Researchers from Captor Therapeutics Inc. presented the preclinical characterization of CT-01, a first-in-class GSPT-1 targeted degrader under investigation for the treatment of hepatocellular carcinoma.
The most common form of hereditary deafness in humans is caused by mutations in the GJB2 gene, which encodes the gap junction protein connexin 26. That regulates the transport of potassium and metabolites between inner ear cells. The coding sequence of this gene fits in an adenovirus-associated vector (AAV), making it an attractive approach for gene therapy.
Patients with metastatic or unresectable gastric cancer are usually given 5-fluorouracil (5-FU) and platinum-based chemotherapy, but patients with advance disease usually have a poor prognosis. The use of chemotherapy increased the levels of cyclooxygenase-2 in tumor cells, which in turn increase the levels of prostaglandin E2 (PGE2) in the tumor microenvironment. When PGE2 binds to their receptors EP1 to EP4 on immune cells, it triggers an immunosuppressive tumor microenvironment. The use of the EP2 and EP4 dual antagonist OCT-598 was tested in the preclinical setting for gastric cancer.
Researchers from The University of Edinburgh have presented data from a study that aimed to investigate the mechanisms behind intestinal stem cell (ISC) dysfunction in ulcerative colitis (UC).
Hepatoblastoma is a form of liver cancer affecting children and for which the current treatment option available is surgical resection followed by chemotherapy based on cisplatin or doxorubicin. Its prognosis is still poor, and the recurrence rate is high. Neddylation is a biological process that has been well studied for its role in cancer biology; Spanish researchers have hypothesized that neddylation may play a significant role in the development and progression of hepatoblastoma.
Pannexin 1 (Panx1) is a high-conductance, voltage-sensitive ion channels that exhibit flexible gating behavior upon activation, enabling the passage of ions such as Ca2+, Na⁺, K⁺, and ATP. Panx1 is expressed in cardiac tissue, but its role in ATP release and electrophysiological processes affecting cardiac function is not fully understood. Researchers from Vanderbilt University Medical Center have found that in isolated ventricular cardiomyocytes, Panx1 activation during spontaneous sarco/endoplasmic reticulum Ca2+ release amplified the NCX-driven transient inward current.
The treatment of hepatocellular carcinoma (HCC) has made progress due to immune checkpoint inhibitors (ICIs), but still many patients present innate or acquired resistance to ICIs, thus there is a need for the discovery of new therapeutic approaches for HCC treatment. Epigenetic alterations play a key role in liver cancer, so that they can suppress the expression of proteins involved in triggering the immune response against tumors. Spanish researchers have now identified a promising target for liver HCC treatment, named histone-lysine N-methyltransferase EHMT2, also known as protein G9a.
Investigating the relationship between IL-1 and inflammation in inflammatory bowel disease (IBD), researchers unveiled mesenchyme homeobox 1 (MEOX1), an IL-1-dependent transcription factor that is known to regulate fibrosis in cardiac ischemia that is tied to IBD ulcers and positively expressed in ACKR1+ ECs.
At the recent 2025 congress of the European Crohn’s and Colitis Organisation, researchers from Helixon Therapeutics presented the discovery and preclinical characterization of HXN-1002, a bispecific antibody simultaneously targeting both α4β7 and TL1A that is being investigated as a potential treatment for inflammatory bowel disease.
Anterior gradient 2 (AGR2) is a protein located in the endoplasmic reticulum involved in protein folding and when stressed, it is secreted into the extracellular space and it attracts monocytes, activates fibroblasts and disrupts the epithelial integrity. The relationship between the expression of AGR2 in tissues from patients with inflammatory bowel disease (IBD) was investigated, as well as the efficacy of an anti-AGR2 antibody in murine models of colon inflammation and fibrosis.
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