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Mustang Bio Announces First Patient Successfully Treated by Ex Vivo Lentiviral Gene Therapy to Treat RAG1 Severe Combined Immunodeficiency
WORCESTER, Mass., July 27, 2022 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on
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[{"type":"text","content":"WORCESTER, Mass., July 27, 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 the first patient successfully received LV-RAG1 ex vivo lentiviral gene therapy to treat recombinase-activating gene-1 (“RAG1”) severe combined immunodeficiency (“RAG1-SCID”), in an ongoing Phase 1/2 multicenter clinical trial taking place in Europe. LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID. Patients with SCID have mutations in blood stem cell genes that are responsible for the development and function of infection-fighting immune cells. As a result, they are unable to mount a normal defense response against infections. The administration of LV-RAG1 includes reduced intensity conditioning prior to reinfusion of the patients’ own gene-modified blood stem cells. “The patient was administered LV-RAG1 without any complications. LV-RAG1 allowed the patient’s body to create a functioning immune system, which is responding well to the standard vaccinations for newborns,” said Arjan Lankester, Principal Investigator and Professor of Pediatrics and Stem Cell Transplantation at Leiden University Medical Centre (“LUMC”). Manuel Litchman, M.D., President and Chief Executive Officer of Mustang said, “This first successful administration to a RAG1-SCID patient of a stem-cell based gene therapy represents a significant positive step forward for our MB-110 development program. This treatment, along with our X-linked severe combined immunodeficiency (“XSCID”) programs, which includes MB-107 and MB-207, has established Mustang as a leader in developing treatments for SCID patients, who are in great need of these life-saving therapies. XSCID and RAG1-SCID make up almost 60% of all SCID cases combined.1 We look forward to continuing to advance these clinical candidates, including plans to initiate a multicenter pivotal Phase 2 trial for MB-107 under Mustang’s IND in the second half of this year.” LV-RAG1 has been granted Orphan Drug Designation by the European Medicines Agency. Additional clinical trial sites are ex...