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. Read More
ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone. Read More
Akari Therapeutics plc has released new preclinical data indicating the therapeutic potential of AKTX-101 in pancreatic cancer driven by KRAS mutations. AKTX-101 is an antibody-drug conjugate (ADC) that delivers a novel RNA spliceosome modulating payload, PH1, into cancer cells that express TROP2. Read More
Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications. Read More
Ideaya Biosciences Inc. has submitted an IND application to the FDA for IDE-574, a KAT6/7 dual inhibitor with potential to treat hormone receptor-positive breast cancer and lung adenocarcinoma. A phase I trial of IDE-574 monotherapy is expected to begin in the first quarter of next year. Read More
A consortium including Korea Disease Control and Prevention Agency (KDCA), International Vaccine Institute (IVI), ST Pharm Co. Ltd. and Seoul National University (SNU) is joining forces with CEPI to advance a new AI-designed mRNA vaccine to protect against tick-borne severe fever with thrombocytopenia syndrome (SFTS) virus, or Dabie bandavirus. CEPI is providing up to US$16 million to the project, led by IVI, to test the vaccine’s safety and ability to generate a suitable immune response in healthy adults in preclinical and phase I/II trials in Korea. Read More
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. Read More
Oxford Biotherapeutics Ltd. has entered into a multiyear, multitarget strategic collaboration with GSK plc to discover novel antibody-based therapeutics for the treatment of cancer. Read More
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. Read More
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. Read More
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. Read More
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.
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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. Read More
