CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co. Ltd. has reported new monoamine oxidase-B (MAO-B) inhibitors potentially useful for the treatment of depression, stroke, hypertension, obesity, Alzheimer’s and Parkinson’s disease.
Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).
Work at Genecode AS has led to the identification of sulfonamide or sulfone compounds acting as GFRα1-RET receptor complex activators potentially useful for the treatment of neurological disorders.
Parkinson’s disease (PD), the second most prevalent neurodegenerative disorder worldwide, is characterized by the progressive degeneration of dopaminergic neurons in the substantia nigra, leading to a decline in both motor and cognitive functions. Incretin-based therapies, initially designed for the treatment of type 2 diabetes, have demonstrated relevant neuroprotective effects in preclinical models of PD.
Work at Ensem Therapeutics Inc. has led to the discovery of anilino-pyrazole derivatives acting as cannabinoid CB2 receptor agonists. As such, they are reported to be useful for the treatment of anxiety disorders, autoimmune disease, depression, neurodegeneration, neuroinflammation, osteoarthritis, chronic pain and substance abuse and dependence, among others.
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.
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