CRISPR Therapeutics. Current clinical trials. Last year, Chinese scientist He Jiankui shocked the medical community by announcing that he had altered the genes of two human children. The doubly treated T cells also seemed better at identifying and killing cancer cells compared to only CAR T cells. Knocking out the two genes related to the T cell receptor was also theorized to boost the expression of a lentiviral-inserted gene that produces the CAR, the cancer-targeting T cell receptor called NY-ESO-1. The approach in this study is closely related to CAR T cell therapy, which engineers patients’ own immune cells to fight their cancer, but it has some key differences. Stadtmauer will present the findings next month at the 61st American Society of Hematology Annual Meeting and Exposition in Orlando (Abstract #49). Follow her on Twitter @AshleyJYeager. Before that, I was at Johns Hopkins University where I double-majored in writing and public health. FLICKR, ALEX RITTER, JENNIFER LIPPINCOTT SCHWARTZ, AND GILLIAN GRIFFITHS, NATIONAL INSTITUTE OF HEALTH. See the stories that matter in your inbox every morning. The trial (ClinicalTrials.gov identifier: NCT03399448) results are published in the journal Science. The trial, which is the first of its kind to publish its results, tested three individuals with advanced cancer to see whether or not genetically edited immune cells would be safely tolerated by the patient's bodies. While the study aimed to examine safety only, an initial look at efficacy indicates that the intervention was not a cure. One has died since receiving the treatment. The whole process took more than two years. They then used CRISPR to remove three genes from the T cells, which should make them more effective at seeking out and destroying cancer cells. CRISPR has been touted as a revolutionary tool that has the potential to cure diseases or turn a person’s own T cells into potent cancer killers. The other two removed receptors from the T cells’ surface, paving the way for a fourth edit that inserted a new synthetic T cell receptor that guides the immune cells towards a particular cancer antigen. “First, we can successfully perform multiple edits with precision during manufacturing, with the resulting cells surviving longer in the human body than any previously published data have shown. And so researchers wanted to know: “Can CRISPR live up to its hype?” study coauthor Carl June, a cancer researcher at the University of Pennsylvania, tells HealthDay News. The Ascent is The Motley Fool's new personal finance brand devoted to helping you live a richer life. Could Editas Medicine Be a Millionaire-Maker Stock? These early findings are the first step as we determine if this new, breakthrough technology can help rewrite how we treat patients with cancer and perhaps other deadly diseases,” said Sean Parker, Founder and Chairman of PICI. The patient’s body then takes over and is able to produce new, healthy cells. And in that regard it was successful, showing that genetically-edited cells persisted and functioned for many months and didn’t appear to cause harm. One of the main worries that researchers have about Crispr is that scientists might alter genes to be inherited, a practice called germline engineering. While they don’t yet have a timeline on when the treatment will be commercially available, “we want to get this to patients as soon as possible,” he says. 1. , which together form a $8.6 billion enterprise. None of the patients responded to the therapy, but that isn’t an indictment on the therapy itself. Integrating a foundational collaboration with the University of Pennsylvania (Penn) with the groundbreaking scientific, clinical, and manufacturing expertise, and the demonstrated track record of its founders (Carl June, MD; Bruce Blazar, MD; Bruce Levine, PhD; Yangbing Zhao, MD, PhD; Jim Riley, PhD; and Anne Chew, PhD), Tmunity represents a new center of gravity in translational T-cell medicine.