Dimension Therapeutics Commences Patient Dosing in Global, Multi-Center Phase 1/2 Clinical Trial of DTX301 in Ornithine Transcarbamylase (OTC) Deficiency
DIMENSION THERAPEUTICS (DMTX)
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Source: GlobeNewswire
Twelve trial sites recruiting patients with OTC deficiency in the United States, United Kingdom, Spain, and Canada Initial data from the company’s Phase 1/2 study anticipated by late 2017 CAMBRIDGE, Mass., Aug. 31, 2017 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today announced the initiation of patient dosing in the company’s global, multi-center Phase 1/2 clinical trial to evaluate DTX301, the only AAV gene therapy in clinical testing for the treatment of patients with Ornithine Transcarbamylase (OTC) Deficiency. DTX301 is designed to deliver stable expression and activity of OTC following a single intravenous infusion and has been shown in academic preclinical studies in relevant mouse models to normalize levels of urinary orotic acid, a marker of ammonia metabolism. In the late onset form of the disease, elevated a
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