Mount Sinai scientists have become the first to report a potentially serious side effect related to a new form of immunotherapy known as CAR-T cell therapy, which was recently approved for the treatment of multiple myeloma. Their findings were published as a case study in Nature Medicine in December. Multiple myeloma is a complex and incurable type
Broad and robust transgene expression across brain regions of marmoset is shown after systemic delivery with engineered capsid. Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington’s disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into
Clinical trial planning underway to test the widely used diabetes drug metformin as a preventive treatment for the blinding eye disease Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute (NEI), part of
The CompacT SelecT (CTST) platform is a modular robotic system that integrates a full range of cell culture proceduresunder sterile conditions that mimic the manual cell culture process. These procedures include automated handling of different cell culture vessels, pipetting large and small volumes at adjustable speeds, cell counting, cell viability analysis, cell density assessment, microscopic
Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.” That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and
About 20,000 people in the U.S. live with hemophilia A. It’s a rare X-linked genetic disorder that affects predominantly males and causes their blood to clot poorly when healing wounds. For some, routine daily activities can turn into painful medical emergencies to stop internal bleeding, all because of changes in a single gene that disables
The first tests on eight children show favorable preliminary results, with an adjustment of motor and cognitive skills and improvements in some brain and joint parameters Gene therapy takes steps forward in the fight against rare diseases. Eight children with severe Hurler’s syndrome, a disease associated with reduced life expectancy, benefited from this therapy. The
Chimeric antigen receptor T-cell (CAR T) therapy has revolutionized the field of malignant hematology. CD19-targeted CAR T-cell therapy has resulted in effective, often durable, responses for chemotherapy-refractory B-cell lymphoma and B-acute lymphoblastic leukemia (B-ALL) in prospective clinical trials. CAR T cells against B-cell maturation antigen have also shown overall response rates in the range of
The therapeutic benefit to patients receiving mesenchymal stromal cell (MSC) therapy is not because the injected cells remain viable, but because of cell death, researchers at the Monash Biomedicine Discovery Institute (BDI) have found. In recent years, significant efforts have been made to develop stem cell-based therapies for difficult-to-treat diseases. MSC therapy is regenerative cell-based
The new EFPIA Pipeline Review reveals that infectious, inflammatory, cancer, Alzheimer’s and rare disesases areas are among the top priorities of researchers running clinical trials today to investigate new medicines and vaccines. The comprehensive report found that in 2020 alone, around 5,000 clinical trials were launched – despite the disruption caused by the COVID-19 pandemic. In fact, the volume of trials has
