DDP 2010: Case Studies: Creating Novel Therapeutics with Superior Potency

Case Studies: Creating Novel Therapeutics with Superior Potency, Duration of Action and Unique Delivery Potential by Harnessing Second Generation Dicer Substrate RNAs (DsiRNAs)
Sujit K. Basu, PhD
Senior Director, Formulation
Dicerna Pharmaceuticals, Inc.

siRNA Delivery Options
Flexible delivery of DsiRNAs
Multiple delivery options:

->chemically stabilized DsiRNA ->IDDS Functionally conjugated -> Nanoparticle Lipids, Polymers->

DsiRNA uses natural gene silencing pathway – optimized for mammalian cells
-Stringest screening criteria and molecular architecture produce highly specific DsiRNAs,
-High potency low dose, long duration of action (infrequent dosing observed)
-Immunostimulatory events can be abrogated by appropriate chemical modification

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