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To advance and support innovative small molecule and biologic (antibodies, oligonucleotides, peptides, gene therapy) drug discovery programs for FTD. Current funding priorities for this program include:
Lead optimization of novel disease-modifying compounds (e.g., medicinal chemistry refinement, in vitro ADME)
In vivo testing of novel compounds, biologics, or repurposed drugs in relevant animal models for pharmacokinetics, dose-range finding, target engagement, in vivo efficacy and/or preliminary rodent tolerability studies.