Mustang Bio granted RMAT designation for MB-107 for XSCID
Mustang Bio and St. Jude Children's Research Hospital have announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted the Regenerative Medicine Advanced Therapy designation by the U.S. Food and Drug Administration. Mustang and St. Jude, the nation's leading hospital dedicated to understanding, treating and curing childhood cancer and other life-threatening diseases, established a partnership to continue development of the lentiviral gene therapy in August 2018. Under the terms of the RMAT designation, the FDA will help facilitate the program's expedited development and review and provide guidance on generating the evidence needed to support the approval of MB-107 for XSCID. RMAT designation was granted to MB-107 based on positive Phase 1/2 clinical data for infants with XSCID under the age of 2, which were published in the New England Journal of Medicine in April 2019.