Ornithine Transcarbamylase Deficiency

0
Pipeline Programs
1
Companies
1
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
1100%

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Ultragenyx Pharmaceutical
1 program
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson DiseaseN/AGene Therapy1 trial
Active Trials
NCT04909346Terminated51Est. Nov 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalAdeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Clinical Trials (1)

Total enrollment: 51 patients across 1 trials

NCT04909346Ultragenyx PharmaceuticalAdeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Start: Jun 2021Est. completion: Nov 202251 patients
N/ATerminated

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 companies competing in this space