ARCT - Arcturus gets UK Health Research Authority nod to initiate mid-stage ARCT-810 trial

Arcturus Therapeutics (ARCT) announces that the company has received approval from the UK Health Research Authority to initiate a Phase 2 study for ARCT-810, a novel mRNA-based therapeutic candidate for Ornithine Transcarbamylase ((OTC)) Deficiency.OTC deficiency is a serious urea cycle disorder with a prevalence of about 10K people worldwide.The study, which will enroll up to 24 subjects across two dose levels, is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose design for adolescents and adults with OTC deficiency.Safety and tolerability of ARCT-810 are the primary study objectives, and pharmacokinetic and pharmacodynamic measures of urea cycle function will be performed over a 12- week treatment period to access exposure and efficacy of this novel treatment.

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Arcturus gets UK Health Research Authority nod to initiate mid-stage ARCT-810 trial