Orchard Therapeutics announced that the company has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) for use of their proprietary lentiviral stable cell line technology (LV-SCLT) for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome (OTL-103 for WAS) and transfusion-dependent beta thalassemia (OTL-300 for TDT).
“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, M.D., Ph.D., chief executive officer of Orchard. “By increasing the efficiency of the vector manufacturing process, this technology can provide a key competitive advantage and supports our focus on manufacturing innovations that enable commercial scalability.”
The 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. An overview of this technology, co-authored by Orchard and GSK scientists, was presented at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in October 2019 using work done in the OTL-300 program for TDT*. Under the licenses, GSK has granted patents and pending patent applications related to its LV-SCLT**.
Gaspar continued, “We believe the work completed with OTL-300 utilizing a stable cell line can be leveraged and applied to future development plans in OTL-103 for WAS, which will be especially useful in a commercial setting given the large number of patients diagnosed and living with the disease.”
Orchard plans to submit a biologics license application (BLA) and marketing authorization application (MAA) for OTL-103 for the treatment of WAS in the U.S. and EU, respectively, in 2021.
The terms of the license are not expected to have a material impact on Orchard’s financial position or near-term cash needs.