Arrakis Therapeutics Inc. has presented data on its RNA-targeted small-molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1). The company’s proprietary RNA‐specific chemical, biological and structural methods and RNA-directed medicinal chemistry enabled structure-based small-molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology.
Researchers from Northwestern University and Yale University have presented new preclinical data for KL-50, a small molecule under investigation for the treatment of glioblastoma. KL-50 was designed to fluoro-ethylate DNA bases, resulting in DNA inter-strand cross-links and subsequent DNA damage that triggers DNA mismatch repair (MMR)-independent apoptosis.
A ketone body, a molecule derived from the metabolism of acids to obtain energy when glucose is not available, could become an effective ally in treating Alzheimer’s or preventing the effects of aging on the brain. A group of scientists at the Buck Institute for Research on Aging have studied the role of β-hydroxybutyrate (βHB) as a signaling metabolite of misfolded proteins by interacting with them and altering their solubility, a mechanism that allows their elimination, as observed in preclinical models.
Researchers from Suzhou Siran Biotech Co. Ltd. presented the discovery and preclinical characterization of SA-1211, an N-acetylgalactosamine (GalNAc)-conjugated siRNA dimer targeting both hepatitis B virus (HBV) and PD-L1 gene expression, being developed as a potential new therapeutic candidate for the treatment of chronic hepatitis B (CHB).
Bladder cancer is a challenging disease that poses significant risks to patients, often leading to a grim prognosis. Recent research has brought attention to chaperonin-containing tailless complex polypeptide 1 subunit 3 (CCT3), identified as an oncogene in multiple tumor types. However, its specific role in bladder cancer remains largely unexplored.
Benobio Co. Ltd. has discovered new benzothiophene derivatives acting as bromodomain-containing protein 2 (BD1 domain) (Brd2 BD1) inhibitors reported to be useful for the treatment of cancer.
Elgen Therapeutics Inc. has patented new 4-chromanone derivative proteolysis targeting chimera (PROTAC) compounds comprising a von Hippel-Lindau disease tumor suppressor (VHL)-binding moiety coupled with a WD repeat-containing protein 5 (WDR5; BIG3)-targeting moiety through a linker.
Work at Tango Therapeutics Inc. has led to the identification of new ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
RSS
