Within the framework of an international study, Lund University and Skåne University Hospital have started treating patients with hemophilia with gene therapy, something that began in January this year. The hope is that the new treatment will significantly simplify everyday life for those with severe hemophilia. Hemophilia is a genetic disease where the body does
This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more
A patient who received the world’s first transplant of cardiac muscle cells using artificially derived stem cells known as iPS cells this month is in stable condition, an Osaka University team said Jan. 27. After surgery, doctors closely monitored the patient, who had ischemic cardiomyopathy, a condition in which clotted arteries cause heart muscles to
Treatment uses person’s own stem cells instead of donor cells UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and
Establish an ATMP coordination body at EU/EEA level Ensure authorities in regions of treatment are compensated for costs of treating patients from other regions Encourage greater alignment within Europe on product value assessment activities The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector,
Researchers from KU Leuven and University Hospitals Leuven have managed to engineer living implants in the lab by mimicking how bone tissue is created in an embryo. The technology paves the way for bone-regenerating tissue implants created on an industrial scale using 3D bioprinting. The researchers expect the first living implants to be available to
Therapy shown to relieve extreme pain in mice. Now moving towards human trials. Human stem cells and “pain-killing neurons” have successfully relieved chronic pain in mice. The breakthrough method may be tested on human patients suffering untreatable pain within five years. Researchers at the University of Sydney have used human stem cells to make pain-killing
Identification of GPR108 as ‘lock’ for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types–the most commonly
University of Pittsburgh School of Medicine researchers have created a biodegradable nerve guide—a polymer tube—filled with growth-promoting protein that can regenerate long sections of damaged nerves, without the need for transplanting stem cells or a donor nerve. Source UPMC So far, the technology has been tested in monkeys, and the results of those experiments appeared
Blocking a protein makes cells shrink instead, causing fewer problems Techniques to genetically modify patient immune cells have revolutionized the fight against hard-to-treat cancers. But they can come with dangerous side effects. Now, researchers have found one reason why. A particularly messy form of cell death sparks severe inflammation in patients receiving CAR-T cell immunotherapy for blood
