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
Credit: Chinese Medical JournalDaishi Tian from Huazhong University of Science and Technology Autoimmune disease (AID) refers to the condition in which the immune system identifies the body’s own cells and tissues as foreign, resulting in systemic inflammation. The immune system’s self-attack via autoreactive B and T immune cells and autoantibodies—antibodies against body’s own proteins—may present
The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. It is caused by a
A new drug has been shown to increase healing and regeneration of the liver after major surgery, according to a study published in the scientific journal Cell. Researchers hope that this could lead to more surgical options for patients diagnosed with advanced liver tumors and liver failure. “This research is significant because this is the first
Critical limb ischemia is a condition in which the main blood vessels supplying blood to the legs are blocked, causing blood flow to gradually decrease as atherosclerosis progresses in the peripheral arteries. It is a severe form of peripheral artery disease that causes progressive closure of arteries in the lower extremity, leading to the necrosis
A collaborative project to bring the promise of cell therapy to patients with a deadly form of brain cancer has shown dramatic results among the first patients to receive the novel treatment. In a paper published today in The New England Journal of Medicine, researchers from the Mass General Cancer Center, a member of the Mass
International research team develops, validates approach for assessing and responding to elevated risk As new cancer treatments become available, some of the most important ongoing research must look at ways to optimize those new approaches so that more patients can benefit from groundbreaking therapies. In work newly published in Blood Cancer Discovery, a journal of the American Association
