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BioWorld - Friday, April 10, 2026
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Gastrointestinal

TLR4 behind severity of ileitis and enteric neuropathy

Jan. 30, 2025
It was hypothesized that the crosstalk between Toll-like receptors, especially Toll-like receptor 4 (TLR4), and serotonergic machinery during inflammation may be an important factor for understanding the neuroimmune mechanisms participating in inflammatory bowel disease pathogenesis.
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Optogenetics illustration
Ocular

Dual AAV vectors enable precise gene correction for Stargardt disease in preclinical models

Jan. 30, 2025
Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).
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3D illustration of brain cancer
Neurology/psychiatric

Small-molecule GIRK agonist reduces seizure-like activity ex vivo

Jan. 30, 2025
Researchers from Columbia University Irving Medical Center published data from a study that assessed GiGA1, a selective G protein-gated inwardly rectifying potassium channel (GIRK) activator, in physiologically and clinically relevant ex vivo mouse models of tumor-associated and provoked seizures.
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Cancer cell targeted in crosshairs
Cancer

Dewpoint nominates second condensate modulator for Wnt-driven cancers

Jan. 30, 2025
Dewpoint Therapeutics Inc. has nominated a second development candidate, DPTX-3496. The oral, small-molecule condensate modulator targets β-catenin to address Wnt-driven colorectal cancer, triple-negative breast cancer and non-small-cell lung cancer. IND-enabling studies have commenced, with an IND filing planned in the second half of this year.
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Inflammatory

Chinese researchers describe JX-06’s effects in multiple organ injury

Jan. 30, 2025
Multiple organ dysfunction syndrome is a life-threatening condition derived from heat stress or sepsis, among other conditions, that can lead to irreversible organ damage and death if left untreated. Gasdermin (GSDMD) is an executor protein that causes cell lysis, facilitates the release of inflammatory mediators and is involved in pyroptosis, apoptosis and necroptosis processes leading to inflammatory cell death or PANoptosis.
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3D illustration of RNA and proteins
Neurology/psychiatric

Harness going after new target, FAN-1, in Huntington’s disease

Jan. 29, 2025
By Nuala Moran
Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.
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Other news to note for Jan. 29, 2025

Jan. 29, 2025
Additional early-stage research and drug discovery news in brief, from: Silexion Therapeutics, Telomir Pharmaceuticals.
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Inflammatory

Nitric oxide scavengers detailed in Omniamed and Pohang University patent

Jan. 29, 2025
Omniamed Co. Ltd. and Pohang University of Science & Technology have patented compounds acting as nitric oxide (NO) scavengers and reported to be useful for the treatment of cancer, allergy, obesity, myelofibrosis, autoimmune diseases, inflammatory, cardiovascular and neurological disorders, among others.
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Cancer

Synnocare reports new NMT1 inhibitors

Jan. 29, 2025
Nanjing Synnocare Pharmaceutical Technology Co. Ltd. and Tianjin Synnocare Biomedical Technology Co. Ltd. have jointly developed new N-myristoyltransferase 1 (NMT1) inhibitors reported to be useful for the treatment of cancer, malaria, leishmaniasis, rhinovirus and HIV infections.
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Inflammatory

TGM2 inhibitors described in Aston University patent

Jan. 29, 2025
Work at Aston University has led to the identification of tissue transglutaminase (TGM2) inhibitors potentially useful for the treatment of fibrosis, thrombosis, cancer, AIDS, inflammation, transplant rejection, neurodegenerative and autoimmune diseases, among others.
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