Cytokinetics Inc. has disclosed fast type myosin-binding protein C (MYBPC2) inhibitors reported to be useful for the treatment of neuromuscular disorders.
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding.
Huntington’s disease is an inherited neurodegenerative disease in which the huntingtin protein undergoes polyglutamine expansion, leading to both loss and gain of functions. A research collaboration spanning France, Germany, the Netherlands and the U.S. showed in previous work that overexpressing CYP46A1 in the brain can mitigate disease in multiple mouse models.
Bright Minds Biosciences Inc. has released preclinical results for BMB‑201, a selective 5-HT2A/2C receptor agonist, in a validated isosorbide dinitrate (ISDN) rat model of vascular headache. BMB-201 is designed to harness the analgesic potential of serotonin modulation without the hallucinogenic effects commonly associated with 5-HT2A activation.
Humanwell Pharmaceutical US Inc. has described sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, cough, urinary incontinence and multiple sclerosis.
Researchers from Mount Sinai Center for Translational Medicine and Pharmacology at Icahn School of Medicine at Mount Sinai and colleagues have developed a therapeutic humanized antibody that blocks the action of follicle-stimulating hormone (FSH), a pituitary hormone previously thought to only play a role in fertility.
Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy.
Two independent studies have linked neuronal injury, inside or outside the brain, to cancer progression and offer new biomarkers and strategies for prevention. While cerebral cancer cells damage axons and drive tumor development, in other types of cancer affecting other organs, nerve disruption caused by tumor proximity triggers inflammation and a suppressive environment that may also be associated with immunotherapy resistance.
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