Collagen is the most abundant protein in the human body, which would seem to make it an unlikely source for an immunotherapy target. But it is where researchers from Immatics Biotechnologies GmbH and the University of Pennsylvania have found a target that was expressed on stromal cells in a number of different solid tumors, but very rare in normal tissues. Read More
Glutaric acidemia type I (GA1) is caused by severe deficiency in enzyme glutaryl-CoA dehydrogenase activity, which results in an impairment on lysine catabolism. These patients have accumulation of glutaric acid and glutaryl carnitine, with loss of striatal neurons and atrophy of the frontotemporal cortex. The aim of investigators was to investigate plasma markers of neurodegeneration and inflammation, such as brain-derived neurotrophic factor (BDNF) and cathepsin D in patients (N = 6) with GA1 and matched healthy controls. Read More
As patients with Pompe disease age, they develop neurocognitive issues and impairment in their cerebral white matter, as shown in MRI scanning. To date, there are no biomarkers that reflect this disease progression in the brain. The usefulness of neurofilament light chain (NfL) was investigated for this purpose. Read More
Jiangsu Simcere Pharmaceutical Co. Ltd. has divulged bicyclic mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer. Read More
Nanchang Helioeast Technology Co. Ltd. has identified new oxa-azaspiro compounds acting as lysine-specific histone demethylase 1A (KDM1A; LSD1) inhibitors reported to be useful for the treatment of acute myeloid leukemia. Read More
Jacobio Pharmaceuticals Co. Ltd. has synthesized new protein mono-ADP-ribosyltransferase TIPARP (PARP-7; ARTD14) inhibitors reported to be useful for the treatment of cancer. Read More
To target mutant RAS in the GTP-bound RAS(ON) state for cancer treatment, Revolution Medicines Inc. has developed a platform in which binding and inhibition occur through small molecule-driven formation of a high-affinity ternary complex (tri-complex) between the target protein, a small molecule and a chaperone such as cyclophilin A. Read More
Suzhou Alphama Biotechnology Co. Ltd. has described son of sevenless homolog 1 (SOS1) inhibitors reported to be useful for the treatment of cancer. Read More
Doxorubicin is widely used as an anticancer agent, but it is associated with cardiotoxicity. Follistatin like 3 (FSTL3) is known to be involved in the regulation of cardiac hypertrophy and heart failure. Read More
Researchers from Arbutus Biopharma Corp. presented preclinical data for the novel hepatitis B virus (HBV) capsid assembly inhibitor, AB-836, being developed for the treatment of chronic HBV infection. Read More
Bridge Biotherapeutics Inc. recently reported data for BBT-209, an endogenous G protein-coupled receptor 19 (GPCR19) agonist under development for the treatment of idiopathic pulmonary fibrosis (IPF).
