Modulating G protein-coupled receptors (GPCRs) is one of the major challenges in biomedicine. These are flexible proteins with small, deep binding pockets. The scientific community has explored small molecules, antibodies and nanobodies to develop ligands. Skape Bio Inc. is betting on creating miniproteins, a strategy that brings precise solutions for different functions.
TRIM21, an enzyme involved in intracellular substrate degradation, can recognize viruses and bacteria that enter the cytosol when they are coated with antibodies. Just as it tags complex molecules for elimination, it can direct these infectious microorganisms to lysosomes through a mechanism its discoverers have termed antibody-directed xenophagy (ADX). Scientists at the Medical Research Council (MRC) Laboratory of Molecular Biology (LMB) in Cambridge, U.K., have identified the genes involved in this antibody-dependent degradation pathway, which acts as an antimicrobial process, and reported their findings in Molecular Cell on June 4, 2026.
The microbiome and a frontline innate antimicrobial sensor, Toll-like receptor 5 (TLR5), play an essential role in the development of idiopathic pulmonary fibrosis (IPF). A scientific collaboration led by researchers at the National Institute of Environmental Health Sciences has revealed how TLR5 protects against fibrosis through its ability to modulate the lung microbiome. Their study also shows that activating TLR5 protects against fibrosis and corrects pulmonary dysbiosis.
The human pancreas often contains lesions that are common in the population and that, although they can become malignant, usually do not. To understand when this occurs and how these pancreatic cancer precursor lesions, known as pancreatic intraepithelial neoplasias (PanIN), evolve, scientists at the University of Michigan analyzed their gene expression patterns and their cellular microenvironment.
Recent findings are reshaping current understanding of the post-infection landscape of SARS-CoV-2. Although previous studies had already suggested that autoimmunity might underlie the persistent neurological symptoms seen in long COVID, researchers at Yale University and Mount Sinai now reinforce this hypothesis. SARS-CoV-2 infection appears to trigger an autoimmune mechanism that drives chronic pain, fatigue and cognitive impairment in some patients.
WAVES, an algorithm designed to extract menstrual-cycle metrics from physiological signals such as basal body temperature, which oscillates with sex hormones, shows how different parameters change with age and helps determine whether each person maintains a stable individual pattern or personal footprint. A study based on data from 5,674 cycles from 753 women demonstrates through this tool that age is associated with higher temperatures, shorter cycles, and greater irregularity. In addition, several metrics show within-person stability, suggesting they could serve as personalized health markers.
A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.
Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.
Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.
Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.