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Propionic Acidemia

2
Pipeline Programs
3
Companies
3
Clinical Trials
2 recruiting
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
2
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 2 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
1 program
1
N-carbamylglutamatePhase 2
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
1
N-carbamylglutamatePhase 21 trial
Active Trials
NCT01597440Terminated1Est. Feb 2015
Moderna
ModernaCAMBRIDGE, MA
2 programs
mRNA-3927PHASE_1_2RNA Therapeutic1 trial
mRNA-3927PHASE_1_2RNA Therapeutic1 trial
Active Trials
NCT04159103Recruiting77Est. Aug 2027
NCT05130437Recruiting50Est. Dec 2031

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Angeles TherapeuticsN-carbamylglutamate
ModernamRNA-3927
ModernamRNA-3927

Clinical Trials (3)

Total enrollment: 128 patients across 3 trials

NCT01597440Angeles TherapeuticsN-carbamylglutamate

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

Start: Sep 2012Est. completion: Feb 20151 patients
Phase 2Terminated
NCT05130437ModernamRNA-3927

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Start: Nov 2021Est. completion: Dec 203150 patients
Phase 1/2Recruiting
NCT04159103ModernamRNA-3927

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

Start: Apr 2021Est. completion: Aug 202777 patients
Phase 1/2Recruiting

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

2 actively recruiting trials targeting 128 patients
RNA Therapeutic is the dominant modality (100% of programs)
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.