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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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Drug R&D concept image.
Endocrine/metabolic

Cdk5 inhibitor shows anti-diabetic and neuroprotective effects in type 2 diabetes

April 11, 2025
In a newly published study, researchers from the CSIR Institute of Genomics and Integrative Biology and collaborators further explored the potential of Cdk5 targeting as a therapeutic approach for type 2 diabetes and cognitive deterioration.
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Colorful illustration of reconstructed brain cells
Neurology/psychiatric

Mouse brain structure and function shown on high-resolution map

April 11, 2025
By Mar de Miguel
Scientists from the Machine Intelligence from Cortical Networks (MICRONS) consortium have published the microconnectome of a cubic millimeter of the mouse brain. This is the most complete map of this organ to date at nanometer resolution for a mammal. It not only contains the structure and connections of each and every cell in that volume of tissue, but is also linked to the neuronal activity of that portion of the CNS, linking anatomy and function in the same cells.
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Neurology/psychiatric

ROCK2 inhibitor-loaded vesicles restore mitochondrial function and autophagy in PD mice

April 10, 2025
In Parkinson’s disease (PD), damaged mitochondria build up in the dopaminergic system of the substantia nigra, partly due to impaired mitochondrial autophagy (mitophagy) and autophagosome accumulation.
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Neurology/psychiatric

NDC-0524 reverses synucleinopathy progression by targeting nitrated α-synuclein

April 10, 2025
Nitrated α-synuclein is upregulated in the CSF of patients with Parkinson’s Disease (PD), Lewy body dementia (DLB), multiple system atrophy (MSA), and other synucleinopathies.
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Tau protein in cell model of tauopathy
Neurology/psychiatric

VY-1706 shows strong activity across multiple models of tauopathy

April 10, 2025
Researchers from Voyager Therapeutics Inc. presented preclinical activity data of VY-1706, a blood-brain barrier (BBB)-penetrant gene therapy comprising an adeno-associated virus serotype 9 capsid (AAV9-C9P39) vector encoding primary artificial microRNA (pri-amiRNA) consisting of short-interfering RNA (siRNA) targeting human microtubule-associated protein tau (MAPT) protein.
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Endocrine/metabolic

Improved vector enables lower dosing for lysosomal storage disorder

April 10, 2025
Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children.
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Drug design, drug delivery & technologies

Poly(A) tail mimetics boost gene expression in haploinsufficiency models

April 10, 2025
Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.
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Brain with puzzle piece removed
Neurology/psychiatric

SAR-446159 shows potential for α-synucleinopathy treatment

April 9, 2025
Researchers from Sanofi SA reported the preclinical characterization of SAR-446159 (ABL-301), a bispecific antibody construct comprising an antibody targeting α-synuclein fused to an engineered antibody fragment that targets IGF-1R and functions as a blood-brain barrier (BBB) shuttle, known as the Grabody-B platform.
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Neural network
Neurology/psychiatric

First-in-class peptide successfully rescues several neurodegenerative phenotypes

April 9, 2025
BDNF is the brain’s most abundant neurotrophic factor, playing a key role in neuronal survival and synaptic plasticity through the activation of the transcription factor CREB, which is essential for driving beneficial effects in neurons. CREB is downregulated in Parkinson’s disease, Huntington’s disease, frontotemporal dementia, Alzheimer’s disease and other neurodegenerative conditions.
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Neurology/psychiatric

ALIA-1758 clears Aβ aggregates via TfR-mediated transcytosis

April 9, 2025
Transferrin receptor (TfR)-mediated transcytosis is a receptor-mediated mechanism for drug delivery to the brain or other tissues, where, after the antibody binds to transferrin, the therapeutic agent is internalized and released into the brain.
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