Drugs / MMA-101
last change Aug 2025 re-read 2 minutes ago

MMA-101

Gene therapy (AAV / viral vector) targets MMUT

Developed for
methylmalonic acidemia
Investigated by
National Human Genome Research Institute (NHGRI) · Selecta Biosciences, Inc.

Trials 1 · a red edge is where a trial was stopped

2023202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05778877 Dec 2022 → Aug 2025 methylmalonic acidemia Selecta Biosciences, Inc. Withdrawn No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as MMA-101 “a gene transfer therapy that is using an inactive virus, called adeno-associated-virus 8 (AAV8), to deliver the MMUT gene to the liver, by itself called MMA-101NCT05778877
Modality Gene therapy (AAV / viral vector) “a gene transfer therapy that is using an inactive virus, called adeno-associated-virus 8 (AAV8)” NCT05778877
Route Intravenous “IV infusion of MMA-101 in the first patient on Day 1” NCT05778877
Target MMUT “deliver the MMUT gene to the liver” NCT05778877