The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease. Read More
Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons. Read More
Merck Sharp & Dohme Corp. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammation. Read More
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China's National Medical Products Administration (NMPA) has given drug clinical trial approval for two new COVID-19 vaccines against the current XBB variants developed by Westvac Biopharma Co. Ltd. and West China Medical Center at Sichuan University. Read More
MicroRNA-184 (miR-184) mutations have been previously tied to inherited anterior segment dysgenesis, potentially causing cataracts and keratoconus. To elucidate the involvement of miR-184 in ocular disorders, Chinese researchers have developed a miR-184 knockout zebrafish model. Investigators used CRISPR-Cas9 technology to delete two miR-184 paralogs (dre-mir-184-1 and dre-mir-184-2) in zebrafish, which are highly conserved between human and fly. Read More
Bolt Biotherapeutics Inc. has synthesized antibody-drug conjugates (ADCs) comprising cysteine-mutant antibodies targeting programmed cell death 1 ligand 1 (CD274; PD-L1) covalently bound to one or more Toll-like receptor 7 (TLR7) and/or TLR8 agonists through a linker reported to be useful for the treatment of cancer. Read More
Acerand Therapeutics (Hong Kong) Ltd. has identified tetrahydrofuran-containing polycyclic derivatives and their pharmaceutically acceptable salts reported to be useful for the treatment of ovarian cancer. Read More
Researchers from Wenzhou Medical University and affiliated organizations have published data from a study that aimed to investigate the relationship between the tumor suppressor microRNA-143 (miR-143) and RNA-binding protein Musashi homolog 2 (MSI2), the abnormal expression of which has been previously associated with cancer progression. Read More
Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has divulged exportin-1 (CRM1; XPO1) receptor antagonists reported to be useful for the treatment of lymphoma and leukemia. Read More
Sironax has described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Parkinson’s disease, multiple sclerosis, traumatic brain injury, diabetic neuropathy and chemotherapy-induced peripheral neuropathy. Read More
Distant metastasis and drug resistance are the main causes of colorectal cancer (CRC)-derived mortality. Identifying the underlying mechanisms driving metastasis is key for improving its therapy. Epithelial-to-mesenchymal transition (EMT) promotes the migration and invasion of tumor cells, allowing them to metastasize distant organs. Chinese researchers put the focus on dishevelled segment polarity protein 3 (DVL3) due to its involvement in the Wnt signaling pathway. Read More
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: Adicet Bio, Adverum Biotechnologies, Bloomsbury Genetic Therapies, Canbridge Pharmaceuticals, Chroma Medicine, Mink Therapeutics, Orchard Therapeutics, Orna Therapeutics, Outpace Bio, Poseida Therapeutics, Voyager Therapeutics. Read More
