A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
Ascletis Pharma Inc. has selected ASC-35, a once-monthly, subcutaneously administered GLP-1 receptor (GLP-1R)/GIP receptor (GIPR) dual peptide agonist, as a clinical development candidate. Ascletis expects to submit an IND application to the FDA for ASC-35 for the treatment of obesity in the second quarter of next year.
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
Pfizer Inc. has identified new indole analogues acting as sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatotic liver disease (NAFLD; MASLD), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism-spectrum disorders, among others.
Confo Therapeutics NV has secured a 2-year €1 million (US$1.2 million) grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance research and development of ultra-long-acting medicines targeting G protein-coupled receptors (GPCRs), including bi- and multispecific antibody formats for obesity and other metabolic and endocrine disorders.
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.
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