Alchemedicine Inc. has divulged aldehyde dehydrogenase 2 (ALDH2) activators reported to be useful for the treatment of pain, cancer, ischemia-reperfusion injury, nonalcoholic fatty liver disease, peripheral arterial disease, Fanconi anemia, Parkinson’s and Alzheimer’s disease, among others.
The gene for Huntington’s disease “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience. Then, “it took 25 years for the first trial targeting the Huntington gene.”
Mindset Pharma Inc. has patented new indoline derivatives acting as 5-HT1A receptor agonists and thus reported to be useful for the treatment of neurological and psychiatric disorders.
Sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers have been reported in a Vertex Pharmaceuticals Inc. patent as potentially useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
Previous research found that transplanting human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NS/PCs) promoted motor functional recovery in animal models of spinal cord injury (SCI). hiPSC-NS/PC transplantation has been recently evaluated for subacute SCI in a first clinical trial. However, animal studies revealed that the effectiveness of NS/PC transplantation varies depending on the state of the injured spinal microenvironment, with reduced therapeutic effects in severe models. Hepatocyte growth factor (HGF) is a potent growth factor that promotes tissue regeneration through the MET receptor and constitutes an interesting candidate to enhance the efficacy of NS/PC transplantation.
Researchers from the University of Michigan and affiliated organizations presented data from a study that identified a link between a new neurodevelopmental disorder and biallelic variants in the EPB41L3 gene, which encodes band 4.1-like protein 3 that plays important roles in neuronal development, myelination and cytoskeletal organization.
Pasithea Therapeutics Corp. has selected a lead therapeutic candidate for its PAS-003 program, a proprietary humanized monoclonal antibody that targets α5β1 integrin, a protein overexpressed in both humans and mice with amyotrophic lateral sclerosis (ALS).
Prism Biolab Co. Ltd. has identified new 7-membered ring-fused compounds acting as Notch signaling inhibitors and reported to be potentially useful for the treatment of neuronal injury and neurodegenerative diseases.
Kynexis BV has launched with €57 million in series A financing with the aim of using its experience in psychiatry, neurology, and drug discovery and development to advance therapeutics for brain diseases.
At the recent American Society of Human Genetics meeting, researchers from Stanford University reported clinical and functional evidence of the involvement of ARHGAP1, a Rho GTPase-activating protein (GAP) gene, in a patient exhibiting a syndromic neurodevelopmental disorder.
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