Researchers from Splisense Ltd. and affiliated organizations recently reported preclinical data for SPL-84, an inhaled antisense oligonucleotide drug candidate being developed for the treatment of patients with cystic fibrosis carrying the 3849 +10 kb C-to-T (3849) mutation.
Alentis Therapeutics AG has obtained IND clearance from the FDA for ALE.P02, an anti-Claudin-1 (CLDN1) antibody-drug conjugate (ADC) with a tubulin inhibitor payload.
Ege University scientists and their collaborators have detailed the preclinical characterization of a novel DNA vaccine encoding the SRS13 protein being developed for the prevention of chronic toxoplasmosis.
The gene encoding methylthioadenosine phosphorylase (MTAP) is expressed in normal tissues but 10 to 15% of tumors present deletions of MTAP expression that lead to accumulation of its metabolite 5-methylthioadenosine (MTA). Previous research has shown that selective inhibition of protein arginine methyltransferase 5 (PRMT5) in the presence of MTA may be considered a potential therapeutic strategy for the treatment of MTAP-deleted cancer.
A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work. By using electron microscopy and three-dimensional reconstruction supported by AI tools, the researchers have revealed the neural wiring of the Drosophila melanogaster brain, a connectome of 140,000 neurons with 50 million synaptic connections. In the future, researchers could possibly use this map as an artificial in silico model to study the brain as a simulator through its connections, though a lot of work remains to be done for this.
Researchers at QPS Clinical Services GmbH and Universitat de Barcelona have divulged dual bifunctional epoxide hydrolase 2 (EPHX2; sEH) and glutaminyl-peptide cyclotransferase-like protein (QPCTL; IsoQC) inhibitors reported to be useful for the treatment of Alzheimer’s disease.
The University of Texas System has synthesized mitogen-activated protein kinase 1 (MAPK1; ERK2) inhibitors reported to be useful for the treatment of cancer.
Vesper Bio ApS has disclosed sortilin (NT3; Gp95) antagonists reported to be useful for the treatment of neurodegeneration, cancer, pain, diabetes, diabetic retinopathy, glaucoma, hearing loss and cardiovascular disorders, among others.