TRIM21 marks viruses and bacteria for degradation via autophagy
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.

Technique links non-coding mutations to disease genes they regulate
Genome-wide association studies (GWAS) have identified multiple loci associated with complex diseases, but these are mostly on regulatory genes in the non-coding part of the genome and it has proved difficult to identify the effector genes that they control. Now, researchers in the U.K. have shown how single cell sequencing at scale can be used to precisely link non-coding GWAS loci to specific protein coding genes and cell types.

TRIM21 marks viruses and bacteria for degradation via autophagy
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.
Technique links non-coding mutations to disease genes they regulate
Genome-wide association studies (GWAS) have identified multiple loci associated with complex diseases, but these are mostly on regulatory genes in the non-coding part of the genome and it has proved difficult to identify the effector genes that they control. Now, researchers in the U.K. have shown how single cell sequencing at scale can be used to precisely link non-coding GWAS loci to specific protein coding genes and cell types.
Pfizer’s integrin β6 ADC advances intravesical therapy for NMIBC
Researchers from Pfizer reported preclinical efficacy of PF-08052667, an antibody-drug conjugate (ADC) targeting integrin β6 (ITGB6), in non-muscle-invasive bladder cancer (NMIBC) models.
Arvinas joins MJFF initiatives to advance LRRK2 research
Arvinas Inc. has joined the LRRK2 Investigative Therapeutics Exchange (LITE) program and the Parkinson’s Precision Medicine Initiative (PPMI), both supported by The Michael J. Fox Foundation for Parkinson’s Research (MJFF).
Sichuan Kelun-Biotech patents new anthracyclins
Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. has disclosed new anthracyclins that are useful for the treatment of cancer.
Preclinical evaluation of a CAIX-targeted PET tracer
The University of Texas MD Anderson Cancer Center reported findings from studies of CBT-001-2334, a radionuclide peptide targeting carbonic anhydrase IX (CAIX/CA9) designed for diagnostic gallium labeling and downstream therapeutic isotope pairing. It features a DOTA chelator for use in theranostic applications through chelating either diagnostic or therapeutic radionuclides. Because it has limited expression in normal tissue, CAIX is an attractive target in clear cell renal cell carcinoma (ccRCC).

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Conferences

FB-7033 demonstrates efficacy in MASH management
EASL
17-β-Hydroxysteroid dehydrogenase 13 (HSD17B13) and lipid transferase CIDEB are known to contribute to metabolic dysfunction-associated steatohepatitis (MASH) progression, where HSD17B13 exacerbates hepatic lipid metabolism, while CIDEB mainly mediates lipid droplet dynamics and storage. In this...
SKL-35501 shows broad antitumor activity in solid tumors
Cancer
Neurotensin receptor 1 (NTSR1) is overexpressed in several aggressive solid tumors, including colorectal and pancreatic cancer, making it a promising therapeutic target. SKL-35501 is a novel 225Ac-labeled radiopharmaceutical developed by SK Biopharmaceuticals Co. Ltd. that targets NTSR1 for...
Preclinical results support Actinium’s radioconjugate ATNM‑400 for NSCLC
Cancer
At the recent meeting of the Society of Nuclear Medicine and Molecular Imaging, researchers from Actinium Pharmaceuticals Inc. presented preclinical efficacy data on ATNM-400 in models of non-small-cell lung cancer (NSCLC).
TAK1 emerges as a therapeutic target in MASLD
EASL
Transforming growth factor-β-activated kinase 1 (TAK1) is a crucial central signaling molecule of hepatic cell death, inflammation and fibrogenesis through NF-κB and MAPK in metabolic dysfunction-associated steatotic liver disease (MASLD). Its pharmacological inhibition using the TAK1 inhibitor...

Cancer

Sotio Biotech’s SOT-106 designated orphan drug for osteosarcoma
Regulatory
Sotio Biotech AS’s SOT-106 has been granted orphan drug designation by the FDA for the treatment of osteosarcoma. SOT-106 is a next-generation antibody-drug conjugate targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in...

