Gene Therapy, Licensing

Mustang Bio ventures into gene therapy, licensing ‘bubble boy disease’ treatment from St. Jude

When Mustang Bio $MBIO inked the lease deal for its Worcester, MA manufacturing facility last year, CEO Manny Litchman said a more cost effective manufacturing approach would lend a competitive edge to their early-stage CAR-T programs. As it turns out, gene therapy will also be part of that game plan.

Licensing from St. Jude Children’s Research Hospital in Memphis, Mustang adds an ex vivo lentiviral gene therapy to for one form of “bubble boy disease” to its pipeline, which has up till now been stocked with cancer-fighting cell therapies.

Manuel Litchman

“With our team’s extensive expertise in viral vector design, manufacturing and transduction, we are building a fully integrated cell and gene therapy company, with the goal of leveraging the transformative potential of these technologies to bring life-saving treatments to patients in need,” Litchman said in a statement.

Formally called severe combined immunodeficiency (SCID), “bubble boy syndrome” comes in several variations based on the exact cause for the abnormal functions of T and B cells. St. Jude’s therapy, developed in the lab of its director of experimental hematology Brian Sorrentino, targets the most common form: X-linked SCID.

Brian Sorrentino

It works by genetically modifying a patient’s own blood stem cells and reinfusing them, in a process that starts with giving the patients a low dose of the cancer drug busulfan. Ultimately, the treatment is designed to enable “immune system reconstitution and the resolution of disseminated infections” so that the patients not only live beyond infancy but do so without regularly receiving intravenous immunoglobulin.

The idea is not exactly new. GlaxoSmithKline had been marketing Strimvelis, its own gene therapy aimed at a less common variation of the disease called ADA-SCID, for more than a year before handing it off to Orchard Therapeutics, a UK biotech upstart run by some of its former staffers who helped develop it.

Just this morning, Orchard unveiled its $150 million mega-round to bring that treatment, and its other experimental therapies, to a broader market faster.

Meanwhile, the new partners at Mustang and St. Jude will focus on pushing through their own Phase I/II clinical trials. In the one involving patients under age 2, eight have already been treated to encouraging effects.

“Our therapy has been well tolerated thus far, and none of the infants required any blood product support after low dose of busulfan,” said Ewelina Mamcarz, who’s leading the study. “Most importantly, we observe recovery of all cells of the immune system, which is truly an achievement over prior gene therapy trials, where B cell reconstitution did not occur, and patients required intravenous immunoglobulin for life.”


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