OTL-105

OTL-105 is an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being developed for the treatment of hereditary angioedema (HAE), a rare and potentially life-threatening disorder characterized by recurrent episodes of severe swelling. The therapy aims to deliver a functional copy of the SERPING1 gene, which encodes C1 esterase inhibitor (C1-INH), into a patient’s own HSCs using a lentiviral vector. These modified cells are then transplanted back into the patient, enabling durable production of functional C1-INH protein and potentially preventing HAE attacks. Preclinical studies have demonstrated high levels of SERPING1 expression and C1-INH production in relevant cell types following treatment with OTL-105[3][4][5][6].