Amphista Therapeutics Ltd. has developed and presented data for AMX-883, a novel orally bioavailable bromodomain-containing protein 9 (BRD9) degradation inducer for acute myeloid leukemia (AML)
treatment.
Investigators at the Netherlands Hubrecht Institute have developed a novel gut organoid model, and used it to gain insight into the functions on human microfold (M) cells. Their experiments, which were published in the Dec. 10, 2025, issue of Nature, showed that M cells present gluten-derived antigens to T cells, which suggests a role for this cell type in the onset of celiac disease.
The UK Health Security Agency (UKHSA) has identified a new recombinant strain of mpox (formerly monkeypox) that contains elements of clade Ib and clade IIb of the virus, in a traveler who recently returned from Asia. In a paper describing the new strain, the researchers at UKHSA say it is not possible to determine from a single genome how long the recombinant virus has been in circulation, or whether it will have a fitness benefit over currently circulating lineages.
Immuse Therapeutics Inc. has described heterocyclic compounds acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of cancer.
Nikang Therapeutics Inc. has divulged proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a cyclin-dependent kinase 2 (CDK2)- and/or CDK4-targeting moiety through a linker reported to be useful for the treatment of cancer.
Prelude Therapeutics Inc. has synthesized tyrosine-protein kinase JAK2 inhibitors reported to be useful for the treatment of cancer, myelofibrosis, essential thrombocythemia and graft-vs.-host disease.
Centauri Therapeutics Ltd. has disclosed conjugates consisting of a peptide such as polymyxin or its derivative covalently linked to a rhamnose unit via a linker reported to be useful for the treatment of infections.
Phenylketonuria (PKU) is an autosomal recessive disorder that results in decreased metabolism of the amino acid phenylalanine. Untreated PKU can lead to intellectual disability, seizures, behavioral problems and mental disorders. This metabolic disease is caused by mutations in the phenylalanine hydroxylase (PAH) gene, resulting in patients’ inability to convert phenylalanine.
While current treatments can prolong the life of many patients with the malignant bone cancer osteosarcoma, a substantial proportion have metastasis or recurrence. This highlights the need for more specific, targeted therapies against the disorder, yet the mechanism of pathogenesis is unclear and may be heterogeneous, so no drug targets have been definitively validated.
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