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Mustang Bio Announces Data on Treatment with Lentiviral Viral Vector Gene Therapy for X-Linked Severe Combined Immunodeficiency Selected for Oral Presentation at American Society of Gene & Cell Therapy 25th Annual Meeting
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
About this update from Mustang Bio, Inc.
[{"type":"text","content":"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 greater than 6 months achieved robust immune reconstitution All 23 treated patients are alive and 20 patients with follow-up greater than 4 months recovered from pre-existing infections, are off protective isolation and prophylactic antimicrobials, and have normal growth velocity WORCESTER, Mass., May 03, 2022 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that interim Phase 1/2 data on treatment with the same lentiviral vector used in MB-107, Mustang’s lentiviral gene therapy for X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, in newly diagnosed infants under the age of two, were selected for an oral presentation during the Clinical Trials Spotlight Symposium at the American Society of Gene & Cell Therapy (“ASGCT”) 25th Annual Meeting taking place May 16-19, 2022, both virtually and in Washington, D.C. The presentation will include updated data from a multicenter Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital (“St. Jude”), UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. The lentiviral gene therapy is also being assessed in a Phase 1/2 clinical trial at the National Institute of Allergy and Infectious Diseases (“NIAID”), part of the National Institutes of Health, for XSCID patients who have been previously treated with hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated. The data include 23 infants with XSCID treated with the lentiviral vector at a median age of 3 months (range: 2.4-13.8) with a median follow-up of 2.4 years (range: 1.4 months to 5.4 years), making it the largest known cohort of infants treated with lentiviral (LV) gene therapy with the longest follow-up. Transduced autologous bone marrow CD34+ cells were generated for all patients with a median vector copy number (VCN) of 0.81/cell (range: 0.1...