Nuage Therapeutics SL has secured €2.7 million (US$3.1 million) in funding from the Spanish State Research Agency (AEI) of the Ministry of Science, Innovation and Universities that will support a research project focused on developing a new drug against the most common subtype of small-cell lung cancer (SCLC-A), in collaboration with the Spanish National Cancer Research Centre (CNIO), the European Molecular Biology Laboratory (EMBL), and the Vall d’Hebron Institute of Oncology (VHIO).
Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, which is then converted by ENPP1 into AMP and pyrophosphate (PPi). Because PPi is a potent inhibitor of calcification, reduced systemic PPi production is a key driver of PXE. University of Pennsylvania investigators and collaborators proposed applying liver-targeted variant correction via genome editing as a single-intervention therapeutic approach for PXE, leading to subsequent restoration of systemic PPi.
“I love the idea of ‘micropublications’ (preparing one now),” the neurobiologist Oded Rechavi commented on social media in July. The term clearly suggests a short article, and although the publishing model has been around for more than a decade, not everyone is familiar with this type of scientific communication. What are they? What’s their impact? Rechavi, a professor at the School of Biochemistry, Neurobiology and Biophysics at Tel Aviv University, was pointing to an emerging discussion among scientists, the search for alternative formats for their work.
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has described Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of colorectal cancer.
Genescience Pharmaceuticals Co. Ltd. has divulged thyroid-stimulating hormone (thyrotropin) receptor (TSHR) antagonists reported to be useful for the treatment of hyperthyroidism, Grave’s disease and thyroid-associated ophthalmopathy (Grave’s ophthalmopathy).
Di’ao Group Chengdu Pharmaceutical Co. Ltd. has identified glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, dementia, obesity, metabolic syndrome, metabolic dysfunction-associated steatotic liver disease (MASLD), dyslipidemia, stroke and cardiovascular disorders, among others.
Cornell University and The General Hospital Corp. (Massachusetts General Hospital) have synthesized conjugates comprising a targeting moiety covalently linked to a radiolabeled metal chelator through a linker acting as positron emission tomography (PET) or single-photon emission computerized tomography (SPECT) imaging agents reported to be useful for diagnosis and treatment of cancer.
Shenzhen Icarbonx Intelligent Peptide Pharmaceutical Technology Co. Ltd. has disclosed peptides targeting IQ motif and SEC7 domain-containing protein 1 (IQSEC1; BRAG2) reported to be useful for the treatment of stroke, major depression, substance abuse and dependence, and Alzheimer’s disease.
The canonical G protein-coupled receptors (GPCRs) cannabinoid CB1 (CB1R) and CB2 (CB2R) receptors are the most studied in the endocannabinoid system. CB1R is widely distributed throughout the CNS, but is also present on peripheral tissues.
HER2-targeted antibody-drug conjugates (ADCs) have significantly improved cancer therapy in recent decades. However, ADCs can cause toxicity and resistance, while T-cell engagers (TCEs) show promising antitumor activity in solid tumors but are limited by substantial adverse effects.