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Also known as Dose 1 of BMN 331
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, Normal Saline Administration |
| 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 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
BMN 331 is an investigational gene therapy being developed for the treatment of hereditary angioedema (HAE), a genetic condition characterized by recurrent episodes of severe swelling. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
BMN 331 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of this gene therapy in patients with hereditary angioedema.
BMN 331 is currently in Phase 1 clinical development. It is an investigational gene therapy for hereditary angioedema and has not yet received regulatory approval. The ongoing Phase 1 study is active but not recruiting participants, with a planned enrollment of 44 subjects.
BMN 331 is being studied in a single Phase 1 clinical trial registered as NCT05121376, titled 'A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema.' The trial is active but not recruiting, enrolling 44 participants across the United States, Australia, and Spain.
Yes, BMN 331 is a gene therapy being developed by BioMarin Pharmaceutical Inc. for the treatment of hereditary angioedema. It is currently in Phase 1 clinical trials, and its mechanism of action involves delivering genetic material to address the underlying cause of the condition.