Researchers from Beam Therapeutics Inc. presented the discovery and preclinical evaluation of an engineered stem cell antibody paired evasion (ESCAPE) strategy for antibody (Ab)-mediated autologous hematopoietic stem cell (HSC) therapy conditioning for the treatment of hemoglobinopathies.
The most common cause of anemia in chronically ill hospitalized patients is due to inflammatory anemia (IA) that is caused indirectly by diseases such as autoimmune, cancer, chronic kidney disease, congestive heart failure, or pulmonary disease. The precise and common etiology of these diseases involves hypercytokinemia that leads to excessive increases in hepcidin, a master regulator of iron homeostasis that blocks intestinal iron absorption when levels are too persistently high.
The primary reason for hospitalization of patients with sickle cell disease (SCD) is an acute systemic painful vaso-occlusive episode (VOE), which serves as an antecedent to acute chest syndrome (ACS). It has been previously demonstrated that P-selectin-dependent neutrophil-platelet aggregation and the complement pathway activation contribute to the vaso-occlusive pathophysiology in SCD.
Lunac Therapeutics Ltd. has synthesized coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of arthritis, Alzheimer's disease, disseminated intravascular coagulation, pulmonary embolism, myocardial infarction, diabetic retinopathy, stroke and deep venous thrombosis, among others.
CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.
SLN-140 (Silence Therapeutics plc) is a novel small interfering RNA (siRNA) targeting protein S for the treatment of hemophilia A (HA). Researchers from the University of Bern recently presented data from studies conducted in animal models of HA, performed to evaluate the safety and efficacy of SLN-140.
Vega Therapeutics Inc. has reported promising preclinical data on VGA-039, a first-in-class monoclonal antibody directed against human protein S (ProS) that inhibits ProS cofactor activity for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), thus enhancing thrombin generation by acting on both the initiation (TFPIα) and propagation (aPC) phases of coagulation for potential activity against various bleeding disorders.
Novelmed Therapeutics Inc. has reported topline results with its lead anti-properdin antibody NM-3086 in a rabbit model of paroxysmal nocturnal hemoglobinuria (PNH). NM-3086 is a potent, first-in-class, humanized monoclonal antibody that is highly selective for properdin of the alternative pathway (AP) without blocking the classical pathway (CP), which is critical for maintaining host defense against infections.
Researchers from BioCryst Pharmaceuticals Inc. have developed a series of plasma kallikrein inhibitors with potential for the treatment of hereditary angioedema (HAE).
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