Mustang Bio and Minaris Regenerative Medicine GmbH have signed an agreement to enable technology transfer and GMP clinical manufacturing of Mustang’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, in Europe.
Under the terms of the agreement, Minaris will perform technology transfer of the manufacturing and analytical processes, as well as their adoption to the European regulatory environment, for the GMP-compliant manufacturing of the drug product at its site in Ottobrunn, Germany, with the goal of supplying clinical trials in Europe.
“We look forward to a productive and successful partnership with Mustang where Minaris will be able to support them with our extensive experience in the clinical and commercial manufacturing of autologous gene therapies,” said Dusan Kosijer, Managing Director of Minaris. “We are eager to work together with Mustang in the fight against this devastating disease.”
“This agreement with Minaris is an important step in supporting expansion of our MB-107 pivotal clinical trial into Europe,” said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang. “We look forward to working with Minaris to grow our geographic footprint and bring this potential life-saving therapy to XSCID patients in need internationally.”
MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. Mustang submitted an investigational new drug application (“IND”) to the FDA to initiate a pivotal multi-center Phase 2 clinical trial of MB-107 in this same patient population. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplantation. The primary efficacy endpoint will be event-free survival. The initiation of this trial is expected soon. Mustang is targeting topline data from this trial in the second half of 2022.
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The FDA granted Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy Designations to MB-107 for the treatment of XSCID in newly diagnosed infants.
X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births. It is characterized by 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. Patients with XSCID have no T-cells or natural killer cells. Although their B-cells are normal in number, they are not functional. As a result, XSCID patients are usually affected by severe bacterial, viral or fungal infections early in life and often present with interstitial lung disease, chronic diarrhea and failure to thrive.
The specific genetic disorder that causes XSCID is a mutation in the gene coding for the common gamma chain (γc), a protein that is shared by the receptors for at least six interleukins. These interleukins and their receptors are critical for the development and differentiation of immune cells. The gene coding for γc is known as IL-2 receptor gamma, or IL2RG. Because IL2RG is located on the X-chromosome, XSCID is inherited in an X-linked recessive pattern, resulting in almost all patients being male.