Orchard links with Pharming to develop HSC gene therapy for hereditary angioedema

UK and USA-based clinical-stage gene therapy specialist Orchard Therapeutics (Nasdaq: ORTX) has entered into a strategic collaboration with Dutch firm Pharming Group (Euronext Amsterdam: PHARM) to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of hereditary angioedema (HAE), a life-threatening rare disorder that causes recurring swelling attacks in the face, throat, extremities and abdomen.

News of the deal saw Pharming’s shares edge up 2.2% to 0.98 euros in morning trading, but Orchard was flat in pre-market activity.

OTL-105 is an investigational HSC gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks. OTL-105 inserts one or more functional copies of the SERPING1 gene into patients own HSCs ex vivo which are then transplanted back into the patient for potential durable C1-INH production. In preclinical studies, to date, OTL-105 demonstrated high levels of SERPING1 gene expression via lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs. Furthermore, the program achieved production of functional C1-INH protein, as measured by a clinically validated assay.