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. Read More
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. Read More
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. Read More
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. Read More
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Neushen Therapeutics Inc. has disclosed N-acetyl-β-D-glucosaminidase (O-GlcNAcase; OGA) inhibitors reported to be useful for the treatment of Alzheimer’s disease. Read More
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. Read More
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. Read More
Shenzhen Chipscreen Biosciences Ltd. has synthesized MAPK-interacting kinase 1 (MNK1) and/or MAPK-interacting kinase 2 (MNK2) inhibitors reported to be useful for the treatment of cancer. Read More
Hanx Biopharmaceuticals Inc. has identified antibody-drug conjugates (ADCs) consisting of an antibody targeting TRBV12 covalently linked to cytotoxic drugs through linkers reported to be useful for the treatment of T-cell leukemia and T-cell lymphoma. Read More
Secondary lymphedema is a chronic condition that often affects the upper or lower extremities and is characterized by progressive retention of lymphatic fluid at affected sites. At the affected sites, this progressive fluid retention often leads to pain, decreased function and wounds. The condition is commonly associated with cancer surgery that requires the removal of draining lymph nodes. Read More
Medicines for Malaria Venture and The University of Texas System have divulged dihydroorotate dehydrogenase (DHODH) (Plasmodium falciparum and Plasmodium vivax) inhibitors reported to be useful for the treatment of malaria. Read More
Fortvita Biologics Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of autoimmune diseases, cardiovascular disorders, inflammatory disorders, diabetes, lung diseases, neurological disorders, obesity and renal disorders, among others. Read More
Shenzhen Grit Biotechnology Co. Ltd. and Shanghai Vitalgen Biopharma Co. Ltd. recently presented their work to develop and evaluate a novel anti-CD19 in vivo CAR T candidate, named GT-801. Read More
