Phase 3 clinical trial included children younger than 12 years Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients
Experts say careful application of advanced tech could usher in a golden age of healthcare New technologies could enable medicine to progress in leaps and bounds, but only with the right regulatory and ethical frameworks. That was one of the messages from panelists discussing the future of medicine at the Science|Business conference Horizon Europe: The first assessment as
In this review, it’s provided an overview of the major mechanisms used by tumor cells to evade immune defenses and are critically exposed the most optimistic engineering strategies to make CAR-T cell therapy a solid option for solid tumors. CAR-T cell based cell therapy is a moving field, which showed impressive results in hematopoietic cancer
This eBook gauges shifting expectations for the gene therapy industry amid the COVID-related uncertainties and clinical setbacks of the past couple years. BioProcess Insider founding editor Dan Stanton reports on the January 2022 Phacilitate Advanced Therapies Week event, specifically a standing presentation on the 10 most important industry drivers from the past year. Since 2017, advancements in
Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development. Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate
Heart disease remains the leading cause of death in the world. One reason is that unlike other tissues, such as bone and skin, the heart has remarkably poor regenerative capability after an injury such as a heart attack. Scientists have therefore searched for heart cells that have regenerative properties. A new study by the Yoshinori
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency. “These results confirm the stability and good
A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides
Two patients represent longest-known CAR T cell response to date, providing insight into treatment effects and outcomes In the summer of 2010, Bill Ludwig and Doug Olson were battling an insidious blood cancer called chronic lymphocytic leukemia (CLL). They’d both received numerous treatments, and as remaining options became scarce, they volunteered to become the first
Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient, and predictable gene and cell therapies. Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and
