The University of California has synthesized α-synuclein (SNCA) and/or amyloid-β protein and/or microtubule-associated protein tau (PHF-tau; MAPT) propagation inhibitors reported to be useful for diagnosis and treatment of multiple system atrophy, Parkinson’s disease and Alzheimer’s disease.

Shandong Quanzhong Biomedical Technology Co. Ltd. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of atherosclerosis, obesity, cancer, osteoarthritis, inflammatory bowel disease, fibrosis, psoriasis and Alzheimer’s disease.

The FDA has granted orphan drug designation to FRF-001, the FOXG1 Research Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of rare pediatric disease designation to the investigational therapy.

Convelo Therapeutics Inc. has presented data on their 3-β-hydroxysteroid-Δ8,Δ7-isomerase (EBP) inhibitor CVL-1001 as a remyelinating compound for treating multiple sclerosis.

Chineses researchers investigated the relationship between formyl peptide receptor 1 (FPR1) expression and neurodegeneration in multiple sclerosis (MS), specifically evaluating the therapeutic potential of the FPR1 antagonist T-0080.

Tevard Biosciences Inc. has presented new preclinical data on the use of therapeutic suppressor tRNAs (suptRNAs) for the treatment of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM). The data show potent restoration of full-length functional proteins in models of DMD and DCM caused by titin truncations (DCM-TTNtv).

Hager Biosciences LLC has identified orexin receptor and/or κ-opioid receptor dual antagonists reported to be useful for the treatment of cancer, psychiatric, neurological and cardiovascular disorders.