Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
Homology Medicines Inc. has reported data on an adeno-associated viral (AAV) vector-based therapy, HMI-104, an AAV treatment intended to induce hepatic expression of a complement C5 monoclonal antibody, named as C5mAb, for the potential treatment of paroxysmal nocturnal hemoglobinuria (PNH) as well as other complement-driven pathologies. C5mAb is thought to bind to C5 and inhibit the C5-mediated hemolysis observed in PNH.
A proof of concept of ex vivo genetic modification of cells from patients and their transplantation in mice has demonstrated, for the first time, the therapeutic possibilities of prime editing in sickle cell disease (SCD).
Blood clots can lead to life-threatening conditions such as deep vein thrombosis, heart attack, pulmonary embolism and stroke. Blood thinners are essential in the treatment and prevention of blood clots but carry a significant risk of bleeding as they target enzymes essential for blood clotting. Researchers at the University of British Columbia (UBC) and the University of Michigan have developed a new class of blood thinners that can specifically target clots without increasing the risk of bleeding.
Researchers have gained new insights into physiological mechanisms that protect against blood clotting in immobilized individuals by studying animals that stay immobile for a good chunk of the year at a time: hibernating bears. “As a clinician, if you think about immobility, you always think about thrombosis,” Tobias Petzold told BioWorld. But his team’s work, which was published in the April 13, 2023, issue of Science, demonstrated that “immobility can trigger antithrombotic mechanisms.”
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
Janssen Pharmaceutica NV has disclosed 5-oxo-1,2,3,5,8,8a-hexahydroindolizine-3-carboxamide derivatives acting as coagulation factor XIa and plasma kallikrein (KLKB1) inhibitors reported to be useful for the treatment of thromboembolism, diabetes, diabetic retinopathy, septic shock, hereditary angioedema, arthritis, nephropathy and inflammatory disorders, among others.
Two molecules that affected the cell cycle only of acute myeloid leukemia (AML) cells could be used as a clinical strategy against this pathology. Scientists at Memorial Sloan Kettering Cancer Center and Harvard University have discovered that DEG-35 and DEG-77 arrested the cell cycle and promoted cell differentiation and apoptosis in these cells.
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