Mustang Bio and SIRION Biotech GmbH has announced a licensing agreement under which Mustang has acquired rights to SIRION’s LentiBOOST™ technology for the development of MB-207, Mustang’s lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, who have been previously treated with a hematopoietic stem cell transplantation (HSCT) and for whom re-treatment is indicated. LentiBOOST™ is SIRION’s proprietary non-cytotoxic transduction enhancer for lentiviral vectors.
Under the terms of the agreement, SIRION will receive an undisclosed upfront payment and development and sales milestones, as well as royalties on future product sales.
“We are pleased to enter this agreement with SIRION to enable the utilization of the LentiBOOST™ technology in our development of MB-207,” said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang. “Transduction enhancers were added to the cell processing of the lentiviral gene therapy in 2019 and several advantages have been observed. We look forward to incorporating LentiBOOST™ into MB-207, for which we plan to file an investigational new drug application (“IND”) with the U.S. Food and Drug Administration (“FDA”) to initiate a pivotal Phase 2 clinical trial for XSCID in patients over the age of two who have received prior HSCT in the next few months.”
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“LentiBOOST™ was developed to improve lentiviral transduction of therapeutic cell types like T-cells and hematopoietic stem cells. This technology enables a robust and reproducible process and the reduction of manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product while at the same time improving clinical efficacy,” said Christian Thirion, Ph.D., Chief Executive Officer and founder of SIRION. “We are delighted that Mustang has chosen the LentiBOOST™ technology to develop and bring this groundbreaking XSCID therapy to patients.”
MB-207 is a lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, who have been previously treated with a hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated. The lentiviral gene-therapy method employed in MB-207 was co-developed by scientists at the National Institute of Allergy and Infectious Diseases (“NIAID”), part of the National Institutes of Health, and St. Jude Children’s Research Hospital. MB-207 has been studied at NIAID since 2012 and continues to be assessed in a NIAID-supported Phase 1/2 clinical trial for XSCID in patients over the age of two who have received prior HSCT. Mustang expects to file an IND with the FDA to initiate a multi-center pivotal Phase 2 clinical trial of MB-207 in this patient population in the fourth quarter of 2020. The FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to MB-207 for the treatment of XSCID in previously treated patients with HSCT.