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Active Org.- |
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Active Indication- |
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Drug Highest PhaseDiscontinued |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of MRT5201 administered intravenously to subjects with OTC Deficiency (OTCD). This study will also evaluate the effect of a single dose of MRT5201 on metabolic markers of OTCD and ureagenesis; and determine an acceptable dosing interval of MRT5201.
100 Clinical Results associated with RNA x OTCase
100 Translational Medicine associated with RNA x OTCase
0 Patents (Medical) associated with RNA x OTCase