PMN267 (ProMIS Neurosciences)

ProMIS Neurosciences is developing TDP-43-targeting intrabodies, with PMN267 as the lead candidate, for the treatment of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and limbic-predominant age-related TDP-43 encephalopathy (LATE). These intrabodies are designed to be delivered via gene therapy vectors to target and degrade toxic, misfolded aggregates of TAR DNA-binding protein 43 (TDP-43) within the cytoplasm of neurons. Crucially, the therapy is engineered to be highly selective for the pathogenic forms of TDP-43 while sparing the normal, functional protein required for cellular survival. Preclinical data has demonstrated that these intrabodies can co-localize with mislocalized TDP-43 and reduce aggregate levels in iPSC-derived motor neurons under chronic stress conditions.

Other names

TDP-43 intrabodyTDP43 intrabodyTDP 43 intrabodyProMIS TDP-43 intrabody