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MB-217

Development stage: Preclinical
Lead developer: Mustang Bio
Modality: Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
Administration: Intravenous

Overview

MB-217 is an investigational gene therapy and hematopoietic stem cell therapy being developed for the treatment of X-linked combined immunodeficiency diseases (X-CID). The therapy targets the interleukin-2 receptor subunit gamma (IL-2Rγ), acting as an inhibitor of this receptor. X-CID is a rare congenital disorder affecting immune system function, and MB-217 aims to restore immune competence by correcting the underlying genetic defect in hematopoietic stem cells. The drug is currently in preclinical development, with Mustang Bio as its primary developer[1][2].

Targets

IL2RG (Interleukin-2 receptor gamma subunit)

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