Bioray Pharmaceutical Co. Ltd. has announced clinical trial clearance in China by the National Medical Products Administration (NMPA) for BR-111 for injection for the treatment of ROR1-positive hematological malignancies and solid tumors.
Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients developing hypertrophic cardiomyopathy.
Xaicure Pharmaceuticals (Suzhou) Co. Ltd. has described histone acetyltransferase KAT6A (monocytic leukemia zinc finger protein; MOZ; MYST-3) and/or histone acetyltransferase KAT6B (MOZ2; MYST-4) inhibitors reported to be useful for the treatment of cancer.
Maze Therapeutics Inc. has divulged apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of chronic kidney disease, focal segmental glomerulosclerosis, diabetic retinopathy, diabetic HIV-associated nephropathy, lupus nephritis, pre-eclampsia and sepsis.
Mindrank AI Co. Ltd. and Mindrank Therapeutics (Suzhou) New Drug Research & Development Co. Ltd. have identified molecular glue degraders comprising cereblon (CRBN) ligands acting as eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1) degradation inducers reported to be useful for the treatment of cancer.
Pharmaengine Inc. and Sentinel Oncology Ltd. have synthesized Wee1-like protein kinase (Wee1) inhibitors and proteolysis targeting chimera (PROTAC) compounds reported to be useful for the treatment of cancer.
Hanyang University and Korea Institute of Science and Technology have disclosed HER2 (erbB2) inhibitors reported to be useful for the treatment of cancer.
Early dysfunction of the endothelial/blood-brain barrier (BBB) is involved in the pathogenesis of cerebral small vessel disease (SVD), which is a contributor to about 50% of dementias. Sphingosine-1-phosphate (S1P) is a sphingolipid that regulates the BBB integrity when bound to its receptor S1P1 on endothelial cells.
Cyclin-dependent kinases 4 and 6 (CDK4/6) are crucial cell cycle regulators and have become significant targets in breast cancer therapy. Current CDK4/6 inhibitors, while effective, often come with adverse effects and resistance issues.
Leukemic stem cells (LSCs) and stemness signatures contribute to minimal residual disease in patients with acute myeloid leukemia (AML), which is associated with an increased risk of relapse. The presence of LSCs predicts treatment success and, therefore, eliminating LSCs has been proposed as a promising strategy to avoid relapses.