Atyr Pharma Inc. has advanced ATYR-0101 into IND-enabling studies for pulmonary fibrosis, and is targeting an IND application in the second half of next year.
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
Additional early-stage research and drug discovery news in brief, from: Coave Therapeutics, Cognition Therapeutics, Gain Therapeutics, Hoth Therapeutics, Kriya Therapeutics, Rectify Pharmaceuticals.
The U.S. FDA’s decision to phase out animal testing for INDs is driving a new market of alternative, nonanimal testing technologies like organoids and organs-on-a-chip, speakers at Bio Korea 2025 said.
Shenzhen Zhongge Biotechnology Co. Ltd. has described proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding agent coupled to interleukin-1 receptor-associated kinase 4 (IRAK-4) targeting moiety via linkers acting as IRAK-4 degradation inducers reported to be useful for the treatment of cancer, autoimmune disease, inflammatory disorders, transplant rejection, thromboembolism, atherosclerosis, myocardial infarction and metabolic syndrome.
Sevenless Therapeutics Ltd. has disclosed son of sevenless homolog 1 (SOS1) inhibitors reported to be useful for the treatment of cancer, pain, dementia, Noonan syndrome, Parkinson's disease and neurofibromatosis type 1.
Gachon University has divulged quinoxaline compounds acting as proto-oncogene tyrosine-protein kinase receptor Ret (RET; CDHF12; PTC) and its mutant inhibitors reported to be useful for the treatment of cancer.
Synnovation Therapeutics Inc. has prepared and tested phosphatidylinositol 3-kinase α (PI3Kα) inhibitors reported to be useful for the treatment of cancer and more.
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