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works with gene-editing technology and treats children with blood disorders and cancers. “I am very enthusiastic about this first step towards bringing what are now complicated cell and gene-based therapies to a much broader population.”
What Adair and her coauthors describe in their paper is a system that deploys lentiviruses to insert into HSCs a healthy copy of a gene. Lentiviral gene therapy is also the technology that Bluebird Bio (NASDAQ: BLUE) is using in three clinical studies to genetically alter the HSCs of patients with sickle cell disease, beta-thalassemia, and adrenoleukodystrophy. Bluebird recently said it had changed some manufacturing processes without having to re-do clinical trials, as some observers had worried could happen.
Bluebird officials could not be reached for comment about the benchtop system. Sorrentino of St. Jude called the Prodigy box a potential “competing model for companies now commercializing gene therapy.”
Many academic and for-profit researchers are also using gene editing, especially the widespread CRISPR-Cas9 system, to alter genes in HSCs and develop treatments. Adair says the “gene therapy in a box” could be used with CRISPR-Cas9, too. But in some cases, the Prodigy box wouldn’t be ready as is. One popular way to get the “scissors” of CRISPR-Cas9 into cells to make their cuts is by zapping the cells with electricity—a process called electroporation. The Prodigy box would have to be modified to attach to an electroporator, but Adair says the system is built to accommodate additional hardware and software (or as she puts it, “a choose-your-own-adventure”).
There are other improvements to make. Adair’s Prodigy tests showed less success with stem cells collected directly from bone marrow, which is no surprise. They are more difficult to genetically modify and transplant than stem cells that have been collected from circulating blood. (Patients can be given drugs to chase, or “mobilize” stem cells out into the circulating blood, but for some diseases and some patients, mobilization isn’t an option.) Adair says changes to the machine’s software and to the chemicals used for the gene modification should help the system’s performance with bone marrow cells.
Another hurdle is intellectual property. Adair’s employer Fred Hutch, as the center is known, and Miltenyi are in dispute over the rights to the software program, Adair says. With a resolution, and with a successful first trial in Fanconi anemia, Adair says she thinks companies will adopt the Prodigy system. But ideally, she says, “I’d love to make this readily available to everyone. If this is in hands of graduate students all over the country, the possibilities are endless.”