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.
Merck Sharp & Dohme LLC has described proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a GTPase KRAS G12D mutant-targeting moiety via a linker reported to be useful for the treatment of cancer.
Genome & Co. has divulged antibody-drug conjugates consisting of antibodies targeting basal cell adhesion molecules (BCAM) covalently linked to a cytotoxic drug through a linker reported to be useful for the treatment of cancer.
Uppthera Inc. has identified proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a serine/threonine-protein kinase PLK1 (STPK13)-targeting moiety reported to be useful for the treatment of cancer.
Arc Research Institute and Stanford University have disclosed ectonucleotide pyrophosphatase/phosphodiesterase family member 1 (ENPP1) and/or ENPP3 inhibitors reported to be useful for the treatment of cancer.
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