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.

Does cancer cause autoimmune disease or is it the other way around? In looking at the question of which comes first, the chicken or the egg, researchers at the Garvan Institute of Medical Research in Australia found that a genetic mutation that alters immune cells in leukemia is behind certain autoimmune disorders.