Drugs / BMN 331
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05121376 | Feb 2022 → Nov 2028 expected | hereditary angioedema | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2022-02-17 | BioMarin Pharmaceutical | Regulatory BioMarin Provides Updates on Progress in Gene Therapy Programs biomarin.com ↗
In addition, the FDA granted Orphan Disease Designation status to BMN 331. |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2022-02-17 | BioMarin Pharmaceutical | BioMarin Provides Updates on Progress in Gene Therapy Programs biomarin.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BMN 331 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05121376 ↗ |
| Known as | AAV5 hSERPING1 | “The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor...” NCT05121376 ↗ |
| Action | Restore | “resulting in restoration of the deficient circulating levels of hC1-INH that cause HAE” NCT05121376 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “an adeno-associated virus (AAV5)-based gene therapy vector” NCT05121376 ↗ |
| Route | Intravenous | “solution for intravenous infusion” NCT05121376 ↗ |