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.
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.
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.
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.
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.
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.
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