NuCyRNA TDP-43 dual-targeting oligonucleotide

NuCyRNA Therapeutics is developing a dual-targeting oligonucleotide platform for the treatment of Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and other central nervous system (CNS) disorders. This preclinical program focuses on the TDP-43 (TAR DNA-binding protein 43) pathology, which is characterized by both the toxic aggregation of TDP-43 in the cytoplasm and its depletion from the nucleus (loss-of-function). The dual-targeting mechanism is designed to simultaneously knock down modifier genes that exacerbate TDP-43 proteinopathy and correct specific loss-of-function phenotypes, such as cryptic splicing errors in genes like STMN2 or UNC13A. Developed in collaboration with researchers from Northwestern University and UMass Chan Medical School, the therapy aims to provide a comprehensive approach to neurodegeneration by addressing multiple facets of TDP-43 dysfunction.

Other names

NuCyRNA TDP-43 programTDP-43 dual-targeting oligonucleotideTDP43 dual-targeting oligonucleotideTDP 43 dual-targeting oligonucleotide