up needed to confirm).
In cohort 2, one female patient showed a response starting at week 52 that was confirmed at week 78. Two previously reported responders in cohorts 1&2 remain clinically and metabolically stable at weeks 104 and 78, respectively.
Management will host a conference call at 4:30 pm ET to discuss the results.
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