Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.
AR-V7 is the most clinically relevant androgen receptor (AR) splice variant associated with endocrine resistance and poor prognosis in patients with prostate cancer.
Steroid-resistant nephrotic syndrome (SRNS) is a disease characterized by hypoalbuminemia, proteinuria, edema and hyperlipidemia, and a cause of chronic kidney disease in the pediatric population.
Researchers from Zhuhai Grit Biotechnology Co. Ltd. recently presented preclinical characterization of a new genetically modified tumor-infiltrating lymphocyte (TIL) product, GT-216, being developed for the treatment of solid tumors.
Researchers from Biocytogen Pharmaceuticals (Beijing) Co. Ltd. presented the development of a humanized VEGFA model (B-hVEGFA mice) as a new tool for in vivo testing of VEGFA-targeting therapeutics.
Using its artificial intelligence/machine learning platform, Aurigen, Auron Therapeutics Inc. has identified histone acetyltransferase KAT2A/B as a driver of tumor cell plasticity and designed new small-molecule degraders of KAT2A/B.
Dysfunction of the complement system plays an important role in the pathogenesis of renal diseases. Complement inhibitors, such as C5 inhibitors, have shown efficacy in clinical trials, but may not be sufficient to block disease progression as monotherapy.
Despite GTPase KRAS being the most common mutated oncogene in human cancers, there is still an unmet medical need for treating KRAS G12C-mutated cancers.
Unknown etiology is commonly encountered in the kidney pre-transplant routine program. A screening program was performed to detect patients and study recipients that meet the following features: hypertension with no clear etiology and biopsies that do not match with clinical features of classical glomerulopathies.
Researchers from Astrazeneca plc reported preclinical data for AZD-8421, a selective cyclin-dependent kinase 2 (CDK2) inhibitor, currently being evaluated in early-phase clinical trials as a treatment for solid tumors.
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