Researchers from the Universities of Bologna and Torino recently presented their hematopoietic stem cell gene therapy (HSC-GT) strategy based on microglia-mediated delivery using a lentiviral vector encoding a secretable, cell-penetrating CDKL5 protein (Igκ-TATk-CDKL5).

Researchers from Ajou University School of Medicine reported the preclinical efficacy of KBN-2201 in a mouse model of late-stage Alzheimer’s disease.

The NIH Accelerating Medicines Partnership Program for Alzheimer’s disease (AD) has named tyrosyl-tRNA synthetase (TyrRS, YARS1) as a therapeutic target in AD. Functional Longevity Labs Inc. has developed FLL-001, a potential first-in-class small-molecule TyrRS modulator designed to restore TyrRS levels and rescue its triad in post-mitotic neurons for the treatment of AD.

Researchers from the University of Strasbourg and the Centre National de la Recherche Scientifique (CNRS) have presented data on LIT-002, a nonpeptide oxytocin receptor (OXTR) agonist, for the potential treatment of autism and neuropathic pain.

Anle-138b (emrusolmin) is a diphenylpyrazole compound that directly interacts with oligomeric species of disease-related aggregating proteins, reducing aggregate formation and toxicity. It has shown benefit in preclinical models of prion disease, Parkinson’s disease, multiple system atrophy and Alzheimer’s disease.

Reduced endocannabinoid signaling has been linked to neuronal hyperexcitability in Alzheimer’s disease (AD), suggesting that dual inhibition of fatty acid amide hydrolase (FAAH) and monoacylglycerol lipase (MAGL) may restore endocannabinoid tone and alleviate hyperexcitability-driven symptoms, including agitation, a common neuropsychiatric manifestation of AD.

Pyroglutamate-modified amyloid-β (pE3-Aβ) is a highly pathogenic Aβ species that accumulates within amyloid plaques and contributes to aggregation, neuroinflammation and neuronal dysfunction in Alzheimer’s disease (AD). Researchers from Sound Biologics (Qilu Puget Sound Biotherapeutic Corp.) described the preclinical profile of PSB-229, a novel brain-shuttled anti-pE3-Aβ antibody in models of AD.