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
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
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
4 companies competing in this space
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