EHA 2026: With new lenses, a changing view on pediatric cancers
In the most simplistic view, adult cancers occur because “immature cells are exposed to mutagens, accumulate mutations, and across life ultimately transform into cancer cells,” Franck Bourdeaut told his audience at the 2026 Annual Congress of the European Hematology Association (EHA 2026). “On the contrary, in pediatric cancers, it is assumed that very few mutations are responsible for a maturation block, make these cells derail from their normal differentiation trajectory and ultimately result in an early onset typical pediatric cancer.”

EHA 2026: With new lenses, a changing view on pediatric cancers
In the most simplistic view, adult cancers occur because “immature cells are exposed to mutagens, accumulate mutations, and across life ultimately transform into cancer cells,” Franck Bourdeaut told his audience at the 2026 Annual Congress of the European Hematology Association (EHA 2026). “On the contrary, in pediatric cancers, it is assumed that very few mutations are responsible for a maturation block, make these cells derail from their normal differentiation trajectory and ultimately result in an early onset typical pediatric cancer.”
Novel IGF2BP3 inhibitor shows potent antileukemic profile
Insulin-like growth factor 2 mRNA binding protein 3 (IGF2BP3) is an oncofetal RNA-binding protein aberrantly overexpressed in multiple cancers, where it promotes tumor growth by stabilizing oncogenic mRNAs. Researchers at the University of California described the efficacy of I3IN-002, a potent IGF2BP3 inhibitor, in leukemia models.
JW Pharmaceutical’s DDC-02 improves cognitive function
JW Pharmaceutical Corp. has presented data on DDC-02 across animal models of several rare neurodevelopmental disorders, including Pitt-Hopkins syndrome, fragile X syndrome and Rett syndrome. Across these models, DDC-02 consistently restored deficits in cognitive and behavioral function.
Antag’s GIPR antagonist shows promise in obesity and cardiometabolic disease
Glucose-dependent insulinotropic polypeptide receptor (GIPR) signaling regulates key metabolic processes, including postprandial insulin secretion, appetite control, lipid storage and overall energy balance. Researchers from Antag Therapeutics Aps presented preclinical efficacy data on the combination of AT-7687, a GIPR antagonist, and cagrilintide in obese insulin-resistant monkeys.
Changchun Genescience Pharmaceuticals identifies new FGFR2/3 inhibitors
Changchun Genescience Pharmaceuticals Co. Ltd. has discovered salts of fibroblast growth factor receptor 2 (FGFR2) and/or fibroblast growth factor receptor 3 (FGFR3) inhibitors reported to be useful for diagnosis and treatment of cancer, achondroplasia and osteochondrodysplasia.
Applied Biologics begins osteoarthritis research program
Applied Biologics LLC has initiated a preclinical research program focused on osteoarthritis and other degenerative diseases. The program marks an expansion of the company’s research and development efforts beyond chronic wounds.

Conferences

Researchers hit back after colleagues removed from ADA meeting
American Diabetes Association
Former principal officers of the American Diabetes Association (ADA) have hit back at the association’s board and CEO after it prohibited the distribution of an editorial published in its flagship journal Diabetes Care at the ADA’s meeting in New Orleans last Friday.
MTRX-31 restores mitochondrial metabolism in obesity
American Diabetes Association
Glucagon-like peptide 1 receptor agonist (GLP-1RA) compounds have become game changers in the treatment of obesity, but they have limitations due to long-term efficacy, lean mass preservation and tolerability in certain populations. Mitorx Therapeutics Ltd. has presented data for MTRX-31 (Myo-004),...
VRB-103 shows potent and selective amylin receptor activity
American Diabetes Association
Researchers from Verdiva Bio Ltd. and collaborators recently presented preclinical data on VRB-103, an oral amylin analogue, comparing its profile with those of cagrilintide and eloralintide, two subcutaneously administered analogues currently in clinical development.
CD45RC identified as therapeutic target in Sjögren disease
Biomarkers
CD45RC is an isoform of protein tyrosine phosphatase receptor type C, a protein that plays a key role in regulating antigen receptor signaling in T and B cells. While it is expressed on most circulating B cells, it is only highly expressed on Th1 precursors, Th1 cells and T effector memory CD45RA+...

