Toronto, Canada – When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids) in March 2022, the course of his condition was dramatically altered. Michael has spastic paraplegia type 50 (SPG50), an “ultra-rare” progressive neurodegenerative disorder that causes developmental delays,...
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Sidney, Australia – In a worldwide first-of-its-kind study published in the prestigious journal Nature Communications this week, a team of scientists from Children’s Medical Research Institute (CMRI) tested novel gene therapies in a whole human liver, with the goal of developing more effective treatments for life-threatening inherited diseases. Gene therapy...
Boston, Mass. – A new study co-led by investigators from Mass Eye and Ear, a member of Mass General Brigham, demonstrated the effectiveness of a gene therapy towards restoring hearing function for children suffering from hereditary deafness. In a trial of six children taking place at the Eye & ENT...
PHILADELPHIA, PA— The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it. Some patients even experienced a 10,000-fold improvement in their vision after...
University of Florida researchers have come up with a new gene therapy method to disrupt cancer growth by using a synthetic protein to induce blood clotting that cuts off a tumor’s blood and nutrient supply. In mice implanted with human colorectal cancer cells, tumor volume decreased 53 percent and cancer...
Bethesda, MD – In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed...
Auckland, New Zealand – A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful...
Stamford, Conn. — GeneDx Holdings Corp. (Nasdaq: WGS) (“GeneDx” or the “Company”), a leader in delivering improved health outcomes through genomic and clinical insights, reported its financial results for the fourth quarter and full year ended December 31, 2022. “Entering 2023 with a new corporate strategy and a focus on...
MILAN, Italy and NEW YORK, NY — Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology (I/O) company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors, today announced that: We have successfully dosed the first of three patients in Cohort...
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced the latest data from three pivotal Phase III studies of Venclexta® (venetoclax) – CLL14, MURANO and VIALE-A – to be presented at the European Hematology Association Virtual Congress, June 9-17 (EHA2021). Long-term follow-up data from the CLL14...