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.
Luckily, eight infants with this bubble boy disease
appear to have been cured thanks to an experimental gene therapy.
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.
The new fetal genome sequencing method paves the way for prenatal detection of a wide range of conditions - not just DiGeorge syndrome, but phenylketonuria (PKU), where babies can't digest the amino acid phenylalanine and severe combined immunodeficiency (sometimes referred to colloquially as "bubble boy disease
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.