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Other news to note for May 25, 2023

May 25, 2023
Additional early-stage research and drug discovery news in brief, from: Immunome, Tavanta Therapeutics.
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Cancer

Simcere Zaiming Pharmaceutical reports new CBL-B inhibitors for cancer and autoimmune disease

May 25, 2023
Research at Simcere Zaiming Pharmaceutical Co. Ltd. has led to the development of polycyclic compounds acting as E3 ubiquitin-protein ligase CBL-B inhibitors and thus reported to be useful for the treatment of cancer and autoimmune disease.
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Cancer

China Pharmaceutical University and Jiangsu Chia Tai Tianqing Pharmaceutical identify Wee1 inhibitors for treatment of cancer

May 25, 2023
China Pharmaceutical University and Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. have jointly developed Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer.
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Immune

Kymera Therapeutics prepares and tests TYK2 degradation inducers

May 25, 2023
Proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to non-receptor tyrosine-protein kinase TYK2-targeting moiety have been reported in a Kymera Therapeutics Inc. patent as potentially useful for the treatment of neurological, inflammatory and endocrine disorders, autoimmune diseases, transplant rejection, graft-vs.-host disease and cancer.
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Cancer

Perha Pharmaceuticals patents CLK1, CLK4 and/or DYRK1A inhibitors

May 25, 2023
Recent Perha Pharmaceuticals patents describe imidazolone derivatives acting as inhibitors of dual specificity protein kinase CLK1 and/or CLK4 and/or and dual-specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A). As such, they are reported to be useful for the treatment of diabetes, cancer, CDKL5 deficiency disorder, chromosome 22q13 deletion syndrome, Alzheimer’s disease, viral infections, Niemann-Pick disease type C and tauopathies, among others.
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Neurology/Psychiatric

Marvel Biotechnology patent details new compounds for treatment of depression

May 25, 2023
Marvel Biotechnology Inc. has reported the identification of compounds potentially useful for the treatment of depression.
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Degradation of motor neurons
Neurology/Psychiatric

Next-generation gene therapy shows superior efficacy for spinal muscular atrophy

May 25, 2023
Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for SMA that confer better safety and efficacy.
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Illustration of the inside of an eye with macular degeneration
Ocular

SKG-0106 shows durable efficacy in preclinical models of neovascular AMD

May 25, 2023
Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).
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Alpha-galactosidase enzyme
Endocrine/Metabolic

AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease

May 25, 2023
Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.
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Cancer cells under magnifying glass
Cancer

Discovery of new IDO-1 inhibitors with potent anticancer activity

May 25, 2023
Researchers from Guilin Medical University (GLMU) reported the discovery and preclinical evaluation of novel indoleamine 2,3-dioxygenase 1 (IDO-1) inhibitors as potential anticancer agents. Synthesis and optimization of a series of chromone-oxime derivatives containing piperazine sulfonamide moieties led to the identification of compound [I] as the lead IDO-1 inhibitor.
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