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 (RMAT) designation by the U.S.
The nickname "
bubble boy disease" comes from a famous case in the 1970s -- a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs.
He hopes that Cas9-based therapies for T cell-related disorders, which include autoimmune diseases as well as immunodeficiencies such as "
bubble boy disease," will enter the clinic in the future.
Recently, CDC helped put into practice a newborn screening blood test for Severe Combined Immune Deficiency (SCID), better known as "
Bubble Boy Disease." SCID prevents babies from fighting infections and can cause death.
SCID, often called "
bubble boy disease", causes a defect in the white blood cells that helps protect the body from viruses, bacteria and fungi.
Bubble boy disease is a genetic disorder diagnosed in about 40 to 100 babies annually in the US.
Three products are currently marketed which utilize Enzon's technology: PEG-INTRON marketed by Schering-Plough for hepatitis C, ONCASPAR(R) for Acute Lymphoblastic Leukemia (ALL), and ADAGEN(R) a treatment for a form of Severe Combined Immunodeficiency Disease (SCID), commonly known as the "
Bubble Boy Disease." In addition to three approved products, Enzon has several products in various stages of clinical development by itself and with partners, including additional indications for PEG-INTRON with Schering-Plough.
The strategy should work to treat hemophilia, thalassemia and severe combined immunodeficiency disease (the so-called
bubble boy disease), Daley said.
Accumulating knowledge about severe combined immunodeficiency (SCID) or "
bubble boy disease," a lethal condition in which children are born without a functioning immune system, led to the development of the new compound.