Orchard Therapeutics (London, U.K.) raised $30.7 million in a series A round to develop ex vivo autologous hematopoietic stem cell gene therapies for primary immune deficiencies, metabolic diseases and hematological disorders. The newco's lead program is a lentiviral gene therapy in the clinic to treat severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID).
F-Prime Capital led the round. UCL Business plc also participated, as did the UCL Technology Fund, which University College London launched this year.
Orchard said it has development partnerships with five research institutions: UCL, the Great Ormond Street Hospital for Children NHS Foundation Trust, the University of Manchester, the University of California Los Angeles and Boston Children's Hospital.
The start-up said interim data from March showed the ADA-SCID gene therapy led to "significant immune reconstitution" and 100% survival among 32 patients treated at Great Ormond and UCLA.
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) is developing Strimvelis (GSK2696273), a gene therapy that delivers ADA via a retroviral vector for ex vivo hematopoietic stem cell therapy, to treat ADA-SCID in patients for whom no suitable stem cell donor is available. Last month, EMA's CHMP recommended Strimvelis' approval.
Article adapted from BioCentury.