Shots:
The interim results from the P-I/II study evaluates MB-107 in 23 newly diagnosed infants aged ≤2yrs. with XSCID
The results showed that all patients are alive at 2.6yr. median follow-up without evidence of malignant transformation at a median age of 3mos. & experienced complete hematopoietic recovery, transduced autologous bone marrow CD34+ cells with a median VCN of 0.81/cell & median CD34+ cell dosage of 9.61×106/kg
Patients also received busulfan at a dose of 22 mg*hr/L before cell infusion & was well tolerated, 15 patients were able to discontinue IV immunoglobulin & 14 have successfully immunized. The company plans to initiate the P-II trial of MB-107 with an expected IND filing in H2’22
Ref: Globe Newswire | Image: Mustang Bio