Cancer

Novel USP7 inhibitor exhibits antileukemic activity
Ubiquitin-specific protease 7 (USP7) is a central regulator of multiple oncogenic pathways and is associated with tumor aggressiveness and poor prognosis across several cancer types. Researchers from Molecure SA characterized the preclinical profile of OAT-4828, a selective USP7 inhibitor with...

Infection

U.S. researchers report new antimalarial compounds
Patents
Brown University, Florida Atlantic University and Florida International University have jointly identified new compounds potentially useful for the treatment of malaria.

TRIM21 marks viruses and bacteria for degradation via autophagy
Science
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...
Assembly Biosciences synthesizes new anti-cytomegalovirus agents
Patents
Assembly Biosciences Inc. has divulged new dihydroquinazolines potentially useful for the treatment of cytomegalovirus (CMV) infections.

Neurology/psychiatric

Scenic Biotech joins MJFF LITE program to expand PLA2G15 research
Collaboration
Scenic Biotech BV has joined The Michael J. Fox Foundation for Parkinson’s Research (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE) program to explore the therapeutic potential of its PLA2G15 inhibitor program in Parkinson’s disease, including in patients carrying LRRK2 mutations.
Microbiota modulates the antidepressant effects of GLP-1 analogues
Endocrine/metabolic
Liraglutide, a glucagon-like peptide 1 (GLP-1) receptor agonist used in diabetes and obesity, could alleviate depression through a pathway that does not depend on the GLP-1 receptor but instead on the gut microbiota, since the treatment increases the presence of the bacterium Lactobacillus delbrueckii...
New 5-HT2C receptor agonists reported in Hansoh patent
Patents
Researchers from Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have disclosed tricyclic compounds acting as 5-HT2C receptor agonists reported to be potentially useful for the treatment of mood disorders.
Grant supports Harness Therapeutics’ Parkinson’s disease program
Financings
Harness Therapeutics Ltd. has been awarded a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance its Parkinson’s disease program targeting glucocerebrosidase (GCase) and lysosomal integral membrane protein-2 (LIMP2) in GBA1-Parkinson’s disease.
Gen1e Lifesciences receives US FDA designations for GEn-1123
Regulatory
Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD).

Endocrine/metabolic

Ractigen’s saRNA therapy delivers sustained fat loss while preserving muscle
Conferences
Researchers from Ractigen Therapeutics Co. Ltd. presented preclinical data on LiCO-saUcp1, a small activating RNA (saRNA) therapeutic designed to upregulate UCP1 expression in adipose tissue.

Biomarkers

Pre-disease plasma signature may help redefine lung cancer risk
Science
Researchers have identified a 14-protein blood signature that can predict lung cancer risk as much as five years before diagnosis, and the findings could help identify people who could benefit from preventive drugs. Published in Cell, the study was a collaboration between the Francis Crick...

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...

Gastrointestinal

Targeting TRIM54 mitigates liver injury in cirrhosis
Conferences
Researchers from Beijing University of Chinese Medicine described the role of TRIM54 in cirrhosis progression using two independent animal models of chronic liver injury – a DEN-induced rat model and a CCl4-induced mouse model of liver cirrhosis.
Dual ABCB4/ABCB11 modulation shows promise in cholestatic disease models
Conferences
Researchers from Rectify Pharmaceuticals Inc. presented preclinical efficacy data on RTY-406, a novel dual-acting ABCB4/MDR3 and ABCB11/BSEP modulator, in animal models of primary sclerosing cholangitis.
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...
TAK1 emerges as a therapeutic target in MASLD
Conferences
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...
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...
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.

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

Recent episodes:

View all

BioWorld

The news source of record covering the development of innovative human therapies for 25+ years

BioWorld Asia

A weekly monitor of biopharmaceutical news from the industry’s fastest-growing region

BioWorld Science

Essential discovery and preclinical research news to support crucial drug R&D decisions at the earliest stages

BioWorld Premium

Enjoy extended coverage for the most complete market view with BioWorld, BioWorld Science and BioWorld Asia in a single, easy to access subscription

Today's news in brief

BioWorld

BioWorld Asia

BioWorld Science

BioWorld Premium