Rac1 is a small GTPase, the hyperactivation of which is linked to tumor progression and drug resistance. The oncogenic variant Rac1b has been shown to be overexpressed in cancers, such as colorectal cancer (CRC), and it has also been correlated with poor prognosis and CRC resistance to oxaliplatin.
The U.S. FDA has cleared Centessa Pharmaceuticals plc’s IND to initiate a phase I first-in-human, clinical trial of ORX-750 for the treatment of narcolepsy.
In tumors with amino acid deprivation, eIF-2α kinase GCN2 is activated and triggers a signaling response to promote cell survival and proliferation. This is important in high metabolically active hematological cancers, such as acute myeloid leukemia (AML).
The generation of pathogenic autoantibodies is a crucial event in the development of inflammation and complement activation, leading to immune cell responses.
Ipsen SA and Skyhawk Therapeutics Inc. have entered an exclusive worldwide collaboration to discover and develop small molecules that modulate RNA for rare neurological diseases. Skyhawk has a unique platform that accelerates building RNA-targeting small molecules across several therapeutic areas.
Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.
Cross-talk between macrophages and tumor cells could modulate cachexia in pancreatic cancer patients. A group of scientists from the University of Oklahoma has discovered a new pathway that promoted muscle wasting after the recruitment of this immune cell in the tumor microenvironment, activating cachexia-inducing factors. Macrophage depletion and the inhibition of this signaling could be developed as a therapeutic target for this condition.
New York University has described protein spinster homolog 2 (SPNS2) inhibitors reported to be useful for the treatment of acute lung injury, autoimmune disease, colitis, Alzheimer’s disease, fibrosis, inflammatory bowel disease, multiple sclerosis and rheumatoid arthritis.
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