Seismic Therapeutic Inc.'s S-1117 was designed as a pan-IgG protease fused to an effector function silent human IgG1 Fc domain. The candidate, being developed for the treatment of autoantibody-mediated diseases, was engineered for chronic subcutaneous administration using a proprietary machine learning-enabled platform, with the aim of reducing immunogenicity while maintaining potency.
Researchers from Astrazeneca plc presented the discovery and preclinical characterization of AZD-9793, a novel CD8-guided T-cell engager (TCE) being developed for the treatment of hepatocellular carcinoma (HCC).
Researchers from Pyxis Oncology Inc. presented preclinical data for PYX-201, an antibody drug conjugate (ADC) that targets extra domain B of fibronectin (EDB+FN).
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Researchers from Cogent Biosciences Inc. have presented data on their PI3KA H1047R mutant inhibitor CGT-6297 PI3KA wild-type inhibitors can lead to toxicity issues such as hyperglycemia, gastrointestinal toxicity and skin reactions.
Researchers from KU Leuven presented novel urokinase plasminogen activator receptor-associated protein (uPARAP)-targeted antibody-drug conjugates (ADC), ADCE-017 and ADCE-202, being developed for the treatment of soft tissue sarcoma.
Researchers from Caedo Oncology AS presented the discovery and preclinical characterization of CO-005, a novel anti-CD47 fusion protein being developed for the treatment of lymphoma.
Blood dendritic cell antigen 2 (BDCA2) is expressed in plasmacytoid dendritic cells. These cells’ overproduction of type I interferon is closely linked to the pathogenesis of systemic lupus erythematosus and other autoimmune diseases.
Pulmonary arterial hypertension (PAH) is a condition characterized by high blood pressure in the pulmonary arteries, potentially leading to heart failure. Previous research had found that knockout of Egln1 specific to endothelial cells, which encodes prolyl 4-hydroxylase-2, led to spontaneous PAH development.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.