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All 23 treated patients are alive at 2.6-year median follow-up without evidence of malignant transformation Data representing largest cohort of infants with XSCID treated with gene therapy presented...
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Data represent largest cohort of infants with XSCID, also known as bubble boy disease, who received lentiviral gene therapy with the longest follow-up to date Seventeen of 18 patients with follow-up...
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FDA removes clinical hold for pivotal Phase 2 MB-107 clinical trial Company plans to enroll first patient in MB-107 pivotal trial in the second quarter of 2021 Clinical outcomes in investigator-IND...
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MB-107 preceded by low-dose busulfan conditioning continues to be well tolerated and results in development of functional immune system in newly diagnosed infants with XSCID Enhanced transduction...