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BioWorld - Friday, April 24, 2026
Breaking News: Best of BioWorld Science: Q1Breaking News: Best of BioWorld: Q1Breaking News: Best of BioWorld: Q1
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Brain illustration
Neurology/psychiatric

QED-701 results in significant phenotypic improvements in models of neurodegenerative disease

Sep. 9, 2024
It has been previously demonstrated that the activation of spleen tyrosine kinase (SYK) contributes to processes that are central to the pathogenesis of neurodegenerative diseases, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD).
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Colorized transmission electron micrograph of mpox virus particles found within an infected cell.
Infection

TC Biopharm plans preclinical studies of TCB-008 to treat mpox

Sep. 9, 2024
TC Biopharm (Holdings) plc has announced plans to begin proof-of-concept preclinical studies for its lead therapeutic, TCB-008, for the treatment of mpox.
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Genetic/congenital

Gene therapy rescues multiple sulfatase deficiency in animals

Sep. 9, 2024
Multiple sulfatase deficiency (MSD) is a rare lysosomal storage disorder caused by pathogenic variants in the sulfatase modifying factor 1 gene (SUMF1). Researchers from the University of Pennsylvania described the efficacy of hematopoietic stem cell transplantation (HSCT) with ex vivo SUMF1 lentiviral gene therapy (SUMF1-GT) in a mouse model of MSD.
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Liver illustration
Infection

Novel PDE4 inhibitor shows excellent hepatoprotective activity in hepatic sepsis model

Sep. 9, 2024
Researchers from Southern Medical University have divulged the discovery and preclinical characterization of novel PDE4 inhibitors for the treatment of hepatic sepsis. Synthesis and optimization of a series of 7-alkoxybenzofurans led to the identification of compound [I] as the lead PDE4 inhibitor, with PDE4B and PDE4D IC50 values of 10.0 and 15.2 nM, respectively.
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Ovarian cancer illustration
Immuno-oncology

Bivictrix’s BVX-002 shows tumor growth inhibition and regression in murine model of ovarian cancer

Sep. 9, 2024
Bivictrix Therapeutics plc has announced promising initial in vivo data for BVX-002, its lead solid tumor bispecific antibody-drug conjugate (ADC) program. The preclinical study is investigating BVX-002 dosed once weekly, with a total of four doses given, in a murine xenograft model of ovarian cancer, using the human cell line OVCAR-3.
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Drug capsule spilling onto brain
Neurology/psychiatric

4M Therapeutics reports target engagement data for GSK-3β inhibitor 4MT-2001

Sep. 9, 2024
4M Therapeutics Inc. has released new preclinical data for its lead program, 4MT-2001, a novel small-molecule glycogen synthase kinase 3β (GSK-3β) inhibitor, demonstrating central nervous system (CNS) target engagement.
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Cancer

Darifenacin sensitizes PDAC cells to standard chemotherapy, reverses resistance

Sep. 9, 2024
Pancreatic ductal adenocarcinoma (PDAC) is still one of the most aggressive cancers, with a high mortality rate among patients. Previous findings have pointed to chitinase 3-like 1 (CHI3L1) as being behind PDAC resistance to gemcitabine, and thus a promising therapeutic target.
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T cells
Neurology/psychiatric

Autoimmune T cells may be beneficial for CNS injuries

Sep. 9, 2024
By Xavier Bofill Bruna
Immunotherapy based on T cells is the vanguard of cancer treatments. Researchers from Washington University in St. Louis have shown that similar approaches using T cells could be applied for treating injuries of the central nervous system (CNS). They reported their findings in Nature on Sept. 4, 2024.
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Other news to note for Sept. 6, 2024

Sep. 6, 2024
Additional early-stage research and drug discovery news in brief, from: Palisade Bio.
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Endocrine/metabolic

Gasherbrum Bio discovers new GLP-1R agonists for type 2 diabetes

Sep. 6, 2024
Gasherbrum Bio Inc. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes.
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