Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity.
Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute SCI, a single injection of AMFX-200 into the spinal cord enabled motor neurons from the brain to regrow past the injury site.
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload.
Illimis Therapeutics Inc. raised ₩58 billion (US$42 million) in a series B financing round. The funds will support development of ILM-01, its lead bispecific fusion protein candidate, into preclinical development for Alzheimer’s disease by the second half of 2025, along with the company’s neuroimmunology portfolio.
Biosplice Therapeutics Inc. has prepared and tested 4-alkoxypyrrolo[2,1-F][1,2,4]triazines acting as dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) inhibitors potentially useful for the treatment of Alzheimer’s disease.
Atai Therapeutics Inc. has synthesized new tetrahydro pyridine-substituted indole and azaindoles acting as 5-HT2A receptor partial agonists reported to be useful for the treatment of psychiatric disorders.
Acumen Pharmaceuticals Inc. and JCR Pharmaceuticals Co. Ltd. have entered a collaboration, option and license agreement to develop a novel therapeutic candidate for the treatment of Alzheimer’s disease (AD).
Pfizer Inc. has identified signal transducer and activator of transcription 6 (STAT6) inhibitors reported to be useful for the treatment of Alzheimer’s disease, cancer, atherosclerosis, metabolic dysfunction-associated steatohepatitis, respiratory and dermatological disorders.
2A Biosciences Inc. has described phenethylamines acting as 5-HT2A receptor agonists reported to be useful for the treatment of psychiatric, neurodegenerative, neurodevelopmental, inflammatory and eye disorders.
Merck Sharp & Dohme LLC has prepared and tested triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of Alzheimer’s disease and neurodegeneration.
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