Chinese researchers have published data regarding phosphatidylserine (PS) derivatives acting as neuroprotective compounds for Alzheimer’s disease (AD) therapy.

A modified version of CRISPR-Cas9 has enabled, for the first time, the efficient integration of a large transgene capable of inactivating entire chromosomes into one of the three copies of chromosome 21 in Down syndrome-derived cells. The goal is to silence the extra copy to limit the gene-dosage imbalance that drives many features of trisomy 21. Researchers at Beth Israel Deaconess Medical Center turned to XIST, the long noncoding RNA responsible for the natural silencing of the X chromosome in females. Using this strategy, they achieved integration efficiencies of 20% to 40% and a partial reduction in the overexpression of chromosome 21 genes.

Researchers from the Government College University Faisalabad reported the discovery and preclinical characterization of IMS-48, a benzimidazole analogue designed to inhibit both acetylcholinesterase (AChE) and butyrylcholinesterase (BChE).

Genes that are switched on or off in the human brain differ between men and women. Moreover, these differences are not uniform. They vary across cortical regions and cell types. Scientists at the National Institute of Mental Health (NIMH) and the National Institute on Aging (NIA) used single-cell sequencing and unveiled distinct gene expression patterns regulated by hormones and sex chromosomes. This detailed map of the brain’s molecular biology shows how women and men switch on and off more than 3,000 brain genes differently and expands the catalogue of X chromosome genes that escape inactivation.

Naturecell Co. Ltd.’s U.S. subsidiary, Naturecell America, has received IND clearance from the FDA for Astrostem-AU, an autologous adipose-derived mesenchymal stem cell therapy for adults with autism spectrum disorder (ASD).

Transient receptor potential melastatin 3 (TRPM3) is a calcium-permeable TRP channel that is highly expressed in somatosensory neurons, including nociceptors of rodents and humans. Its activation triggers acute pain, making drugs that target TRPM3 a potential approach to alleviate pain. Biohaven Ltd.’s BHV-2100 is the only selective TRPM3 antagonist in clinical development. The company recently disclosed the work leading to its discovery and early development, as well as the chemical structure.

Mabwell (Shanghai) Bioscience Co. Ltd. has announced that SST-001 (18F-FD4), an α-synuclein-targeted PET tracer developed by Mabwell’s incubated company Synusight Biotech, has received clinical trial clearance from China’s National Medical Products Administration (NMPA). The planned phase I trial will enroll healthy volunteers, patients with multiple system atrophy (MSA), and patients with Parkinson’s disease.

Investigators have shown that REED1 expression was elevated during stress, which in turn suppressed hippocampal synaptogenesis and induced major depressive disorder (MDD)-like behavior through mTORC1 signaling. The impact of REDD1 modulation was investigated in the preclinical setting for MDD.

Quiver Bioscience Inc. has received a strategic investment from the Porta family office (Argentina) to advance the company’s antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome. The investment will fund preclinical activities, including safety studies for candidate selection as the program advances toward clinical development.