Ultrarare transmitted Alzheimer’s cases give clues to common forms
Investigators have identified five cases of so-called iatrogenic Alzheimer’s disease (AD), that is, AD that was acquired as a result of undergoing medical procedures. A team led by University College London scientists reported their findings online in Nature Medicine on Jan. 29, 2024.

Ultrarare transmitted Alzheimer’s cases give clues to common forms
Investigators have identified five cases of so-called iatrogenic Alzheimer’s disease (AD), that is, AD that was acquired as a result of undergoing medical procedures. A team led by University College London scientists reported their findings online in Nature Medicine on Jan. 29, 2024.
PDE4 prodrug exhibits colon-selective bioactivation in colitis models
Phosphodiesterase-4 (PDE4) is an intracellular pro-inflammatory enzyme that is considered a therapeutic target in inflammatory disorders such as psoriasis, asthma or ulcerative colitis (UC), among others. Researchers from Palisade Bio Inc. have reported preclinical data on PALI-2108, a PDE4 inhibitor prodrug, in models of UC.
DP Technology nominates selective Kv1.3 inhibitor as candidate for autoimmune diseases
DP Technology Ltd. has nominated DPT-0218, a novel small molecule targeting Kv1.3, as a preclinical candidate for the treatment of various autoimmune diseases, including inflammatory bowel disease and atopic dermatitis.
Scynexis’ candidate demonstrates efficacy in mucormycosis mouse model
Researchers from Scynexis Inc. have recently presented details on the preclinical characterization of SCY-247, a second-generation triterpenoid with glucan synthase inhibitor activity and intended to be used for the treatment of invasive mucormycosis.
Synthekine and Sanofi collaborate on IL-10 receptor agonists for inflammatory diseases
Synthekine Inc. has established a worldwide collaboration with Sanofi SA to develop and commercialize IL-10 receptor agonists for the treatment of inflammatory diseases.
Zentaur Therapeutics patents new USP1 inhibitors for cancer
Zentaur Therapeutics USA Inc. has disclosed ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
GPR3 activation in Kupffer cells inhibits liver pathology development
Kupffer cells are macrophages located in the liver that play a key role in liver pathology, concretely in metabolic dysfunction-associated steatotic liver disease (MASLD; formerly nonalcoholic fatty liver disease or NAFLD), but the mechanisms behind this are poorly understood.
Photodynamic therapy candidate for individualized tumor real-time tracing and treatment
Researchers from Shanghai Xianhui Pharmaceutical Co. Ltd. have detailed the discovery and preclinical characterization of novel photosensitizers as candidates for photodynamic therapy (PDT) and photodiagnosis in cancer.

BioWorld Insider Podcast

Six biopharma executives consider a tough 2023 and are hopeful for a better 2024.

Listen now

Conferences

Encapsulated nucleic acid therapy shows promise for sickle cell disease
Hematologic
Hemoglobinopathies affect 7% of the global population, and sickle cell disease (SCD) is a common form affecting 300,000 newborns per year.
Galectin-9 is a therapeutic target in biliary tract cancer
Cancer
The identification of novel immunotherapeutic targets for the treatment of biliary tract cancer (BTC) is still a need. It has been reported that galectin-9 (GAL-9) is expressed in BTC and induces immunosuppressive effects, such as inducing apoptosis in T cells when binding to its receptor TIM-3.
Targeting VSTM-1 using anti-VSTM-1 agonist MAb reduces COPD pathology in vivo
Respiratory
Researchers from Nextcure Inc. have reported the development and preclinical characterization of a novel agonist monoclonal antibody (MAb) against V-set and transmembrane domain containing 1 (VSTM-1).
LINC00853 may promote gastric cancer progression, study shows
Biomarkers
There is increasing interest in studying exosome molecules, and particularly long non-coding RNAs (lncRNAs), in the diagnosis of gastric cancer. Microarray analysis was performed from samples obtained from patients with gastric cancer who underwent endoscopic resection.

Cancer

Targeting TXNRD1-cGAS interaction regulates senescence-associated secretory phenotype
Inflammatory
It has been previously demonstrated that genetic variability of thioredoxin reductase 1 (TXNRD1) is associated with aging and age-associated phenotypes. Researchers from MD Anderson Cancer Center have now conducted work to assess the role of TXNRD1 in regulating tissue aging.

