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.
Pathological tau accumulation is a key driver of neurodegeneration in Alzheimer’s disease (AD) and other tauopathies such as progressive supranuclear palsy. Researchers at Alnylam Pharmaceuticals and Regeneron Pharmaceuticals Inc. presented preclinical efficacy data on ALN-5288 in tauopathy models.
Rumblings that psychedelic treatments would soon be ready for prime time gained credence aplenty, and shares of Ataibeckley Inc. (NASDAQ:ATAI) ended July 16 at $7.15, up $1.79, or 33%, as Wall Street learned that Eli Lilly and Co. is taking over the psychedelics firm for $6.75 per share in cash plus up to $2.50 per share in the form of a contingent value right related to milestones for two Ataibeckley compounds.
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.
Nuoshen Pharmaceutical (Shanghai) Co. Ltd. has disclosed new NLRP3 inflammasome inhibitors potentially useful for the treatment of neuroinflammation, multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s, Parkinson’s and Huntington’s disease.
FTO is an α-ketoglutarate-dependent RNA demethylase that regulates N6-methyladenosine (m6A), one of the most abundant epitranscriptomic modifications in mammalian mRNA. Emerging evidence suggests that aberrant m6A signaling may contribute to neuronal dysfunction and neurodegeneration. Researchers at the University of Barcelona and collaborators described the discovery and preclinical characterization of a new series of FTO inhibitors designed to target neurodegenerative disease by modulating m6A RNA demethylation.
At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD).