Inmune Bio Inc., together with a collaborator at University of California, Irvine School of Medicine, has shown targeting soluble TNF (sTNF) using a dominant-negative TNF (DN-TNF) biologic significantly decreased muscle damage in a murine mdx model of Duchenne muscular dystrophy (DMD) and showed a statistically significant increase in muscle regeneration.
Amyotrophic lateral sclerosis (ALS) is the most frequent adult-onset motor neuron disease, and it is pathologically related with frontotemporal dementia (FTD). Genetic studies have identified C9ORF72 as a major genetic cause of ALS/FTD. Further genetic analyses and validation studies have identified some other genes associated with ALS risk, highlighting among them the NUP50 gene, which encodes nuclear pore complex protein Nup50.
Research shows that individuals with schizophrenia have abnormal gene expression patterns in their brains compared with people without the condition. In a study published in PLOS Biology on Jan. 24, 2023, the authors reported that many genes in brain tissue from the dorsolateral prefrontal cortex, a region involved in cognition, have expression patterns that follow a 12-hour cycle. The investigators found that these patterns were largely lost in people with schizophrenia.
A genome-wide association study (GWAS) from The University of Queensland has linked blood cell traits (BCTs) and neurological and psychiatric disorders (NPDs), providing a tool to improve patient treatments or repurposing different drugs. The researchers also found a cause-effect relationship between Parkinson's and platelet distribution width. In their study, published Jan. 25, 2023, in Cell Genomics, the scientists observed the genetic overlap between common NPDs and 29 BCTs, including functional genes, regulatory elements and new genetic correlations linked to hematological data and for these diseases.
West China Hospital, Sichuan University has described heteroatom-substituted aromatic compounds reported to be useful for the treatment of pain and as general anesthetics.
Neurogene Inc. has received FDA clearance of its IND application for NGN-401 for the treatment of Rett syndrome. The company plans to initiate a phase I/II trial in female pediatric patients with Rett syndrome this year.
Researchers at the Dana-Farber Cancer Institute have been able to identify proteins that were released from muscles during exercise in relatively small quantities. Using their method, the team was able to demonstrate that the neurotrophic factor prosaposin was produced during exercise. Prosaposin is “a well-known CNS neurotrophic factor, but has never been seen to come out of muscle or fat,” Bruce Spiegelman told BioWorld. Spiegelman is a researcher at the Dana-Farber Cancer Institute and Stanley J. Korsmeyer Professor of Cell Biology and Medicine at Harvard Medical School.
A new epigenetic editing technique has restored the functionality of neurons in Rett syndrome (RTT), a rare neurodevelopmental disease associated with a mutation of the MECP2 gene on the X chromosome. The origin of this disorder is a heterozygous mutation of the MECP2 (methyl CpG-binding protein 2) gene. Homozygosity is lethal and as it is associated with the X chromosome; Rett mostly affects girls, inhibiting the development of their motor skills and communication.
Gilgamesh Pharmaceuticals Inc. has identified arylcyclo-hexylamine derivatives targeting N-methyl-D-aspartate receptor (NMDAR) and/or serotonin transporter (SERT) reported to be useful for the treatment of substance abuse and dependence, depression and mood and anxiety disorders.