Orchard Therapeutics is a biotechnology company, develops and commercializes transformative ex-vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in primary immune deficiencies, metabolic diseases, and hematological disorders. The сompany also develops adenosine deaminase severe combined immunodeficiency, mucopolysaccharidosis type IIIA, and ex-vivo lentiviral gene therapy programs.