Investigators from Cerevance Inc. have reported the discovery and preclinical characterization of a novel tandem pore domain halothane-inhibited K+ channel 1 (THIK-1) inhibitor, C-101248, being developed for the treatment of neuroinflammation in Alzheimer’s disease (AD). NETSseq and histological analysis revealed that THIK-1 expression was up-regulated in microglia from different cortical regions of AD donors compared with aged matched nondemented control brains.
Tuberous sclerosis complex (TSC) is a multisystemic disorder caused by loss-of-function mutations in the TSC1 or TSC2 genes. Its clinical phenotype is heterogenous, but most patients with TSC have epilepsy.
Qrons Inc. has established a collaboration with scientists at a public research university in Israel, by which Tellurium-based compounds in combination with Qrons' QS-200 product candidate and other configurations will be explored as treatment for diffused axonal injuries (concussions), which accounts for approximately 89% of traumatic brain injuries (TBIs).
The HIV regulatory protein transactivator of transcription (Tat) is a viral protein believed to play a key role in the neurotoxicity and cognitive impairment seen in HIV-associated neurocognitive disorders. Tat allosterically modulates dopamine (DA) reuptake through the human DA transporter (hDAT). In the current study, researchers from University of South Carolina and affiliated organizations aimed to assess the effects of the novel allosteric modulator of DAT, SRI-32743, on the Tat-DAT interaction.
Abata Therapeutics Inc. has selected its first development candidate, ABA-101, an autologous regulatory T cell (Treg) therapy for the treatment of progressive multiple sclerosis (MS). ABA-101 targets MS patients with nonrelapsing progressive disease who have a DRB1*15:01 genetic haplotype and for whom imaging evidence of ongoing inflammatory tissue injury has been observed.
The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).
Arthex Biotech SA has achieved key regulatory milestones in its program to develop ATX-01 in myotonic dystrophy type 1 (DM1). Having held a pre-IND meeting with the FDA last year and received scientific advice from the EMA, Arthex plans to file an IND application in the U.S. and a clinical trial application (CTA) in Europe next year.
Bristol Myers Squibb Co. has identified nonreceptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer's and Parkinson's disease, multiple sclerosis, neuromyelitis optica and optic neuritis.
Dravet syndrome is a type of congenital epilepsy caused by nonsense mutations in the SCN1A gene in about 20% of cases; SCN1A gene encodes the alpha subunit of the voltage-gated sodium channel Nav1.1. Spanish researchers and their collaborators have developed a novel murine model of Dravet syndrome; the model was developed by CRISPR/Cas9-generated A>T point mutation at nucleotide 1837 that converts Arg613 to a STOP codon, and which was introduced into exon 12 of the murine Scn1a gene using 129S1/SvImJ embryos.