SIRION Biotech licenses LentiBOOST™ Technology to Mustang Bio for development of XSCID gene therapy

Martinsried, Germany: – Specialist viral vector supplier SIRION Biotech GmbH (SIRION) has jointly announced  a licensing agreement with Worcester, Massachusetts-based Mustang Bio, Inc. (Mustang) under which the American company acquires rights to SIRION’s LentiBOOST™ technology for the development of its MB-207 lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (XSCID).

XSCID, or ‘bubble boy disease’, has previously been treated with a hematopoietic stem cell transplantation (HSCT). The MB-207 therapy is particularly applicable to patients for whom re-treatment is indicated.

Leveraging LentiBOOST™ for enhanced transduction

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.

Mustang’s President and CEO, Dr. Manuel Litchman, commented: “We are pleased to enter this agreement with SIRION to enable the utilization of the LentiBOOST™ technology in our development of MB-207.”

“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) in the next few months 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,” Dr. Litchman explained.

Improving lentiviral transduction

SIRION CEO and founder Dr. Christian Thirion commented: “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.”

“We are delighted that Mustang has chosen the LentiBOOST™ technology to develop and bring this groundbreaking XSCID therapy to patients,” Dr. Thirion concluded.

About SIRION Biotech GmbH

Founded at Martinsried, near Munich, in 2007, SIRION Biotech has become Europe’s leading commercial supplier of viral vectors used for genetic research, clinical target validation, cell and gene therapy and vaccination studies. It is SIRION’s mission to change the paradigm for viral vector supplies, with their ability to develop and supply all major viral vector types (adenovirus, lentivirus, AAV) within a matter of weeks at the concentration titres and quantities needed for preclinical human and animal testing.

Its unique focus on improving transduction efficiencies and safety makes SIRION Biotech a valuable technology partner for gene and cell therapy trials. LentiBOOST^™ transduction reagent is actively used to improve, among others, hematopoietic cell transductions in clinical trials. NextGen AAV capsid evolution projects aim to improve tissue targeting and immune escape of capsids to usher in a new generation of therapeutics for international gene therapy companies. SIRION offerings include the RNAiONE^™ knockdown validation platform for short-hairpin RNA (shRNA) screening along with a full range of virus related services, ranging from viral particle production to virus driven cell modelling.

SIRION Biotech International is a wholly-owned subsidiary of SIRION Biotech GmbH providing custom engineering and manufacturing services of viral vectors for the life sciences industry.

Learn more at: www.sirion-biotech.com

About Mustang Bio and MB-207

Founded by Fortress Biotech, Inc., Mustang Bio, Inc. is 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. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to out license or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang was. (NASDAQ: FBIO).

MB-207 is 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. It employs a lentiviral gene-therapy method 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.

For more information, visit: www.mustangbio.com

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