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BioWorld - Friday, May 15, 2026
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Home » Topics » BioWorld Science, Neurology/psychiatric

BioWorld Science, Neurology/psychiatric
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Neurology/psychiatric

DMD base editing candidate shows safety and efficacy in preclinical models

March 20, 2025
At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).
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Illustration of DNA, magnifying glass
Neurology/psychiatric

Quiver Bioscience collaborates with Dup15q Alliance to advance ASOs for Dup15q syndrome

March 20, 2025
Quiver Bioscience Inc. is collaborating with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome.
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Test tubes, dropper
Inflammatory

Domain Therapeutics nominates biased antagonist of PAR2 as drug candidate for inflammatory diseases

March 20, 2025
Domain Therapeutics SA has nominated PAR2 antagonist DT-9046 as a drug candidate with potential to treat various inflammatory diseases, including atopic dermatitis, inflammatory bowel disease and arthritis, as well as neuroinflammatory conditions such as migraine.
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Neurology/psychiatric

US and Canadian researchers describe new PARP-1 inhibitors

March 18, 2025
Scientists at St. John’s University, The University of Pennsylvania and Université de Montréal have identified poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment of cancer, Lewy body dementia, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
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Elderly hands holding broken brain structure
Neurology/psychiatric

GSK-3β inhibitor demonstrates efficacy in mouse model of Alzheimer’s disease

March 18, 2025
In work conducted by Jining Medical University investigators, synthesis and optimization of a previously reported ATP-competitive pyrrolo[2,3-b]pyridine-based glycogen synthase kinase 3β (GSK-3β) inhibitor led to the discovery of compound [I], a candidate with significant inhibitory activity on GSK-3β (IC50=0.35 nM), with its inhibitory effect being approximately 12-fold greater than that of broad-spectrum GSK-3β inhibitor staurosporine and 189-fold greater than that of the parent compound.
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Neurology/psychiatric

Academic researchers describe new κ-opioid receptor antagonists

March 17, 2025
Scientists at Medical University of Vienna, The University of Queensland and Universität Innsbruck have identified cysteine stapled dynorphin A (CSD) analogues acting as κ-opioid receptor antagonists reported to be useful for the treatment of anxiety disorders, depression, substance abuse and dependence, and stress disorders.
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Illustration of man holding magnifying glass to human body model showing muscle anatomy
Neurology/psychiatric

New in vitro model of inclusion body myositis based on human iPSC-myotubes

March 17, 2025
Inclusion body myositis (IBM) is an inflammatory myopathy causing proximal and distal muscle weakness. IBM’s cause remains unknown, lacking validated models, biomarkers and effective treatment strategies. Histopathological studies identified inflammatory infiltrates, rimmed vacuoles, and mitochondrial changes in the muscles of IBM patients.
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Brain maze
Neurology/psychiatric

Iqure’s EAAT2 modulator gains IRB clearance for first-in-human study

March 17, 2025
Iqure Pharma Inc. has received institutional review board (IRB) approval for a first-in-human study of iQ-007 in healthy volunteers. This follows recent formal approval from the Human Research Ethics Committee (HREC) in Australia for the phase I study, which is expected to begin next month.
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Cross section illustration of ion channel in cell membrane
Drug design, drug delivery & technologies

Series A financing at Maxion Therapeutics to advance Knotbodies for ion channel and GPCR-driven diseases

March 17, 2025
Maxion Therapeutics Ltd. has raised $72 million (£58 million) in a series A financing to support its development of antibody-based Knotbody drugs for ion channel- and G protein-coupled receptor (GPCR)-driven diseases.
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Epstein-Barr virus 3D illustration
Immune

Genes and Epstein-Barr virus immune response increase MS risk

March 17, 2025
By Mar de Miguel
A combination of Epstein-Barr virus (EBV) antibodies and genetic factors may be linked to an increased risk of multiple sclerosis (MS), according to a study led by scientists at Karolinska Institutet and Stanford University. “The Epstein-Barr virus has been a suspect for many years for having a role in causing MS. The evidence for it has increased though one has not really reached complete proof of its role,” Tomas Olsson told BioWorld.
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