Study 331-201 Recruiting
Study 331-201: A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Subjects with Hereditary Angioedema (HAE) due to Human C1 Esterase Inhibitor (C1-INH) Deficiency
Indication
Hereditary Angioedema (HAE)
Goal
This open label, two-part study aims to establish the safety, tolerability, and efficacy of BMN 331
Investigational therapeutic
BMN 331
Status
This study is currently recruiting participants
Study Type
Phase 1/2