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. Read More
Italian researchers have presented a case study of pediatric advanced sleep-wake phase syndrome in a 4-year-old girl. Autosomal dominant forms of this syndrome due to loss-of-function mutations in the gene coding casein kinase I isoform delta (CSNK1D) have previously been described. Read More
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR. Read More
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease. Read More
Artelo Biosciences Inc. has presented data on its fatty acid-binding protein 5 (FABP5) inhibitor ART-26.12 from testing of its efficacy in preclinical models of psoriasis. Read More
Epivario Inc. has been awarded a small business innovation research (SBIR) grant of $275,000 from the National Institute on Drug Abuse (NIDA) to advance research toward the development of a novel epigenetic regulator as a treatment for opioid use disorder. Read More
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. Read More
Shattuck Labs Inc. has announced a strategic shift to focus on SL-325, a death receptor 3 (DR3) antagonist antibody for the treatment of inflammatory bowel disease (IBD) and other inflammatory autoimmune diseases. Read More
Darier disease (DD) is a rare cutaneous disease caused by heterozygous mutations in the ATP2A2 gene, which encodes sarco/endoplasmic reticulum calcium ATPase isoform 2 (SERCA2). Read More
The University of Texas System has synthesized mitogen-activated protein kinase 1 (MAPK1; ERK2) inhibitors reported to be useful for the treatment of cancer. Read More
Phosphodiesterase 4 (PDE4) is an enzyme known to play a role in inflammatory responses, and it has been pinpointed as an interesting therapeutic target for diseases like atopic dermatitis (AD), but limited efficacy and side effects have prevented the approval of oral formulations. Read More
The Methodist Hospital System has identified triphenyl phosphonium compounds targeting mitochondria reported to be useful for the treatment of cancer. Read More
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. Read More
Loqus23 Therapeutics Ltd. has closed a £35 million (US$43 million) series A financing, with the aim of supporting its work developing small-molecule somatic expansion inhibitors for the treatment of Huntington’s disease and other triplet repeat disorders. Read More
