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Worcester biotech to treat bubble boy disease

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.

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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.

– Digital Partners -

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