Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.
Mercury Bio Inc. has released promising results from a preclinical study that successfully utilized its extracellular vesicle (EV) drug encapsulation platform (yEV) to deliver proteins, in the form of nanobodies, into neurons across the blood-brain barrier. The yEV platform utilizes yeast-derived exosomes, a subtype of EV.
Pop Biotechnologies Inc. has been awarded a $2.46 million grant by the National Institutes of Health (NIH) to pursue development of a ‘mosaic’ active immunotherapy against Alzheimer’s disease.
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).
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