Plan to initiate dosing of FTD-C9orf72 patients in 1H 2025 FTD-C9orf72 is estimated to affect approximately 21,000 individuals in the United States and Europe. Similar to FTD-GRN, FTD-C9orf72 is marked by TDP-43 pathology, where the transactive response DNA binding protein 43 (TDP-43) abnormally accumulates in the cytoplasm of neurons, leading to neuronal dysfunction and degeneration. Third-party preclinical studies have demonstrated that increasing PGRN levels can reduce TDP43 pathology and slow neurodegen ...