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.
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.
GSK plc said in paperwork filed with the U.S. SEC that the potentially whopping deal inked in the summer of 2020 with Ideaya Biosciences Inc. has gone down the tubes. The two firms were collaborating on three of Ideaya’s synthetic lethality programs, MAT2A (methionine adenosyltransferase 2a), Pol Theta (polymerase theta) and Werner Helicase.
Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy.
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