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. Read More
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. Read More
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. Read More
The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3). Read More
Abcellera Biologics Inc. has received a no objection letter from Health Canada authorizing its clinical trial application (CTA) for ABCL-635. Read More
Gen1e Lifesciences Inc. and the University of Maryland have patented mitogen-activated protein kinase 1 (MAPK1; ERK2) and/or mitogen-activated protein kinase 3 (MAPK3; ERK1) inhibitors. Read More
Merkel cell carcinoma is an aggressive skin carcinoma that, when advanced or metastatic, is typically treated with chemotherapy, which often leads to resistance. Read More
Researchers at the China Pharmaceutical University have developed a series of highly active receptor-interacting protein kinase 1 (RIPK1) inhibitors with potent anti-inflammatory activity. RIPK1 is a key regulator of necroptosis, a form of programmed cell death associated with various inflammatory diseases. Read More
ADC Therapeutics SA has synthesized antibody-drug conjugates comprising antibody targeting neutral amino acid transporter B(0) (SLC1A5; ASCT2) covalently linked to exatecan through a linker reported to be useful for the treatment of cancer. Read More
CCR8 is highly expressed on immunosuppressive regulatory T cells (Tregs) in various solid tumors, making it a potential target to enhance antitumor immunity and the efficacy of cancer therapies, including checkpoint inhibitors. However, the impact of CCR8 expression on the Treg phenotype and its role in cancer progression remain unclear. Read More
Shanghai Ennova Biopharmaceutical Co. Ltd. has identified condensed ring compounds acting as ATP-dependent 6-phosphofructokinase, liver type (PFKL) activators reported to be useful for the treatment of cancer, autoimmune disease, inflammatory disorders, lung diseases, metabolic diseases, sepsis and thrombosis. Read More
Suzhou Zion Pharma Technology Co. Ltd. has described transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) and/or probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) inhibitors reported to be useful for the treatment of cancer and viral infections. Read More
Pancreatic cancer is one of the deadliest malignancies, with a 5-year overall survival rate of only 13%. Earlier studies demonstrated that the Lister strain of oncolytic vaccinia virus (VVL) is capable of replicating and delivering therapeutic genes even under hypoxic conditions, highlighting its potential as a promising vector for targeting hypoxic tumors like pancreatic cancer. Read More
Esophageal cancer accounts for the sixth leading cause of cancer-related deaths worldwide. The clinical efficacy of therapies for esophageal squamous-cell carcinoma (ESCC) remain limited due to drug resistance and side effects. There is an urgent need to identify new therapeutic targets to shed light on its pathogenesis. Read More
