A gene therapy for children with X-linked severe combined immunodeficiency (X-SCID) will be made at Mustang Bio’s facility in Massachusetts, which opened in June.

Biotech firm Mustang Bio has partnered with St. Jude Children’s Research Hospital to develop a lentiviral gene therapy for the rare disease, commonly known as bubble boy disease, caused by a lack of immune cells necessary to fight off viruses, bacteria and fungi.

As the therapy progresses through early-phase clinical trials, Mustang will begin to manufacture batches of the gene therapy from its 27,000 sq. ft. facility in Worcester, Massachusetts, which opened its doors in June this year.

“The manufacturing and supply chain orchestration of the XSCID gene therapy program is a natural fit for our Worcester cell processing facility, as the process is similar to the way in which we are manufacturing our CAR T therapies.

“The starting patient cell population for the XSCID program, which is hematopoietic stem cells (HSCs) in this case rather than T cells for CAR T therapies, will be shipped to our Worcester facility from clinical trial sites,” the firm told BioProcess Insider.

“Mustang will genetically modify the cells at our manufacturing facility to create cells that contain normal copies of the gene that is mutated in XSCID, and then distribute the cells back to the trial sites for reinfusion.”

Gene therapies for the ongoing Phase 1/2 trials are currently being manufactured at St. Jude, Mustang anticipates manufacturing will be transferred to the facility to support clinical development under its own IND beginning in the fourth quarter of 2020.

Medical institution partnerships

The partnership with St. Jude Children’s Research Hospital to develop a lentiviral gene therapy for the disease, commonly known a s bubble boy disease, is the fourth partnership that Mustang, a subsidiary of Fortress Biotech ($FBIO), has secured with a medical institution.

“Our strategy of completing the early stage work at top medical institutions and then filing our own IND down the line is more cost effective and de-risks our CAR T and gene therapy programs,” we were told.

Mustang has inked partnerships with City of Hope, Fred Hutchinson Cancer Research Center and Harvard University / Beth Israel Deaconess Medical Center.