Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels. Read More
Researchers from Liaoning Jinqiu Hospital and affiliated organizations explored potential new genetic targets in heart failure and breast cancer through combining genetic inference and single-cell expression analysis. Genome-wide association study (GWAS) identified multiple genetic variants that were causally related in heart failure and breast cancer. Read More
Werewolf Therapeutics Inc. has expanded its pipeline with the addition of a novel IL-10 Indukine development candidate, WTX-921, for inflammatory bowel disease (IBD) and potentially other inflammatory diseases. Read More
Endevica Bio Inc. has announced the spin-off of a newly created company, Abisati LLC, to develop an oral melanocortin MC4 receptor agonist, 710GO, as a weight loss therapy. Read More
Researchers from Astrazeneca plc recently reported preclinical data for AZD-3470, a second-generation MTA-cooperative PRMT5 inhibitor currently in early clinical development for the treatment of patients with MTAP-deficient solid tumors (NCT06130553) and hematological cancers (NCT06137144). Read More
Regeneron Pharmaceuticals Inc. has patented rapamycin analogues acting as peptidyl-prolyl cis-trans isomerase FKBP1A (FKBP12; Rotamase) ligands reported to be useful for the treatment of Alzheimer’s disease, graft-vs.-host disease, cancer, sarcopenia and more. Read More
Researchers from Abbisko Therapeutics Inc. presented the preclinical characterization of ABSK-141, a potent bioavailable small-molecule KRAS G12D inhibitor. Read More
PARP1 is critical for repairing DNA single-strand breaks. First-generation PARP1/2 inhibitors have proven effective in the treatment of tumors with mutations in the essential homologous recombination repair (HR) genes including BRCA mutations. However, hematological toxicity associated with PARP2 emphasizes the need to find second-generation compounds with better safety profiles. Read More
Mitochondria In Motion Inc. has identified new N-(trans-4-hydroxycyclohexyl)-6-phenylhexanamide derivatives acting as mitofusin activators and reported to be useful for the treatment of Alzheimer’s, Parkinson’s and Huntington’s disease, cancer, amyotrophic lateral sclerosis, stroke, mitochondrial myopathy and Charcot-Marie-Tooth disease, among others. Read More
Work at Acelink Therapeutics Inc. has led to the identification of Kelch-like ECH-associated protein 1 (Keap1)/Nrf2 interaction inhibitors reported to be useful for the treatment of cancer, lung, mitochondrial and sickle cell diseases, cardiovascular, inflammatory, neurological and renal disorders. Read More
The c-MYB oncogene plays a key role in hematopoietic cell differentiation and proliferation. Genetic abnormalities and dysregulation of MYB have been found in several cancers, including adenoid cystic carcinoma (ACC) (80% of cases), making it an attractive druggable target for ACC treatment. Read More
The PLN-R14del mutation is often tied to dilated or arrhythmogenic cardiomyopathy, which can progress to end-stage heart failure. Cardiac phospholamban (PLN) plays a crucial role in calcium modulation in cardiomyocytes, which is disrupted by the PLN-R14del mutation. Read More
Scientists from INSERM and affiliated organizations presented findings from their work that aimed to investigate the involvement of heat shock protein 27 (HSP27) in the activation and pathological accumulation of parietal epithelial cells (PEC) during crescentic glomerulonephritis (CrGN). Read More
Guangdong HEC Pharmaceutical Co. Ltd. has reported compounds acting as GTPase KRAS and mutant inhibitors and thus reported to be useful for the treatment of cancer. Read More
