Brain-derived neurotrophic factor (BDNF) is a neurotrophin that supports neuronal growth and survival and plays a critical role in regulating synaptic plasticity and cognitive function. Reduced BDNF levels have been observed in Alzheimer’s disease, Parkinson’s disease and mood disorders, suggesting that agents capable of enhancing BDNF expression may represent promising therapeutic strategies for both neurodegenerative diseases and depression.
The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes.
Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions.
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.
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