Integration of Lead Optimization Data for Candidate Selection and Development

5 INTEGRATION OF LEAD OPTIMIZATION DATA FOR CANDIDATE SELECTION AND DEVELOPMENT 

Final candidate selection for the development of an RNAi therapeutic integrates safety-based information from in silico target assessments, siRNA design features, and traditional screening in cell-based systems and rodent 

TABLE 4.1 A Comparison of Drag Properties among Small Molecules, Oligonucleotides, and Biologies 

Manufacture Chemical synthesis Chemical synthesis Biotechnology driven 

Structure (drug Single entity of high purity Single entity of high purity Complex entity heterogeneity 

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