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.
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.
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.
Montclair State University and the University of Maryland have discovered new dual 3’,5’-cyclic-AMP and -GMP phosphodiesterase 11A4 (PDE11A4) inhibitors potentially useful for the treatment of cognitive disorders.
The University of Toronto has patented new histone deacetylase 6 (HDAC6) inhibitors designed for use in the treatment of cancer, inflammation, neurodegeneration, infections, renal, neuromuscular, respiratory disorders and cardiometabolic syndrome.
Biofront Ltd. has designed new proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase-binding moiety coupled to a mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety as HPK1 degradation inducers. They are described as potentially useful for the treatment of cancer.
Tyligand Bioscience Ltd. has reported new dual-drug antibody-drug conjugates comprising monoclonal antibodies covalently linked to stimulator of interferon genes protein (STING; TMEM173) agonist and a cytotoxic drug.
Nimbus Salacia Inc. has disclosed new serine/threonine-protein kinase SIK2 (QIK) inhibitors potentially useful for the treatment of autoimmune disease, cancer, diabetes, inflammatory bowel disease, osteoarthritis, osteoporosis, rheumatoid arthritis and skin hyperpigmentation, among others.
Infinimmune Inc. has recently presented results at the annual American Academy of Dermatology conference regarding their anti-IL-22 antibody IFX-101 for the treatment of atopic dermatitis.
Dysregulated type 2 immune responses are central in atopic dermatitis (AD) pathophysiology. Key cytokine IL-13 drives epidermal barrier dysfunction and inflammation, while IL-31 mediates pruritus via activation of sensory neurons. Both represent clinically validated, complementary targets addressing both inflammation and itch in AD.
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