Mustang Bio Positive Data on ‘Bubble Boy Disease’ Cure

Mustang Bio has reported positive data from a Phase 1/2 clinical trial of a lentiviral gene therapy for treating newly diagnosed infants under two years old with XSCID, also referred to as SCID-X1 and commonly known as “bubble boy disease.” Under a licensing agreement with St. Jude Children’s Research Hospital, Mustang says it will develop the lentiviral gene therapy for commercial use as MB-107. Just-published data in the New England Journal of Medicine show that: 

• Bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.
• All patients cleared previous infections and are growing normally.
• Seven of eight infants treated have developed normal immunoglobulin M levels to date.
• Most patients were discharged from the hospital within one month. 

"The results have been very good thus far," said St. Jude’s Ewelina Mamcarz. “We’ve been able to restore a full immune system pretty quickly.”