The canonical Wnt signaling pathway participates in synapse function and stability, and previous research confirmed its impairment in Alzheimer's disease (AD).
Satellos Bioscience Inc. has reported results from preclinical studies in a disease model of Duchenne muscular dystrophy (DMD). The company's proprietary Myoregenx assay platform identified a protein kinase (K9) as a potential drug target to modulate polarity in muscle stem cells.
Researchers from Arkuda Therapeutics reported the discovery and preclinical evaluation of novel oxazoline enhancers of cellular progranulin (PGRN) secretion as candidates for cognition disorders therapy. Phenotypic screening of a diverse library of compounds was applied with the aim of identifying structures that effectively enhanced PGRN release. The lead hits were optimized and assessed in an immortalized murine microglial cell line (BV-2).
Egret Therapeutics, a portfolio company of Turret Capital Management LP, has announced FDA clearance of its IND application for EGT-101 for the treatment of delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage.
The suppression of the SYF2 factor could be a new therapeutic strategy for the treatment of the different types of amyotrophic lateral sclerosis (ALS). According to a study from the University of Southern California, SYF2 acts on the TDP-43 protein, improving the survival of motor neurons affected by this disease. “We wanted to find something that would improve neuron survival across many different iPSC lines for ALS,” Justin Ichida told BioWorld.
The lack of dystrophin causes Duchenne muscular dystrophy (DMD), a muscle-wasting disease that is often accompanied by heart failure due to cardiomyocyte death and fibrosis that can lead to death of the patient. It has been proven that telomere shortening is a hallmark of DMD cardiomyocytes. Researchers from the Stanford School of Medicine have recently investigated whether preventing telomere shortening and attrition may be a therapeutic approach in DMD.
Biogen Inc. and C4 Therapeutics Inc. have patented proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to interleukin-1 receptor-associated kinase 4 (IRAK-4)-targeting moiety through a linker acting as IRAK-4 degradation inducers reported to be useful for the treatment of cancer, inflammation, autoimmune and metabolic diseases, Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis and multiple sclerosis, among others.
Herophilus Inc. is conducting in vivo studies of lead candidate HRP-12975, a small-molecule therapy for Rett syndrome, with funding from the Rett Syndrome Research Trust. The company is generating efficacy and safety data with HRP-12975 using genetic mouse models of Rett syndrome.
Lapix Therapeutics Inc. has announced the successful outcome of its pre-IND meeting request with the FDA to achieve alignment on the company's IND-enabling plan for LPX-TI641, being developed for neuro-autoimmune indications such as multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSD), and myelin oligodendrocyte glycoprotein antibody disease (MOGAD).
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