Dtx Pharma Inc.’s Dtx-1252, an investigational small interfering RNA (siRNA) therapeutic, has been awarded U.S. orphan drug designation for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).

Sanofi SA has prepared and tested isoxazolidines acting as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors. As such, they are reported to be useful for the treatment of Alzheimer’s disease, multiple sclerosis, amyotrophic lateral sclerosis and incontinentia pigmenti.

Two Vigil Neuroscience Inc. patents describe triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of frontotemporal dementia, multiple sclerosis, rheumatoid arthritis, stroke, prion infections, Parkinson’s, Alzheimer’s and Nasu-Hakola diseases.

Psychedelic drugs could have two distinct overlapping effects based on their affinity for two receptors involved in neuronal plasticity. Scientists at the University of Helsinki have observed that lysergic acid diethylamide (LSD) and psilocin (a drug isolated from the mushroom Psilocybe mexicana) have an antidepressant effect that is independent of their hallucinogenic outcome. These two different pathways are established through their binding to the tyrosine kinase receptor (TrkB), which has an antidepressant nature, or to the serotonin receptor, with a hallucinogenic activity.