- Patient dosing is underway in a Phase 1/2 open-label, dose-finding trial assessing Dimension Therapeutics' (DMTX +2.4%) DTX301 in patients with Ornithine Transcarbamylase Deficiency, a genetic urea cycle disorder characterized by the buildup of ammonia in the blood.
- The primary endpoint is safety as measured by the incidence of treatment-emergent adverse events. Secondary endpoints include efficacy measures of the change from baseline in ureagenesis rate and the change from baseline in the 24-hour area-under-the-curve of ammonia. According to ClinicalTrials.gov, the estimated primary completion date is November 2018.
- DTX301 is an AAV gene therapy product designed to deliver ornithine transcarbamylase gene expression in a sustainable way.
- Previously: REGENXBIO to acquire Dimension Therapeutics in all-stock deal, Dimension up 163% premarket (Aug. 25)
- Now read: Regenxbio (RGNX) To Acquire Dimension Therapeutics (DMTX) - Slideshow
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