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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

BMN 331 is an experimental drug that is being tested for the treatment of patients with HAE. This study is the first time BMN 331, which is a gene therapy, is being used in humans. BMN 331 is given only once during this research study and it is given as an infusion. The purpose of this study is to find out what effects, good and/or bad, the Study Drug has on you and your HAE.

You will have a total of 68 in person visits over 68 months where there will be blood drawn and other lab tests. You will have to travel to an off site location to receive the drug once and travel will be paid for by the sponsor of the trial.

Timothy Craig
Hunter Smith - at

18 year(s) or older
This study is NOT accepting healthy volunteers
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Inclusion Criteria:
Male or Female, 18 years of age or older
Have a diagnosis of Hereditary Angioedema type I or type II
Willing to abstain from alcohol consumption for 52 weeks after the infusion
Females of childbearing potential must be willing to use contraception

Exclusion Criteria:
Evidence of active or chronic infection, including Covid-19, or any immunosuppressive disorder
Active cancer, autoimmune, hematologic, cardiac, or renal diseases which require regular treatment
Long term use of attenuated androgens
using immunosuppressants including corticosteroids
Infectious Diseases & Immune System, Allergies
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Hershey, PA ,