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...
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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...
LOS ANGELES, Calif. – An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, or LAD-I, in an international clinical trial co-led by UCLA. LAD-I is a genetic condition that affects approximately one in...
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...
Boston, Massachusetts – Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological function and ultimately leading to early death. Researchers from Massachusetts General Hospital, a founding member of the Mass General Brigham healthcare system, Boston Children’s Hospital, and collaborators...
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...
HONG KONG, BEIJING and BOSTON — GenEditBio Limited (“GenEditBio”), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic solutions through the discovery of novel and precise Cas nucleases and the development of safe and efficient cargo delivery platforms, today announced a key milestone in the development of its pipeline: First patient has been...
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...