BMN 331

BMN 331 is an experimental gene therapy for hereditary angioedema, delivered by adeno-associated virus type 5 and targeting the SERPING1 gene.[1][2]

References

1. Betschel, Stephen D.; Banerji, Aleena; Busse, Paula J.; Cohn, Danny M.; Magerl, Markus (August 2023). "Hereditary Angioedema: A Review of the Current and Evolving Treatment Landscape". The Journal of Allergy and Clinical Immunology: In Practice. 11 (8): 2315–2325. doi:10.1016/j.jaip.2023.04.017. PMID 37116793. S2CID 258390214.
2. Zanichelli, Andrea; Montinaro, Vincenzo; Triggiani, Massimo; Arcoleo, Francesco; Visigalli, Debora; Cancian, Mauro (3 April 2022). "Emerging drugs for the treatment of hereditary angioedema due to C1-inhibitor deficiency". Expert Opinion on Emerging Drugs. 27 (2): 103–110. doi:10.1080/14728214.2022.2105834. PMID 35876094. S2CID 251038265.