Polymer-based nanocontainers could improve the delivery of gene therapies, a Johns Hopkins team reported. One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune
CAR-T cells are remarkably effective against blood cancers, but their effect can be transient as the cells become exhausted. Stanford researchers found a way to keep the cells effective in mice with human tumors. A new approach to programming cancer-fighting immune cells called CAR-T cells can prolong their activity and increase their effectiveness against human
The terms MSC and MSCs have become the preferred acronym to describe a cell and a cell population of multipotential stem/ progenitor cells commonly referred to as mesenchymal stem cells, multipotential stromal cells, mesenchymal stromal cells, and mesenchymal progenitor cells. The MSCs can differentiate to important lineages under defined conditions in vitro and in limited
Novartis’ big push into cell and gene therapy hasn’t been the smoothest of rides, with a data manipulation scandal hamstringing Zolgensma and manufacturing concerns hurting CAR-T cancer therapy Kymriah. Could a new European facility help turn around the latter’s production bottleneck? Novartis last week opened its newest cell and gene therapy facility in Stein, Switzerland, giving the
Source REUTERS Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world’s priciest medicines. The full scope of Novartis’ $500 million plan, revealed to Reuters in an interview
Study in Nature Sheds New Light on Heart Attack Treatment Controversy Stem cell therapy helps hearts recover from a heart attack, although not for the biological reasons originally proposed two decades ago that today are the basis of ongoing clinical trials. This is the conclusion of a Nov. 27 study in Nature that shows an entirely different
Source Trends in Molecular Medicine Mitochondria and lysosomes are pivotal organelles for cellular metabolism and also have important roles as signaling platforms. Many diseases are characterized by impaired mitochondrial or lysosomal function. These are often referred to as ‘mitochondrial diseases’, which have a primary cause in mitochondria, or as lysosomal storage diseases. Although each specific
High-throughput synthetic biology approach reveals hidden AAV features and could help fast-track future gene therapies Adeno-associated viruses (AAVs) have become the go-to vehicle for delivering therapeutic gene cargo to target tissues for the wave of gene therapies that are in development in academic and biotechnology laboratories. However, natural AAVs do not specifically target diseased cells
For the first time, The Arthroscopy Association of North America (AANA), the American Orthopaedic Society for Sports Medicine (AOSSM), the International Cartilage Repair Society (ICRS) and the ON Foundation have collaborated to form The Biologics Alliance (BA)™, an orthopaedic organization dedicated to providing one voice for all matters on musculoskeletal biologics and regenerative medicine. Recently, randomized clinical trials and clinical practice guidelines
Funds paved the way for rigorous tests of therapies, but unintentionally boosted a market for potentially dangerous fakes, says Jeanne F. Loring. Source Nature For the past dozen or so years, stem-cell researchers in California have been the envy of the world. In 2004, as a rebuke to the restrictions put in place in the
