ARCT - Arcturus Therapeutics initiates dosing of mRNA-based candidate targeting rare metabolic disorder

Arcturus Therapeutics (NASDAQ:ARCT) has initiated the dosing in its Phase 1b study of ARCT-810, its leading rare disease asset targeting Ornithine Transcarbamylase deficiency, a urea cycle disorder with no FDA-approved therapy.OTC deficiency affects approximately 10,000 people worldwide, with the lack of the OTC enzyme in liver cells causing seizures, coma, and death in untreated patients.ARCT-810, utilizing Arcturus' LUNAR® lipid-mediated delivery platform, deliver OTC messenger RNA to liver cells.“The initiation of dosing involving approximately twelve patients with OTC deficiency builds upon the favorable prior Phase 1 study results, as well as encouraging preclinical data,” said Steve Hughes, M.D., Chief Development Officer of Arcturus. “We look forward to obtaining clinical data throughout 2021," he further added.

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Arcturus Therapeutics initiates dosing of mRNA-based candidate targeting rare metabolic disorder