- Mustang Bio Inc (NASDAQ:MBIO) announced interim data from the Phase 1/2 trial of MB-107, lentiviral gene therapy for X-linked severe combined immunodeficiency (X-SCID), also known as bubble boy disease, in newly diagnosed infants under the age of two.
- X-SCID is characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death if untreated by one year of age.
- The data support the company's plans to initiate a multicenter pivotal Phase 2 trial for MB-107 under its Investigational New Drug application in 2H 2022.
- Transduced autologous bone marrow CD34+ cells were generated for all patients with a median vector copy number (VCN) of 0.81/cell and a median CD34+ cell dose of 9.61x106/kg.
- Also Read: Mustang Bio's CAR T Cell Therapy Shows Favorable Safety Profile, Response Rates In Blood Cancer Patients.
- The treatment was well-tolerated, and all patients experienced complete hematopoietic recovery.
- Severe adverse events occurred in three patients, and all were resolved.
- Seventeen patients had active infections before therapy, and in all cases, these infections cleared. In addition, 15 patients have been able to discontinue intravenous immunoglobulin, and to date, 14 patients have been successfully immunized.
- All patients are currently alive with stable vector marking in all cell lineages without malignant transformation evidence.
- Price Action: MBIO shares are up 5.98% at $0.74 during the market session on the last check Thursday.
