Cure Rare Disease (CRD) announced that the FDA has approved the first-in-human IND for CRD-TMH-001, its first therapeutic candidate using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy.
Beckley Psytech has divulged ergoline analogues acting as 5-HT2A and/or 5-HT2B and/or 5-HT2C receptor agonists reported to be useful for the treatment of depression.
To explore the role of histone deacetylase 11 (HDAC11) in Alzheimer's disease, Massachusetts General Hospital researchers generated selective, brain-penetrating HDAC11 inhibitors.
Beta-site APP-cleaving enzyme 1 (BACE1) inhibitors have a long history of failure in patients with Alzheimer's disease (AD). Clinical development of verubecestat, elenbecestat, lanabecestat, umibecestat, atabecestat and LY-3202626 were all discontinued.
After stroke, chondroitin sulfate proteoglycans (CSPGs) inhibit neuroblast migration and axon sprouting, but these effects can be blunted by modulating receptor-type tyrosine-protein phosphatase S (PTPsigma).
Data from preclinical studies conducted to examine the activities of the novel estrogen receptor beta (ERbeta) agonist EGX-358, previously under study for the treatment of hot flushes at Estrigenix Therapeutics, for the potential treatment of Alzheimer's disease (AD) were presented at the recent Alzheimer's Association International Conference.
Design Therapeutics has discovered conjugates consisting of a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to a protein-binding moiety through an oligomeric backbone linker reported to be useful for the treatment of Friedreich ataxia.
Sosei Group Corporation and AbbVie have entered a new drug discovery collaboration and option-to-license agreement to discover, develop and commercialize small molecules that modulate novel G protein-coupled receptor (GPCR) targets associated with neurological disease.
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