According to a story from BioSpace, SIRION Biotech GmbH and Mustang Bio, Inc. have announced a new agreement for licensing the use of SIRION’s proprietary LentiBOOST™ technology for use by Mustang in their development of a lentiviral gene therapy, designated MB-207. It is being developed as a gene therapy for patients living with the X-linked form of severe combined immunodeficiency. It is intended for use in patients that have already received a stem cell transplant.
About Severe Combined Immunodeficiency (SCID)
Severe combined immunodeficiency is a form of primary immunodeficiency. It is a genetic disorder which is characterized by the improper development of critical B-cells and T-cells, which play an essential role in the operation of the immune system. There are nine different genes that, when mutated, can lead to the development of severe combined immunodeficiency. In most cases, these mutations are not inherited. The X-linked form is the result of an inherited recessive mutation of a gene found on the X chromosome. These patients are extremely susceptible to infections, as they effectively lack any immune function whatsoever. Symptoms of the disorder include frequent and severe infections of either viral, bacterial, or fungal origin; failure to thrive, diarrhea, and interstitial lung disease. Ear infections, candidiasis (an oral fungal infection), and pneumonia are common. The most common treatment for this disease is a bone marrow transplant, which has a good rate of success if conducted in the first three months of life; gene therapy may one day be an alternative. To learn more about severe combined immunodeficiency, click here.
As part of this agreement, SIRION shall receive an upfront payment, milestones on development and sales, and royalties on all potential future sales of the gene therapy.
About MB-207
MB-207 has been in development for several years and is currently being evaluated in a phase 1/2 clinical trial of patients over two years of age that have received a stem cell transplant. Mustang is aiming to initiate a full phase 2 trial with MB-207 before the end of the year. The gene therapy has earned the US Food and Drug Administration (FDA) Rare Pediatric Disease designation and Orphan Drug designation.
