Organic Acidemia Clinical Trials

Clinical Trials

Propionic Acidemia Clinical Trials

Moderna - mRNA-3927

mRNA Therapy

IND enabling
Phase 1
Phase 2
Phase 3
FDA review

mRNA-3927 is an mRNA therapy designed to treat Propionic Acidemia in patients with mutations in PCCA or PCCB. Detailed information about the clinical trial including eligiblity criteria, sites, and endpoints can be found on Moderna’s website or at clinicaltrials.gov

mRNA-3927 Program Update – February 2026

  1. Koeberl D, Schulze A, Sondheimer N, et al. Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature. Apr 2024;628(8009):872–877. doi:10.1038/s41586-024-07266-7
  2. Baek R, Coughlan K, Jiang L, et al. Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria. Nat Commun. May 7 2024;15(1):3804. doi:10.1038/s41467-024-47460-9

Mayo Clinic - AAVrh10-PCCA

Gene Therapy

IND enabling
Phase 1
Phase 2
Phase 3
FDA review

AAVrh10-PCCA is an gene therapy designed to treat Propionic Acidemia in patients with mutations in PCCA. Detailed information about the clinical trial including eligiblity criteria, sites, and endpoints can be found  at clinicaltrials.gov

1. Hofherr SE, Senac JS, Chen CY, Palmer DJ, Ng P, Barry MA. Short-term rescue of neonatal lethality in a mouse model of propionic acidemia by gene therapy. Hum Gene Ther. Feb 2009;20(2):169–80. doi:10.1089/hum.2008.158

2. Guenzel AJ, Hofherr SE, Hillestad M, et al. Generation of a hypomorphic model of propionic acidemia amenable to gene therapy testing. Mol Ther. Jul 2013;21(7):1316–23. doi:10.1038/mt.2013.68

3. Guenzel AJ, Hillestad ML, Matern D, Barry MA. Effects of adeno-associated virus serotype and tissue-specific expression on circulating biomarkers of propionic acidemia. Hum Gene Ther. Sep 2014;25(9):837–43. doi:10.1089/hum.2014.012

Methylmalonic Acidemia Clinical Trials

Moderna - mRNA-3705

mRNA Therapy

IND enabling
Phase 1
Phase 2
Phase 3
FDA review

mRNA-3705 is an mRNA therapy designed to treat methylmalonic acidemia (MMA) caused by MUT deficiency. Detailed information about the clinical trial including eligiblity criteria, sites, and endpoints can be found on Moderna’s website or at clinicaltrials.gov

  1. Baek R, Coughlan K, Jiang L, et al. Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria. Nat Commun. May 7 2024;15(1):3804. doi:10.1038/s41467-024-47460-9

NHGRI - MMA101

Gene Therapy

IND enabling
Phase 1
Phase 2
Phase 3
FDA review

MMA-101 is an gene therapy designed to treat methylmalonic acidemia (MMA) caused by MUT deficiency. A phase 1/2 clinical trial will be sponsored by the National Human Genome Research Institute (NHGRI), one of the National Institutes of Health (NIH). 

May 2026 – FDA clears IND for MMA101

1. Chandler RJ, Di Pasquale G, Sloan JL, et al. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9. Mol Ther Methods Clin Dev. 2022;27:61-72. Published 2022 Sep 6. doi:10.1016/j.omtm.2022.09.001

Genespire - GENE202

Gene Therapy

IND enabling
Phase 1
Phase 2
Phase 3
FDA review

GENE202 is an gene therapy designed to treat methylmalonic acidemia (MMA) caused by MUT deficiency. 

January 2026 – GENE202 granted orphan drug designation by FDA and EC

1. Barbon E, Simoni C, Argabright A, et al. Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice. J Hepatol. Published online May 25, 2026. doi:10.1016/j.jhep.2026.05.011

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