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BioWorld - Sunday, June 15, 2025
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Neurology/psychiatric

Preclinical profile of LY-3954065, a pioneering siRNA for Alzheimer’s disease

April 16, 2025
Researchers from Eli Lilly & Co. reported preclinical data on LY-3954068, a MAPT small-interference RNA (siRNA) strategy in AD models. The therapy is designed to reduce tau protein synthesis by inhibiting the translation of MAPT mRNA, with the goal of effectively lowering all forms of tau.
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Illustration of antibodies attacking nerve cells
Immune

Biohaven’s BHV-1310 shows promise for autoimmune disorders

April 16, 2025
Biohaven Pharmaceuticals Inc. has presented preclinical data on their novel bispecific degrader BHV-1310 for the potential treatment of autoimmune disorders. To date, about 10% of the global population is affected by autoimmune diseases. IgG-lowering agents have suboptimal pharmacology and pharmacodynamic effects and have safety and tolerability challenges.
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Vaccine vial and syringe
Neurology/psychiatric

β-Glucan vaccine outperforms standard immunization approaches

April 15, 2025
Researchers from Tridem Bioscience GmbH & Co. KG provided details on the development of TRB-001, an anti-α-synuclein construct consisting of a B-cell epitope derived from human α-synuclein (SNCA), conjugated to CRM197 and C-type lectin (CLEC) β-glucan.
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Neurology/psychiatric

FKBP51 induces astrogliosis and seizures through NF-κB signaling, study shows

April 15, 2025
The etiology of epilepsy, as well as its pathology, still remains evasive. The role that FK506-binding protein 51 (FKBP51) might play in this disease was investigated in a murine model of kainic acid-induced excitotoxic brain injury.
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Parkinson's disease illustration showing neurons containing alpha-synuclein
Neurology/psychiatric

AC Immune unveils first-in-class Morphomer targeting pathological α-synuclein

April 15, 2025
Researchers from AC Immune SA recently presented the discovery of ACI-21018, a novel α-synuclein aggregation inhibitor developed using AC Immune’s proprietary Morphomer platform.
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Neurology/psychiatric

PTG-630 improves cognitive deficits in Alzheimer’s mouse models

April 15, 2025
Praetego Inc. and University of California San Diego scientists reported preclinical results for the new small-molecule amadorin PTG-630, a brain-penetrant drug candidate that inhibits the formation of advanced glycation end-products (AGEs).
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Illustration of head with maze that is missing parts
Neurology/psychiatric

Neurizon’s NUZ-001 confers neuroprotection in preclinical neurodegeneration models

April 14, 2025
Neurizon Therapeutics Ltd. has presented preclinical data on their compound NUZ-001 for the potential treatment of neurological disorders.
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Tau neuron illustration
Neurology/psychiatric

Novel strategy for treating 4-repeat tauopathies unveiled

April 14, 2025
The specific tau isoforms, such as 3-repeat (3R) and 4-repeat (4R) isoforms, and the distinct conformational strains that misfolded tau can adopt are determinants of the molecular and clinical heterogeneity observed across tauopathies.
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Concept art for targeting cancer
Cancer

DCN1 inhibitor TK-59 shows antitumoral activity in preclinical setting

April 10, 2025
Neddylation is a post-translational modification that conjugates the NEDD8 protein to protein substrates, such as the cullins, which once neddylated join complex to form cullin-RING ubiquitin E3 ligases (CRLs), which in turn play a crucial role in regulating proteasomal degradation. The University of Kentucky has presented preclinical data on their neddylation inhibitor TK-59 as a cancer therapeutic.
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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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