A genome-wide association study (GWAS) from The University of Queensland has linked blood cell traits (BCTs) and neurological and psychiatric disorders (NPDs), providing a tool to improve patient treatments or repurposing different drugs. The researchers also found a cause-effect relationship between Parkinson's and platelet distribution width. In their study, published Jan. 25, 2023, in Cell Genomics, the scientists observed the genetic overlap between common NPDs and 29 BCTs, including functional genes, regulatory elements and new genetic correlations linked to hematological data and for these diseases.
Mabwell Therapeutics Inc., a wholly owned subsidiary of Mabwell (Shanghai) Bioscience Co. Ltd., has entered into a license agreement with Disc Medicine Inc. for 9MW3011 (MWTX-001, MWTX-002 & MWTX-003) for hematologic diseases.
By combining drug sensitivity with genomic profiling of tumor cells, a study from St. Jude Children's Research Hospital with more than 800 patients has shown a wide diversity in drug sensitivity for pediatric acute lymphoblastic leukemia (ALL) and defined six patterns of response to treatment. “This work provides a framework for ‘functional precision medicine’,” corresponding author Jun Yang, vice chair of the Department of Pharmacy and Pharmaceutical Sciences at St. Jude Children's Research Hospital, told BioWorld.
Scientists based at Beijing Institute of Radiation Medicine have reported preclinical evaluation of a novel recombinant activated human factor VII (rFVIIa), GEN-0828, being developed as potential candidate for the treatment of hemophilia and trauma hemorrhage. GEN-0828 is rFVIIa that was mainly produced from Chinese hamster ovary (CHO) cells.
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.