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BMN 331 · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| BMN 331 | EXPERIMENTAL | AAV Gene Therapy Infusion |
| Name | Type | Description |
|---|---|---|
| Dose 1 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 2 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 3 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 4 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 5 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 6 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
| Dose 7 of BMN 331 | GENETIC | BMN 331 AAV Gene Therapy |
Inclusion Criteria: 1. Female or male adults ( ≥ 18 years old) 2. Part A only: Confirmed diagnosis of Type I HAE due to C1-INH deficiency confirmed by genotyping of the SERPING1 gene Part B only: Confirmed diagnosis of Type I or II HAE due to C1-INH deficiency confirmed by genotyping of the SERPING...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | Deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
BMN 331 is an investigational gene therapy being developed by BioMarin Pharmaceutical Inc. for hereditary angioedema, a rare immunological condition characterized by recurrent swelling attacks. It is designed to deliver a functional copy of the SERPING1 gene and is currently in Phase 1 clinical testing.
BMN 331 is being studied as a potential treatment for hereditary angioedema, a genetic disorder that causes episodes of severe swelling. The goal is to address the underlying genetic cause by providing a working copy of the SERPING1 gene, which encodes the C1 inhibitor protein.
BMN 331 targets the SERPING1 gene. Hereditary angioedema is often caused by mutations in this gene, which lead to deficient or dysfunctional C1 inhibitor protein. By delivering a functional copy of SERPING1, BMN 331 aims to restore production of the C1 inhibitor protein and help regulate the pathways that trigger swelling attacks.
BMN 331 is being developed by BioMarin Pharmaceutical Inc., a biotechnology company that trades on the Nasdaq under the ticker symbol BMRN. BioMarin specializes in therapies for rare genetic diseases.
BMN 331 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA or any other regulatory agency. The Phase 1 trial is currently active but not recruiting new participants.
BMN 331 is being evaluated in a single Phase 1 trial, NCT05121376, titled A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema. The trial is active but not recruiting, has an estimated enrollment of 44 participants, and is being conducted in the United States, Australia, and Spain.
Yes, Dose 1 of BMN 331 is an alternative name for BMN 331. Both refer to the same investigational gene therapy developed by BioMarin Pharmaceutical Inc. for hereditary angioedema. The dose designation reflects the specific dose level being tested in the Phase 1 trial.