A new method, based on gene editing with oligonucleotides and functional analyses, identifies which variants of DNA repair genes associated with Lynch syndrome are truly harmful and which are not. Scientists at The Netherlands Cancer Institute have developed this technique and classified these gene variants in both coding and noncoding regions, distinguishing those that are pathogenic from those that are benign. Read More
Metabolic dysfunction-associated steatotic liver disease (MASLD) covers a series of pathogenic conditions including steatosis, inflammation and fibrosis with limited therapeutic options to date. Recent findings have shown upregulation of hepatic murine double minute 2 (MDM2) in patients with MASLD. Additionally, genetic deletion of hepatic MDM2 and its pharmacological inhibition were seen to improve steatosis and fibrosis in MASLD murine models. Read More
Scribe Therapeutics Inc. is aiming to advance STX-1150, its lead product candidate for the treatment of hypercholesterolemia, into the clinic around the middle of this year. Hypercholesterolemia is a major driver of atherosclerotic cardiovascular disease. Read More
Insilico Medicine Cayman Topco and Hygtia Therapeutics Co. Ltd. have entered into an exclusive license and co-development collaboration to advance ISM-8969 worldwide. ISM-8969 is an orally available, brain penetrant NLRP3 inhibitor for CNS disorders. Read More
Orexin OX2 receptor agonists have demonstrated the ability to enhance wakefulness in rodent models, as well as in nonhuman primates and patients with narcolepsy and idiopathic hypersomnia. Based on recent findings, it has been hypothesized that they may also regulate cognition, mood and other neuropsychiatric functions. Furthermore, dysregulated orexin signaling has been reported in patients with major depressive disorder (MDD) with suicide attempts. Read More
Boundless Bio Inc. has obtained IND approval from the FDA for its novel kinesin oral degrader program, BBI-940. A first-in-human trial (KOMODO-1) for metastatic breast cancer is expected to begin in the first half of this year. Read More
Hanmi Holdings Co. Ltd. has patented proteolysis targeting chimeras (PROTACs) comprising a cereblon (CRBN)-binding moiety coupled to a histone acetyltransferase p300 (EP300)-targeting moiety through a linker acting as EP300 degradation inducers reported to be useful for the treatment of cancer. Read More
Gachon University and Jeonbuk National University Industry Foundation have disclosed proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN) E3 ubiquitin ligase-binding moiety coupled to a serine/threonine-protein kinase PAK4-targeting moiety through a linker potentially useful for the treatment of cancer. Read More
Novacell Technology Inc. has identified oligopeptides acting as N-formyl peptide receptor 2 (FPR2; FPRL1; LXA4) agonists described as potentially useful for the treatment of cancer and inflammatory disorders. Read More
Researchers at DEM Biopharma Inc. reported preclinical findings demonstrating the efficacy of DEM-301, a bifunctional antibody-drug conjugate (ADC) engineered to selectively recognize and eliminate tumor cells that express DEM-TXX. Read More
Nippon Zoki Pharmaceutical Co. Ltd. has discovered alkyne derivatives acting as GABA-A receptor subunit α1β2γ2 positive allosteric modulators. As such, they are believed to be useful for the treatment of sleep disorders. Read More
Multidrug-resistant (MDR) gram-positive bacteria can evade last-line therapies, such as vancomycin, daptomycin and linezolid, through the development of sophisticated resistance mechanisms that include inactivation of target enzymes, mutations in binding sites, alterations in membrane permeability and overexpression of efflux pumps. Read More
