When the U.S. Food and Drug Administration (FDA) announced in November 2023 that it was investigating a link between receiving CAR T therapy and developing secondary cancers later on, it spurred uncertainty for patients and researchers alike. As many of the more than 30,000 patients who have received the life-saving treatment face new questions about
Study results formed the basis of the therapy’s FDA approval in April 2024 Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of
Scientists from UNIGE and HUG have developed CAR-T cells capable of targeting malignant gliomas while preserving healthy tissues. Glioblastoma is the most common and aggressive primary brain tumor—average survival after diagnosis is less than two years—and current treatments remain ineffective. In recent years, immunotherapies have been giving patients hope, albeit with relatively modest success. A
In an early Stanford Medicine study, CAR-T cell therapy helps some with intractable lymphoma, but those who relapse have few options. Modifying the therapy’s molecular target improved response. CAR-T cell therapy, which targets a specific protein on the surface of cancer cells, causes tumors to shrink or disappear in about half of patients with large
In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large Stanford Medicine study finds the risk is low and not related to the CAR-T cells. A large study by researchers at Stanford Medicine has found that the risk of secondary blood cancers after CAR-T cell therapy — a
Administering gene therapy in both ears of five children with DFNB9 led to additional benefits compared to previous trial, including sound source localization and hearing in noisy environments. Key Takeaways A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The
Regeneron presented results from an ongoing Phase I/II clinical trial for its investigational gene therapy, DB-OTO, at the annual American Society of Gene and Cell Therapy (ASGCT) meeting, being held in Baltimore, Md. from May 7–11. DB-OTO, a gene therapy for genetic deafness, improved hearing in one child, treated at 11 months old to normal levels within
First-of-its-kind trial suggests CRISPR acting inside the body can be safe and effective At a glance: Results from a small proof-of-concept study indicate that CRISPR gene editing is safe and can improve vision in some people with inherited blindness. In the multicenter clinical trial, 11 of the 14 individuals treated had measurable improvements on at
Fred Hutch Cancer Center scientist Jennifer E. Adair, PhD, is on a mission to foster worldwide collaboration on potentially curative gene therapies. Holder of the Fleischauer Family Endowed Chair in Gene Therapy Translation, Adair just co-authored two articles published today in Science Translational Medicine as part of a special series on global access to these therapies that she
EXACT Trial Demonstrated Improvements in Exercise Duration, Ischemia, and Decreased Symptoms for Patients with Advanced CAD Final 12-month data from the EXACT trial demonstrates safety and efficacy results for a vascular endothelial growth factor (VEGF) gene therapy treatment for patients who have advanced coronary artery disease (CAD). The late-breaking results were presented today at
