PWI-001 COMPARISON OF METHODS OF FIRST-IN-HUMAN DOSE PREDICTION FOR ADENO-ASSOCIATED VIRUS-MEDIATED GENE THERAPIES IN FABRY DISEASE F. Gibbons1, N. Boukharov2, N. Chen3, K. Sun4; 1Preclinical and Translational Sciences, Takeda Pharmaceuticals, Cambridge, MA, United States, 2Takeda Pharmaceuticals, Cambridge, MA, 3Preclinical and Translational Sciences, Takeda Pharmaceuticals, 4Data Science Institute, Takeda Pharmaceuticals.
PWI-002 A TRANSLATIONAL MODELING AND SIMULATION APPROACH TO INFORM FIRST-IN-HUMAN DOSE SELECTION FOR AN SIRNA TARGETING COMPLEMENT C3. R. Crass, A. Carvalho, A. Park, F. Gauderault, E. Richardson, K. Prem; Apellis Pharmaceuticals.
PWI-003 USE OF QUANTITATIVE MAGNETIC RESONANCE IMAGING BIOMARKERS IN CLINICAL TRIALS FOR DUCHENNE MUSCULAR DYSTROPHY: MULTIVARIATE DISEASE PROGRESSION MODELS BRIDGING TIMED MOTOR FUNCTION TESTS AND FAT FRACTION D. Yoon1, M. Daniels2, R. Willcocks3, J. Morales4, W. Triplett3, R. Belfiore-Oshan4, G. Walter5, W. Rooney6, K. Vandenborne3, S. Kim7; 1Center for Pharmacometrics & Systems Pharmacology College of Pharmacy, University of Florida, 2Department of Statistics, University of Florida, 3Department of Physical Therapy, University of Florida, 4Critical Path Institute, 5Department of Physiology and Aging, University of Florida, 6Advanced Imaging Research Center, Oregon Health & Science University, 7Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida.
PWI-004 EVIDENCE SUPPORTING EFFICACY EXTRAPOLATIONS FOR TARGETED THERAPIES ACROSS MOLECULARLY DEFINED SUBSETS OF DISEASES IN THE ABSENCE OF SUBSET-SPECIFIC CLINICAL DATA. L. Akinola, S. Ridge, M. Pacanowski, I. Zineh, A. Ramamoorthy; U.S. Food and Drug Administration.
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