Pharmaceutical

Dimension Therapeutics announced the start of patient dosing for their Phase 1/2 clinical trial to evaluate DTX301 for the treatment of patients with Ornithine Transcarbamylase deficiency.

Dimension Therapeutics (NASDAQ:DMTX) announced the start of patient dosing for their Phase 1/2 clinical trial to evaluate DTX301 for the treatment of patients with Ornithine Transcarbamylase (OTC) deficiency.
As quoted in the press release:

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 ammonia can lead to significant medical issues for patients who are in need of better disease-modifying therapies.
“OTC deficiency is an inherited metabolic disease (IMD) for which approved therapies are unable to eliminate the risk of metabolic crises from elevated ammonia. We believe that DTX301, based on our differentiated AAV8 platform in IMD, holds great promise for addressing the unmet need among patients, and we look forward to the continued advancement of the trial,” said Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. Based on the current progress, Dimension anticipates reporting initial clinical data from the Phase 1/2 DTX301 clinical trial by late this year.

Click here to read the full press release.

Source: globenewswire.com

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