As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.
Washington University in St. Louis has disclosed new autophagy inducers reported to be useful for the treatment of alpha-1 antitrypsin deficiency, amyotrophic lateral sclerosis, Alzheimer’s, Huntington’s and Parkinson’s disease.
A major contributor to obesity-related pathology is inflammation involving a shift in macrophage polarization toward the inflammatory M1 phenotype and accumulation of such macrophages in the liver and adipose tissue. Activation of Toll-like receptor 4 (TLR4) on macrophages leads the downstream adaptor protein MyD88 to promote M1 polarization through altered gene expression mediated by the transcription factor NF-κB.
The Scripps Research Institute has disclosed peptides and their drug conjugates acting as dual gastric inhibitory polypeptide receptor (GIPR) and glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, metabolic dysfunction-associated steatotic liver disease and obesity.
KAYO-1732 is a novel, orally available, small-molecule inhibitor of the aldehyde dehydrogenase 1A3 (ALDH1A3) enzyme, being developed for the treatment of type 2 diabetes and cardiovascular disorders.
Obesity is a multifactorial metabolic disorder with limited treatment options capable of sustaining weight loss and improving systemic metabolic health. MicroRNA-22 (miR-22) has emerged as an essential regulator of metabolic homeostasis, influencing lipid biosynthesis, mitochondrial function and brown adipose tissue development. These roles position miR-22 as a promising target for therapeutic intervention in obesity and related disorders such as metabolic dysfunction-associated steatotic disease (MASLD) and its progressive disease state, metabolic dysfunction-associated steatohepatitis (MASH).
Genfit SA is discontinuing its VS-01 program in acute-on-chronic liver failure (ACLF), and has decided to reprioritize development of VS-01 for urea cycle disorder.
A new generative AI model trained on UK Biobank data can simultaneously forecast the risks and timing of developing over 1,000 different diseases a decade into the future. The developers applied similar algorithmic concepts to those used to develop large language models like ChatGPT and Gemini to build the model, using medical records from 402,799 participants in UK Biobank.
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