Loss-of-function mutations in the CTNNB1 gene cause β-catenin deficiency, leading to CTNNB1 syndrome, a rare neurodevelopmental disorder marked by motor and cognitive impairments. Because the disease stems from single-allele mutations that vary widely, a broadly applicable corrective strategy is needed. Since CTNNB1 is dosage-sensitive, therapies must preserve normal regulation, and in some cases may also need to suppress harmful mutant transcripts.
Despite the success of traditional viral-based CAR T-cell therapies against several blood malignancies, their efficacy remains limited against solid tumors. Non-viral engineering of CAR T cells using electroporation or lipid nanoparticle delivery of CAR-encoding mRNA achieves high but transient CAR expression, highlighting the limitations of current preclinical models for evaluating mRNA-based CAR T cells.
Arc Therapies Inc., a startup from the National Cancer Center Japan, has initiated research of YB328, a newly identified gut microbe, toward clinical application. The company has designated the YB328 strain as ARC-0812 (RUX: Lux) and will proceed with preclinical and clinical trials to explore its role as an immune adjuvant in cancer immunotherapy.
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by the formation of an invasive and proliferative tissue called synovial pannus, consisting of fibroblast-like synoviocytes (FLSs), immune cells and newly formed vessels.
Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications.
Researchers from the California Institute of Technology and collaborating institutions have developed a novel HIV vaccine candidate, a new germline-targeting Env SOSIP trimer called 3nv.2, that is designed to elicit antibodies targeting three key epitopes on the HIV envelope protein.
Deficiencies in interferon-stimulated gene 15 (ISG15), a protein that normally regulates the immune response, causes mild but persistent inflammation. However, its absence also provides an unexpected advantage by increasing resistance to viral infections. Inspired by this condition and using mRNA technology, scientists at Columbia University and the Icahn School of Medicine at Mount Sinai have developed a broad-spectrum antiviral platform.
Chengdu Chipscreen Pharmaceutical Ltd. has described ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
Scientists from Jordan Ministry of Higher Education Scientific Research and the Hashemite University have identified doxorubicin and thymoquinone hybridized compounds with reduced side effects reported to be useful for the treatment of breast cancer.
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