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IONS - Biotech News Recap: Mustang Bio Soars On XSCID Data
Ionis Pharmaceuticals Inc.
Mustang Bio (MBIO)- As I write this, shares have risen by over 300% after hours after the company announced that the New England Journal of Medicine had published data from St. Jude Children's Research Hospital regarding an early stage gene therapy trial. Specifically, the Phase 1/2 study is evaluating a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID (also known as "bubble boy disease").
The treatment essentially transfers a normal copy of the IL2RG gene to bone marrow stem cells in newly diagnosed infants