Hematopoietic stem cell (HSC) research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and inflammation, then, may hold the keys to preventing blood cancers, according to John E. Dick’s plenary session at the 2026 Korean Society of Hematology International Conference (ICKSH 2026), held March 26, 2026. Work in Dick’s lab has found acute myeloid leukemia (AML) HSCs that harbor preleukemic mutations long before any disease diagnosis. These insights have enabled predictive models that could identify individuals at elevated AML risk years before the onset of outright disease, opening the door to new prevention strategies. Read More
Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect. Read More
Alltrna has obtained approval in Australia to initiate a phase I trial of AP-003 in healthy volunteers under Australia’s TGA clinical trial notification scheme. AP-003 is a chemically modified, engineered transfer RNA (tRNA) oligonucleotide encapsulated in a clinically validated, liver-directed lipid nanoparticle.
Telomir Pharmaceuticals Inc. has submitted an IND application to the U.S. FDA for its lead candidate, Telomir-1 (Telomir-Zn), for the treatment of advanced and metastatic triple-negative breast cancer (TNBC). Telomir-1 is a first-in-class metal-modulating epigenetic agent designed to restore transcriptional control in tumor cells by targeting intracellular iron-zinc homeostasis. Read More
Ambrosia Biosciences Inc. has announced a $100 million oversubscribed series B financing intended to support progression of the company’s oral small-molecule GLP-1 candidate and other novel cardiometabolic programs into clinical development. Read More
Researchers from Hangzhou Innogate Pharma Co. Ltd. and Innorace Biopharma Co. Ltd. have identified new cyclic GMP-AMP synthase (MB21D1; cGAS) inhibitors that are potentially useful for the treatment of Aicardi-Goutieres syndrome, psoriasis, rheumatoid arthritis, systemic lupus erythematosus and more. Read More
Tippingpoint Biosciences Inc. has closed an oversubscribed $4.5 million seed financing. The company, spun out of a lab at University of California, San Francisco (UCSF), is advancing its novel drug discovery platform designed to identify first-in-class drugs to treat diseases related to dysfunctional DNA packaging (chromatin). Read More
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has discovered new tricyclic compounds acting as cyclin-dependent kinase 12 (CDK12)/cyclin k inhibitors reported to be useful for the treatment of cancer. Read More
Changchun Genescience Pharmaceuticals Co. Ltd. has prepared and tested new steroid compounds acting as estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) inhibitors that are potentially useful for the treatment of endometriosis. Read More
Researchers from Sichuan University reported the discovery and preclinical characterization of a new series of covalent ULK1 inhibitors. ULK1 is frequently overexpressed in colorectal cancer (CRC) and is therefore being investigated as a therapeutic target to disrupt autophagy-dependent tumor survival and growth. Read More
Shanghai Meiyue Biotech Development Co. Ltd. has disclosed new molecular glue degraders comprising an E3 ubiquitin-protein ligase binding agents coupled to proto-oncogene Vav (VAV1) acting as VAV1 degradation inducers reported to be useful for the treatment of cancer, autoimmune diseases, cardiovascular, renal, metabolic, inflammatory and neurological disorders. Read More
Ring finger protein 4 (RNF4) is a SUMO-targeted ubiquitin ligase that modulates proteins involved in cancer progression. Researchers from Chongqing Medical University and collaborators reported the development and preclinical characterization of [I], the first RNF4-targeting PROTAC degrader for the treatment of hepatocellular carcinoma (HCC). Read More
