Dicerna’s novel, next generation approach to developing RNA interference (RNAi)-based therapies and delivery systems is based on its leading-edge Dicer Substrate Technology™ platform and Dicer Substrate siRNA (DsiRNA) molecules. Dicerna’s platform differentiates the company from other RNAi approaches, and as a result, uniquely positions Dicerna to realize the promise of RNAi therapeutics. The company’s internal research and development program is focused in oncology. In addition, Dicerna has active research and development programs focused on the therapeutic areas of oncology, endocrinology, immunology and inflammation with its pharmaceutical partners Kyowa Hakko Kirin and Ipsen.
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Dicerna believes that its Dicer Substrate Technology offers significant advantages over earlier RNAi technologies, in terms of potency and longevity of gene silencing, achieved in in vitro and in vivo experiments to date, and in its enhanced delivery potential. The company’s DsiRNA therapeutic oligonucleotides are designed with different structures from first generation siRNAs – specifically, Dicerna’s DsiRNA molecules are 25 or more base pairs in length and are processed by the Dicer enzyme. The discovery of the ability to engage the Dicer enzyme, which operates early in the gene silencing cascade with beneficial downstream effects, has opened a second proprietary doorway into the RNAi intellectual property space, in addition to the functional benefits over conventional, shorter siRNA molecules.
There are additional benefits to both pharmaceutical features and intellectual property that arise from the enhanced delivery properties of DsiRNA molecules compared to conventional siRNA duplexes. DsiRNA molecules have enhanced delivery potential because their longer length, and the specific cleavage activity of Dicer creates a natural conjugation point for cellular targeting agents. Dicerna has the ability to extensively chemically modify and/or link DsiRNAs directly to other classes of pharmaceutical molecules to broadly improve biodistribution and cellular delivery, such as aptamers, peptides, antibodies and small molecules. A significant strength of Dicerna’s DsiRNA platform is its suitability for pharmaceutical modifications to allow direct administration of DsiRNA molecules conjugated to targeted delivery moieties without sacrificing gene silencing activity.
Dicerna aims to leap-frog current RNAi delivery paradigms by developing DsiRNA molecules that have greatly improved pharmacokinetic and biodistribution properties, to ultimately achieve cell- or tissue-specific targeted delivery. Dicerna believes this new generation of drug delivery strategies is well-positioned to overcome several of the limitations of the earlier RNAi approaches.