First Patient Successfully Treated by Ex Vivo Lentiviral Gene Therapy for RAG1-SCID

Anna Osborne
1 August 2022
Clinical Trials
Gene Therapy
Stem Cells
Viral/Non-Viral Vectors
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Clinical-stage biopharmaceutical company Mustang Bio, has announced that the first patient in their ongoing Phase I/II clinical trial has received the ex vivo lentiviral-based gene therapy, MB-110, to treat recombinase-activating gene-1 (RAG1) severe combined immunodeficiency (RAG1-SCID).

This first-in-class ex vivo gene therapy, MB-110, uses the lentiviral vector LV-RAG1 to transduce patient cells and treat RAG1-SCID.

Mustang signed a worldwide licensing agreement in 2021 for use of the Leiden University Medical Centre’s (LUMC) LV-RAG1 vector – produced by Dr Frank J.Staal, a Molecular Immunologist and Professor of Modern Stem Cell Biology at LUMC.

“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. We look forward to continuing to advance these clinical candidates,” comments Manuel Litchman, President and CEO of Mustang.

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SCID is caused by mutations in the genes responsible for the development and functioning of infection-fighting immune cells. Patients suffering from SCID are therefore unable to establish an effective immune response against infections.

Without treatment, via allogenic hematopoietic iPCS transplant or gene therapy to correct autogenic iPCS, patients will not survive beyond infancy.

“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 LUMC.

Development continues as LV-RAG1 has been granted Orphan Drug Designation by the EMA and Mustang proposes for multiple clinical trial sites to be added shortly.

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