Study Drug

The study drug (BMN 331) is an investigational gene therapy designed to introduce the SERPING1 gene into the liver and make C1-INH protein. The study drug is given as a single intravenous (IV) infusion, and is designed to target liver cells.

What is the study drug?

The study drug (BMN 331) is an investigational gene therapy designed to introduce the SERPING1 gene into the liver and make C1-INH protein. The study drug is given as a single intravenous (IV) infusion, and is designed to target liver cells.

Therapy FAQs


What is an investigational drug?

Investigational means that the study drug has not been approved for use by authorities that regulate new medicines, such as the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) and has not been determined to be safe or effective. The study drug can only be used in clinical research studies.


What is the study drug designed to do?

Gene therapy is the method of using genetic material to potentially treat or prevent disease and other conditions. For this investigational gene therapy, a neutralized adeno-associated virus (AAV5) is used as the vehicle that is designed to transfer a functional gene to the liver cells. The AAV vector, or viral shell, is neutralized to not cause illness since most of the viral components of the vector have been removed and replaced with the functional gene.

The visual below explains what the study drug is designed to do and part of what researchers will be looking at in the HAErmony-1 study:

Illustration of a person in the process of IV infusion which allows for the transfer of the SERPING1 gene
Illustration of a person with SERPING1 gene
Illustration of gene entering liver cell which helps make C1-INH protein

With this investigational gene therapy, there is no gene replacement or editing, just introducing the functional gene into the body. The functional gene is not intended to be passed down to future generations.

Therapy FAQs


Have other people used BMN 331?

No, HAErmony-1 is a Phase 1/2 study, where the study drug has not been researched in people. The study drug has been studied in mice and non-human primates (monkeys) where it showed production of C1-INH protein. The method using AAV5 vectors to deliver functional genes has been tested in clinical trials for other genes and diseases.