August 13, 2018

Worcester biotech to treat bubble boy disease

Photo/Grant Welker
Manny Litchman, the Mustang Bio CEO, at an event in June to mark the opening of the company's Worcester space.

The Worcester facility of New York biotech Mustang Bio will play a role in a new exclusive partnership with a leading children's medical center to help develop treatments for an immunodeficiency disorder.

Mustang Bio, a subsidiary of Fortress Biotech, said Monday it has entered into an agreement with St. Jude's Children's Research Hospital in Tennessee to develop gene therapy treatments for X-linked severe combined immunodeficiency (X-SCID) -- commonly called bubble boy disease.

Mustang Bio opened its Worcester facility in June, where it plans to process cells to help treat cancer and a variety of diseases.

"We are thrilled to announce the expansion of our pipeline into gene therapy for patients with X-SCID, a natural fit for our Worcester, Mass. cell processing facility," Mustang Bio CEO Manuel Litchman said in a statement."

The therapy includes a low dose of a drug used to treat cancer prior to the reinfusion of the patients' own gene-modified blood stem cells, which will be processed at Mustang Bio's Worcester facility.

So far, the therapy has yielded positive results in a multi-center trial in infants under the age of two. The therapy is also being studied in patients over the age of two in a separate trial, according to the company.

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