Infection

Next-generation FXIIa inhibitor shows promise in sepsis models
Factor XIIa (FXIIa) has been explored as a therapeutic target for thrombosis and immune-mediated disorders due to its role at the interface of coagulation and inflammation. However, poor plasma stability and reversible target engagement have limited earlier approaches, underscoring the need for...

SR-1891 achieves prolonged HBV suppression with one dose
Conferences
Researchers from Ospedale San Raffaele presented the preclinical characterization of SR-1891, a long-acting capsid assembly modulator (CAM) in models of chronic hepatitis B. In Hepa D38 cells, the compound exhibited EC50 and EC90 values of 0.2 and 0.8 nM, respectively, without any detectable...
CEPI backs vaccine candidates for Bundibugyo Ebola virus
Immune
The Coalition for Epidemic Preparedness Innovations (CEPI) has announced funding and support to urgently accelerate development of three investigational vaccines targeting the Bundibugyo Ebola virus.

Neurology/psychiatric

Idefine announces Kleefstra syndrome gene therapy collaboration
Financings
Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role...
MJFF awards grant to Montara Therapeutics’ mTOR program
Financings
Montara Therapeutics Inc. has been awarded a research grant of approximately $1 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance a Brainonly therapy targeting mTOR for Parkinson’s disease.
Janssen Pharmaceutica reports new TMEM175 activators
Patents
Janssen Pharmaceutica NV has identified new endosomal/lysosomal proton channel TMEM175 activators that are potentially useful for the treatment of Parkinson’s disease.
MJFF grants supports Lysoway’s TMEM175 agonist program
Financings
Lysoway Therapeutics Inc. has been awarded an additional research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support development of its TMEM175 agonist program for Parkinson’s disease.
Voyager’s tau-targeted gene therapy VY-1706 gains IND approval
Regulatory
Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.

Endocrine/metabolic

GPR119 activation ameliorates alcohol-related liver disease
Conferences
There is a growing consensus that alcohol-related liver disease (ALD) should be considered a metabolic disorder under the influence of the gut-liver axis. Metabolome data have highlighted fatty acid-activated G protein-coupled receptors (GPCRs) as the main affected pathways, where the relationship...

Biomarkers

Researchers identify innate immune barrier against melanoma
Science
Australian researchers have identified a previously overlooked population of immune cells in the skin that physically restrain melanoma growth by engulfing live melanoma cells, and the discovery could reshape thinking around macrophage-targeted cancer therapies and innate immunity in oncology.

Menstrual ‘footprints’ may act as broad personal health markers
Science
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...
Circulating sncRNA transcriptome in diabetic kidney disease and SGLT2 inhibitor treatment
Nephrology
Small noncoding RNAs (sncRNAs), including miRNAs, piRNAs and snoRNAs, can provide further biological insights into the mechanisms of chronic kidney disease (CKD) in diabetes. Researchers from Leiden University Medical Center and collaborating institutions previously discovered that various classes...

Gastrointestinal

Innate immunity and microbiome allied against pulmonary fibrosis
Science
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...
ATGL inhibition reshapes immunometabolism in liver fibrosis
Conferences
Adipose triglyceride lipase (ATGL), a central mediator of triglyceride hydrolysis and fatty acid mobilization, modulates hepatic lipid homeostasis and metabolic signaling pathways that contribute to the activation of fibrogenic responses.
PLT-012 shows efficacy across different MASH models
Conferences
Researchers from Pilatus Biosciences SA recently presented preclinical efficacy data for PLT-012, a humanized IgG4 antibody targeting CD36, in metabolic dysfunction-associated steatohepatitis (MASH) models.
EASL 2026: Emerging liver disease targets to watch
Conferences
At the recently concluded European Association for the Study of the Liver meeting, presentations underscored how increasingly granular insights into liver pathobiology are driving the rapid identification of new druggable targets across diverse...
Pancreatic lesions show cancer traits but lack stromal support
Science
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...
EASL 2026: Rebuilding the liver, from organoids to engineered ducts
Conferences
With a historic WHO resolution adopted this week giving countries, for the first time, a mandate to address liver disease affecting 1.5 billion people worldwide, this momentum is strongly reflected at the ongoing European Association for the Study...

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