At the American Heart Association’s Scientific Sessions, E-Therapeutics plc presented preclinical data for ETX-258, a Galomic siRNA being developed for the treatment of heart failure.
The Wiskott-Aldrich syndrome protein (WASp) is expressed exclusively in hematopoietic cells and presents either an autoinhibited or activated conformation. EG-011 is a first-in-class activator of the autoinhibited form of WASp designed for the treatment of hematological cancers.
Researchers from Ohio State University hypothesized that 12,13-diHOME may act by inhibiting the proarrhythmic molecule Ca2+/calmodulin-dependent kinase II (CaMKII).
Voyager Therapeutics Inc. has selected a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an IND application with the FDA and a clinical trial application (CTA) with Health Canada for VY-1706 in 2026.
As a consequence of chronic liver disease progression, severe cirrhosis, decompensation and fibrosis culminates in end-stage liver disease (ESLD). There are no treatment options approved for ESLD, but, among others, regenerative therapies with autologous, nonengineered, pro-regenerative macrophages have shown good tolerability and improved transplant-free survival in the clinical setting.
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.