BMN 331

BMN 331 is an investigational gene therapy developed for the treatment of hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency. It utilizes an adeno-associated virus serotype 5 (AAV5)-based vector to deliver a functional copy of the human SERPING1 gene, which encodes C1-INH, under the control of a liver-selective promoter. The goal is to enable hepatocytes to produce and secrete functional C1-INH protein, thereby addressing the underlying cause of HAE in affected individuals. BMN 331 is administered as a single intravenous infusion and was designed as a one-time treatment intended to provide long-term correction by restoring normal levels of circulating C1-INH protein[1][3][4][5][6][7][8].