RARE Daily

Mustang Licenses Sirion’s Enhancer to Improve Efficiency of Its X-SCID Gene Therapy

October 6, 2020

Rare Daily Staff

Mustang Bio entered a licensing agreement with Sirion Biotech for rights to the company’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, which is also known as bubble boy disease.

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, CEO of Sirion.

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.

X-linked severe combined immunodeficiency (X-SCID) is a rare X-linked genetic disorder that results in the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death by one year of age if untreated. X-SCID is the most common form of severe combined immunodeficiency, affecting approximately one in 50,000 to 100,000 newborns worldwide.

MB-207 is a lentiviral gene therapy for the treatment of patients with X-SCID severe combined immunodeficiency, also known as bubble boy disease, who have been previously treated with a hematopoietic stem cell transplantation 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 hematopoietic stem cell transplantation.

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 hematopoietic stem cell transplantation.

Photo: Christian Thirion, CEO of Sirion Biotech

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