Infection

UBR5 is required for antiviral immunity, study unveils
Immune
When a cell is invaded by a virus, the cell triggers an innate immune response by activating retinoic acid-inducible gene I (RIG-I) like receptors (RLRs), among others, that are essential for controlling viral replication. Even though several ubiquitin ligases (E3) have been identified to...

mRNA-based vaccine design with predicted immunological response to N. fowleri
Neurology/psychiatric
Naegleria fowleri, colloquially known as the "brain-eating amoeba," is a water-borne amoeba and the leading cause of primary amoebic meningoencephalitis (PAM). In a recent study published in Scientific Reports, researchers from the University of Central Punjab aimed to design an mRNA-based vaccine...
PTR1 inhibitors based on aryl-2-nitroethyl triamino-pyrimidines as promising anti-Trypanosoma agents
To combat trypanosomiasis and leishmaniasis, researchers from the University of Modena and Reggio Emilia and collaborators conducted a study focusing on pteridine reductase 1 (PTR1), a catalytic protein crucial to the folate metabolic pathway in Trypanosomatid parasites.

Neurology/psychiatric

Alzecure nominates drug candidate from TrkA-NAM project to treat severe pain
Alzecure Pharma AB has selected a TrkA negative allosteric modulator drug candidate, ACD-137, to advance into the preclinical development phase.
Biological staging system proposed for Parkinson's disease
An international working group has proposed a new research framework that stages Parkinson's disease (PD) and defines it based on the disease's underlying biology, rather than on clinical symptoms.
Artelo’s FABP5 inhibitor relieves peripheral neuropathy
Researchers from Artelo Biosciences Inc. reported on the preclinical characterization of ART-26.12.
2024 Frontiers of Knowledge Award unfolds the chaperone story
Cancer
The discovery of how proteins fold and what happens if they don’t, or do it wrong, has impacted medicine. Protein function and several pathologies depend on folding. Four scientists, Franz-Ulrich Hartl, Arthur Horwich, Kazutoshi Mori and Peter Walter, revealed the role of chaperones in assisting...
C6-17 immunotherapy ameliorates Huntington’s features in mice
Mutant huntingtin (HTT) protein is the main cause of the pathological features leading to Huntington's disease (HD) development, a neurological disorder characterized by CAG repeat expansion in exon 1 of the HTT gene, which causes neuronal dysfunction and death along the brain.

Endocrine/metabolic

GYS1 inhibitor MZ-101 has activity in Pompe disease models
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders.

Biomarkers

Expression of miR-361-3p promotes breast cancer progression
Cancer
Increasing evidence exists suggesting plasma exosomal microRNAs (miRNAs) are involved in tumor progression, but their role in breast cancer is still unknown. Plasma exosome miRNA sequencing revealed that elevated levels of miR-361-3p were significantly correlated with malignant breast cancer...

RBMX can signal risk or protection, depending on tumor type
Cancer
Researchers from China have elucidated the prognostic role of aberrant expression of RNA binding motif protein, X chromosome (RBMX) in several cancer types using samples from cancer patients.
NAT10 expression is reduced in new-onset ankylosing spondylitis
Musculoskeletal
N-acetyltransferase 10 (NAT10) is an RNA cytidine acetyltransferase involved in multiple biological processes, such as inflammatory responses or osteogenic differentiation, and linked to pathological states such as asthma, viral infections, cancer, or laminopathies.

Gastrointestinal

Astrazeneca divulges new FAPα inhibitors for NASH
Patents
Astrazeneca AB has synthesized fibroblast activation protein-α (FAPα) inhibitors reported to be useful for the treatment of nonalcoholic steatohepatitis (NASH), among others.
V1A agonist PHIN-214 shows promise for cirrhosis-associated portal hypertension
There are several factors that induce cirrhosis. During cirrhosis, there is an increased intrahepatic resistance to blood flow, leading to portal hypertension, among others, where portal vein pressure is increased.
Targeting CTLA-4 without colitis induction is gut microbiota dependent
Cancer
Immune checkpoint inhibitors (ICIs), such as anti-CTLA-4 antibodies, are widely used in cancer immunotherapy. CTLA-4 blockers such as Yervoy (ipilimumab, Bristol Myers Squibb Co.) stimulate antitumoral immune responses, but may also induce...
Pterostilbene derivative shows activity in colitis model
Inflammatory
Researchers from Hefei University and Anhui Medical University have reported the discovery of novel nitric oxide (NO) inhibitors as anti-inflammatory agents for the treatment of acute colitis.
NLRP3 inhibitor’s efficacy and safety demonstrated in colitis models
Inflammatory
Inflammasomes are essential components of the innate immune system with a multiprotein structure.
Characterization of DONQ-52, a multispecific antibody against HLA-DQ2.5/gluten peptide complexes
Antibody
Patients with celiac disease have gluten-specific CD4+ T cells that recognize gluten via disease-associated human leukocyte antigen (HLA) molecules, specifically HLA-DQ2.5, leading to immune activation and enteropathy.

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 MedTech

Actionable and timely intelligence on advances in medical devices and technologies for 20+ 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 MedTech, and BioWorld Asia in a single, easy to access subscription

Today's news in brief

BioWorld

BioWorld MedTech

BioWorld Asia

BioWorld Science

BioWorld Premium