Orchard Therapeutics Licenses Stable Cell Line Technology from GSK for Use in Gene Therapies

Rare Daily Staff

Orchard Therapeutics has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline for use of its proprietary lentiviral stable cell line technology (LV-SCLT) for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.

Wiskott-Aldrich Syndrome (WAS) manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. It is characterized by eczema and an abnormal platelet number and function. Without treatment, the median survival for WAS patients is 14 years of age and treatment with stem cell transplant carries significant risk of mortality and morbidities.

Orchard’s gene therapy OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan for the treatment of WAS, which Orchard acquired from GSK in April 2018.

Orchard’s gene therapy OTL-300 addresses transfusion-dependent beta-thalassemia (TDT), a severe genetic disease characterized by reduced or absent hemoglobin levels that results in severe anemia and ineffective red blood cell production.

The lentiviral stable cell line technology (LV-SCLT) permanently and stably enables all the lentiviral vector components to be introduced into a cell line in one step. Selection and expansion of a resulting clonal producer line in either suspension or adherent culture can deliver consistent levels of high titer lentiviral production comparable to those seen using conventional methods.

“Utilization of a stable cell line provides an opportunity to generate lentiviral vector of consistently high titer and eliminates the need to purchase plasmids prior to the production of each viral vector batch, providing more efficient production processes and shorter lead times,” said Bobby Gaspar, CEO of Orchard.

Orchard plans to submit a biologics license application and marketing authorization application for OTL-103 for the treatment of WAS in the U.S. and EU, respectively, in 2021.

Photo: Bobby Gaspar, CEO of Orchard