The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells. Read More
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA) patients and collagen-induced arthritis (CIA) rats and mice, where it drove osteoclast differentiation. Read More
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms. Read More
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH) and obesity. Read More
Peptidream Inc. has submitted IND applications to the FDA to initiate phase I studies of [225Ac]PD-32766, as a potential radiotherapeutic, and [64Cu]PD-32766, as the radiodiagnostic pair, for patients with clear cell renal cell carcinoma (ccRCC). Read More
Toolgen Inc. has entered into a strategic cross-license agreement with Geneditbio Ltd. to jointly advance the development of next-generation in vivo genome-editing therapeutics. Read More
Captor Therapeutics Inc. has disclosed molecular glue degraders comprising a cereblon (CRBN) binding moiety acting as serine/threonine-protein kinase Nek7 degradation inducers reported to be useful for the treatment of amyotrophic lateral sclerosis, Still’s disease, Behçet’s disease, familial Mediterranean fever, Alzheimer’s disease, pulmonary arterial hypertension and more. Read More
Gemma Biotherapeutics Inc.’s GB-221, a novel gene therapy for spinal muscular atrophy type 1 (SMA1), has received clinical trial clearance from ANVISA, Brazil’s health regulatory agency. Read More
Fibrolamellar carcinoma (FLC) is a rare and lethal cancer accounting for 1% of liver cancers affecting young, otherwise healthy patients. The primary driver of the vast majority of human FLCs is a fusion between the heat shock protein 40 (DNAJB1) and the catalytic subunit α of protein kinase A (PRKACA). Read More
Nanjing Synnocare Pharmaceutical Technology Co. Ltd. has synthesized prodrugs of kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer. Read More
Université de Montréal has identified compounds acting as proto-oncogene tyrosine-protein kinase Src (SRC; SRC1) and/or tyrosine-protein kinase Yes (YES1) inhibitors reported to be useful for the treatment of cancer, fibrosis and liver diseases. Read More
Vaxcellbio Co. Ltd. has signed a research agreement with the Korea Drug Development Fund (KDDF) to pursue the discovery of next-generation targeted anticancer lead compounds based on degradation of the molecular motor protein MYO1D (myosin 1D). Read More
Shanghai Apeiron Biotechnology Co. Ltd. has divulged protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of brain metastatic cancer. Read More
Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Suzhou Vigonvita Life Sciences Co. Ltd. have described NMDA receptor antagonists and/or monoamine transporter inhibitors reported to be useful for the treatment of neurological disorders. Read More
Bakuchiol, a phenolic meroterpenoid from the seeds of Psoralea corylifolia L., has shown promise as an antitumor drug, so researchers at Bengbu Medical University synthesized novel derivatives of bakuchiol bearing modifications on the aromatic ring. Read More
