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.

Hemogenyx Pharmaceuticals plc has obtained approval by the institutional review board (IRB) of the company’s first clinical site to initiate a phase I trial of the company’s lead asset, HEMO-CAR-T, for the treatment of relapsed or refractory acute myeloid leukemia (AML) in adults.

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.