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Mustang Bio's 'bubble boy' gene therapy gets US FDA regenerative medicine tag

The U.S. Food and Drug Administration granted regenerative medicine advanced therapy designation to Mustang Bio Inc.'s experimental MB-107 gene therapy for a rare genetic immune disorder known as bubble boy disease.

The designation allows for expediting the drug development and review processes for therapies that show the potential to treat serious or life-threatening diseases or conditions.

Worcester, Mass.-based Mustang Bio is co-developing MB-107 with St. Jude Children's Research Hospital in Memphis, Tenn., as a potential treatment for X-linked severe combined immunodeficiency, or XSCID. This inherited disorder of the immune system occurs almost exclusively in males.

XSCID is very rare, occurring in about 1 per 225,000 births, and causes recurrent and persistent infections due to the lack of necessary immune cells to fight off certain bacteria, viruses and fungi. The disease's nickname derives from a famous case in which a boy with XSCID named David Vetter lived in a protective sterile plastic "bubble" until his death at age 12.

Results of a phase 1/2 trial showed that seven of eight boys treated with MB-107 showed normal levels of immune cells, known as T cells and natural killer cells, within four months of treatment.

Mustang Bio is a Fortress Biotech Inc. company focused on developing its proprietary cell and gene therapies to treat diseases with unmet needs.

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