Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy.
Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy.
CCNK is frequently upregulated in diverse tumor types, where its increased expression promotes proliferation and its depletion triggers apoptosis. Researchers from Guangdong Pharmaceutical University reported the discovery and preclinical characterization of a novel series of CCNK molecular glue degraders with antitumor activity.
Abion Inc. has presented data for ABN-202, a hyperglycosylated interferon (IFN) β mutein fused to a monoclonal antibody targeting tumor-associated calcium signal transducer 2 (TROP2) that retains activity under acidic conditions.
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted orphan drug, rare pediatric disease and fast track designations to LTS-101.
Theratechnologies Inc. and Transfert Plus SEC have disclosed peptide-drug conjugates comprising a maytansinoid moiety linked to peptides targeting the sortilin (neurotensin NTR3; NT3; Gp95) receptor through a protease-cleavable linker reported to be useful for the treatment of cancer.
Vectory Therapeutics BV has obtained IND clearance from the FDA for a phase I/II trial (PIONEER-ALS) of VTX-002, a first-in-class vectorized antibody targeting TDP-43 pathology in amyotrophic lateral sclerosis (ALS). VTX-002 delivers an engineered antibody designed to selectively target toxic species of TDP-43.
In a study published in Molecular Therapy, researchers from H. Lee Moffitt Cancer Center and Research Institute and the University of South Florida developed an E6-targeting proteolysis targeting chimera (PROTAC) that inhibits the growth of human papillomavirus (HPV)-positive tumors. HPV is the most prevalent sexually transmitted infection worldwide, with persistent infections causing up to six different types of cancer.
T cell-engaging bispecific antibodies (TCEs) are engineered molecules designed to bring cytotoxic T cells into proximity with tumor cells, triggering a targeted, antigen-dependent immune attack. However, TCE may activate T cells in healthy tissues, leading to off-tumor toxicity.
IL-2 is clinically validated as an immunotherapeutic, but its use is limited by toxicity issues. AZD-6750 is an approach from Astrazeneca plc that applies cis-guiding to deliver a modified IL-2 mutein to CD8+ T cells.
Treatment in glioblastoma usually fails due to tumor heterogeneity and persistence of glioblastoma multiforme stem-like cells (GSCs) within the tumor margin. Researchers from Trogenix Ltd. engineered TGX-007, an AAV1-mediated therapeutic that delivers both cytotoxic and immunomodulatory genetic...
Triple-negative breast cancer (TNBC) is a highly aggressive subtype affecting 15%-20% of breast cancer patients. TNBC patients harboring breast cancer susceptibility gene 1/2 (BRCA1/2) mutations have shown improved therapeutic response to poly(ADP-ribose) polymerase (PARP) inhibitors (PARPi).
2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS...
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work uncovers a novel etiology of ulcerative colitis....
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms.
Inhibiting transient receptor potential melastatin 2 (TRPM2) may mitigate injury arising from ischemic stroke. Researchers at Zhejiang University and its medical school previously reported a small-molecule inhibitor of TRPM2, which also inhibited the human ether-a-go-go-related gene (hERG) channel,...
Brenig Therapeutics Inc. has described leucine-rich repeat kinase 2 (LRRK2; dardarin) and (LRRK2; dardarin) (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that...
Biohaven Therapeutics Ltd. has synthesized pyruvate kinase M2 (PKM2) activators reported to be useful for the treatment of age-related macular degeneration, amyotrophic lateral sclerosis, multiple sclerosis, retinitis pigmentosa and fibrosis.
Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Suzhou Vigonvita Life Sciences Co. Ltd. have described NMDA receptor antagonists and/or monoamine transporter inhibitors reported to be useful for the treatment of neurological disorders.
The canonical G protein-coupled receptors (GPCRs) cannabinoid CB1 (CB1R) and CB2 (CB2R) receptors are the most studied in the endocannabinoid system. CB1R is widely distributed throughout the CNS, but is also present on peripheral tissues.
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the...
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH)...
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.
Numab Therapeutics AG and Kaken Pharmaceutical Co. Ltd. have entered into a strategic licensing and co-development agreement for NM-81, a multi-specific antibody for the treatment of inflammatory bowel disease (IBD).
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