Mustang Bio gene therapy for bubble boy disease nabs advanced therapy tag in U.S.

Thinly traded micro cap Mustang Bio (NASDAQ:MBIO) is up 12% premarket on light volume in response to its announcement that the FDA has designated MB-107, its lentiviral gene therapy, a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of a rare inherited disorder called X-linked severe combined immunodeficiency, also known as bubble boy disease, named for child who was placed in a protective enclosure due to the profound infection risk since he had no functioning immune system.

RMAT is akin to Breakthrough Therapy status for drugs/biologics. It provides for the accelerated review of the marketing application and more intensive guidance on development.

The company is developing MB-07 with St. Jude Children’s Research Hospital.

https://seekingalpha.com/news/3493941-mustang-bio-gene-therapy-bubble-boy-disease-nabs-rmat-tag-u-s-shares-12-percent-premarket