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Methylmalonic Acidemia (MMA)

2
Pipeline Programs
4
Companies
2
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 2 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
1
SEL-302Phase 1/21 trial
Active Trials
NCT05778877Withdrawn0Est. Aug 2029
Genome & Company
Genome & CompanyKorea - Suwon
1 program
1
SEL-302Phase 1/2
Moderna
ModernaCAMBRIDGE, MA
1 program
mRNA-3704PHASE_1_2RNA Therapeutic1 trial
Active Trials
NCT03810690Withdrawn0Est. Aug 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Human BioSciencesSEL-302
ModernamRNA-3704

Clinical Trials (2)

A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

Start: Dec 2022Est. completion: Aug 20290
Phase 1/2Withdrawn

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

Start: May 2019Est. completion: Aug 20200
Phase 1/2Withdrawn

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

4 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.