Drugs / MMA-101
MMA-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05778877 | Dec 2022 → Aug 2025 | methylmalonic acidemia | Selecta Biosciences, Inc. | Withdrawn | No outcome recorded |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2020-11-19 | AskBio Inc | Regulatory AskBio and Selecta Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia askbio.com ↗
MMA-101 has obtained both orphan drug designation and rare pediatric disease designation from the FDA |
| 2020-10-20 | AskBio Inc | Regulatory Selecta and AskBio Receive FDA Rare Pediatric Disease Designation for Methylmalonic Acidemia Gene Therapy askbio.com ↗
Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. |
All press releases naming this drug 3 releases
| Date | Issuer | Release |
|---|---|---|
| 2021-04-29 | AskBio Inc | Selecta Biosciences and AskBio Announce Updates to Methylmalonic Acidemia (MMA-101) Program askbio.com ↗ |
| 2020-11-19 | AskBio Inc | AskBio and Selecta Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia askbio.com ↗ |
| 2020-10-20 | AskBio Inc | Selecta and AskBio Receive FDA Rare Pediatric Disease Designation for Methylmalonic Acidemia Gene Therapy askbio.com ↗ |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited 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-101” NCT05778877 ↗ |
| 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 ↗ |