GAS41, the chromatin-associated protein encoded by YEATS4, is frequently overexpressed in non-small-cell lung cancer (NSCLC). Beyond its association with epigenetic dysregulation, GAS41 plays an active role in modulating transcriptional programs that are essential for NSCLC pathogenesis, underscoring its suitability as a mechanistically well-supported therapeutic target.
The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.
Harbour Biomed and Yantai Lannacheng Biotechnology Co. Ltd. have entered into a long-term strategic collaboration to jointly advance the development of next-generation radionuclide-drug conjugates for the treatment of cancer.
Adlai Nortye Ltd. has entered into an exclusive licensing agreement with Jiangsu Aosaikang Pharmaceutical Co. Ltd. (Ask Pharm) for its proprietary pan-RAS(ON) inhibitor AN-9025, an oral small molecule designed to target a broad spectrum of RAS mutations across various tumor types.
Glioblastoma (GBM), the most common malignant brain tumor, remains difficult to treat because cancer stem cells (CSCs) drive resistance and recurrence. Although the Bruton tyrosine kinase (BTK) inhibitor ibrutinib suppresses GBM cell growth and stem-like traits, its limited selectivity and off-target activity raise safety concerns, highlighting the need for more specific BTK inhibitors.
Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
Haisco Pharmaceutical Group Co. Ltd. has described compounds acting as TNF-α/tumor necrosis factor receptor superfamily member 1A (TNFR1) interaction inhibitors reported to be useful for the treatment of psoriasis, Crohn’s disease, ulcerative colitis and rheumatoid arthritis.
Shenzhen Targetrx Inc. has divulged proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN)-binding moiety covalently linked to Raf kinase B (V600E mutant)-targeting moiety through linker reported to be useful for the treatment of cancer.
Researchers at Institute of Materia Medica Chinese Academy of Medical Sciences & Peking Union Medical College have identified 5’-nucleotidase (CD73) inhibitors reported to be useful for the treatment of cancer, fibrosis, cerebral ischemia, depression, sleep disorders, Parkinson’s disease, immunological disorders and inflammatory disorders, among others.
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