A Cure For A Disease Made Well-Known by John Travolta
Mustang Bio Inc. (Nasdaq: MBIO) stock is soaring following a scientific breakthrough that reportedly cured eight infants suffering from the deadly “Bubble Boy” immune system disorder.
Mustang Bio, backed by health-care serial entrepreneur Lindsay Rosenwald, has licensed the treatment for the rare disease from St. Jude Children’s Research Hospital, where the treatments took place. The hospital developed a cutting-edge gene therapy that uses deactivated HIV as a delivery system.
Market Watch reported, “Data from a phase 1/2 clinical trial evaluating the safety and efficacy of a lentiviral vector to transfer a normal copy of the IL2RG gene to bone marrow stem cells in newly diagnosed infants under the age of two with XSCID, showed that bone marrow harvest, busulfan conditioning and cell infusion were well tolerated, all patients cleared previous infections and are growing normally and most patients were discharged from the hospital within one month.
These [gene therapy] patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1.
Prior to this gene therapy, standard treatment for SCID-X1 has been bone marrow transplants from a matching sibling donor. Those donors are in short supply, with 80 percent of SCID-X1 lacking such a match. That treatment also has serious side effects and isn’t a cure.”
Dr. James Downing, president and CEO of St. Jude, said: “The gene therapy … builds on our legacy of combining traditional treatments with new technologies. This experimental treatment used groundbreaking technology … that then led to [a] cure.”
“A key consideration for this particular gene therapy was a prior protocol developed in the early 2000s; that treatment, while effective, had an unexpected side effect of activating a gene that produced leukemia.
Safeguards were an essential part of the new SCID-X1 therapy, including on the virus used to deliver the therapy.”
Dr. Mamcarz added: “All of the active parts of the [HIV] virus have been taken out with only a shell remaining. And, this virus was able to effectively deliver the healthy copy of the [necessary] gene into the stem cells in a way that was not possible before.”
“The [virus] vector has also been designed with insulators that decide where the vector lands in the cell [to prevent] activating unintended genes that could cause side effects,” she continued.
The stock is trading up more than 100% on news of the cure.
MBIO should be considered a buy now.
Look for an entry at $5.65 with a price target of at least $8.78 based on the pattern in the chart.