Gene therapy for hereditary angioedema

Country

United Kingdom

Two European companies have decided to combine their expertise to develop a candidate gene therapy for the treatment of hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. Pharming Group NV, which already has an HAE product on the market, is to pay Orchard Therapeutics Plc for rights to a preclinical gene therapy programme for the same disease. The therapy, OTL-105, is an investigational ex vivo autologous haematopoietic stem cell therapy delivered with a lentiviral vector.