The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency.
The European Commission is announcing the winners of a pan-European competition open to researchers addressing grand scientific and technological challenges that could change our future. The six preparatory actions now launched will have one year to prepare science and technology agendas in areas of strategic importance for Europe. These may contribute to defining and launching
A new study led by scientists in the Center for Cancer Research (CCR) at the National Cancer Institute (NCI) sheds light on one way tumors may continue to grow despite the presence of cancer-killing immune cells. The findings, published March 29, 2019, in Science, suggest a way to enhance the effectiveness of immunotherapies for cancer
Panelists discussed the outlook for cell and gene therapy investing at the Alliance for Regenerative Medicine’s Cell & Gene Therapy Investor Day in New York Thursday. As cell and gene therapy become a focal point of investment in the public markets and private equity alike, one of the biggest barriers to them is still manufacturing.
Exercise helps to prevent the degradation of cartilage caused by osteoarthritis, according to a new study from Queen Mary University of London. The researchers show for the first time how mechanical forces experienced by cells in joints during exercise prevent cartilage degradation by suppressing the action of inflammatory molecules which cause osteoarthritis. The study, published
Predictive program projected to save lives and reduce unnecessary surgeries Combining a wealth of information derived from previous studies with data from more than 500 patients, an international team led by researchers from Johns Hopkins has developed a computer-based set of rules that more accurately predicts when patients with a rare heart condition might benefit—or
Mouse Study Suggests Gut Sensors Monitor the Hydration Potential of Each Drink You Take Water bottles are everywhere these days, along with all kinds of advice about exactly how much water you should be drinking. But how does your brain actually know when you’ve had enough and can stop feeling thirsty? A new UC San
With modern, guideline-directed therapy,1 which includes angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers (ARBs), β-receptor blockers, mineralocorticoid receptor blockers, and angiotensin receptor neprilysin inhibitors, up to 40% of patients with dilated cardiomyopathy (heart failure with reduced ejection fraction) may experience a return of left ventricular ejection fraction (LVEF) and ventricular geometry to normal.2 This encouraging result
An important achievement in stem cell research was recognized in 2012, when the Nobel Prize in Physiology or Medicine was awarded to two scientists who transformed the field: Shinya Yamanaka and John Gurdon. Together, they received the award for “the discovery that mature cells can be reprogrammed to become pluripotent.” The impact of this breakthrough
As artificial intelligence continues to evolve, diagnosing disease faster and potentially with greater accuracy than physicians, some have suggested that technology may soon replace tasks that physicians currently perform. But a new study from the Google AI research group shows that physicians and algorithms working together are more effective than either alone. It’s one of
