November 23, 2020 -- Minaris Regenerative Medicine has signed an agreement with Mustang Bio to enable technology transfer and good manufacturing practice (GMP) clinical manufacturing of Mustang's MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency (XSCID).
Under the agreement, Minaris will perform technology transfer of the manufacturing and analytical processes, including adoption of the European regulatory environment, for the GMP-compliant manufacturing of the drug product at its site in Ottobrunn, Germany. This drug supply will support clinical trials in Europe.
MB-107 is currently being evaluated in a phase I/II clinical trial for XSCID in newly diagnosed infants at St. Jude Children's Research Hospital, University of California, San Francisco Benioff Children's Hospital, and Seattle Children's Hospital.
Mustang Bio has submitted an investigational new drug application to the U.S. Food and Drug Administration (FDA) to initiate a phase II clinical trial. The trial would enroll 10 patients (who together with the 15 patients enrolled in the phase I trial) will be compared to patients who have undergone hematopoietic stem cell transplantation. The primary endpoint will be event-free survival. This trial is expected to begin soon with the goal of having topline data in the second half of 2022.
XSCID is a genetic disorder caused by a mutation in the gene coding for the common gamma chain, a protein that is shared by the receptors for at least six interleukins. The disease 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.
The FDA granted rare pediatric disease and orphan drug and regenerative medicine advanced therapy designations to MB-107 for the treatment of XSCID in newly diagnosed infants.