Ornithine transcarbamylase (OTC) deficiency is an inherited urea cycle disorder caused by OTC enzyme deficiency, leading to hyperammonemia and potentially severe neurologic toxicity.
Arcturus Therapeutics completed three 28-day inhaled mRNA cohorts for cystic fibrosis that were well-tolerated and showed early biological signals. The company is preparing a 12-week Phase 2 study with tightened eligibility criteria and expanded enrollment into Europe and the Middle East.
Arcturus Therapeutics will release fourth quarter and fiscal year 2025 financial results after market close on March 3, 2026, followed by a conference call at 4:30 p.m. Eastern Time.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT03636438 |
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency |
ACTIVE_NOT_RECRUITING | |
| NCT02991144 |
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency |
COMPLETED | PHASE1/PHASE2 |
