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Jan 15, 2010 (Vol. 30, No. 2)

RNAi Moves One Step Closer to Clinic

Researchers Tout Delivery Strategies that Have the Potential to Get New Drugs into Trials Sooner

  • Dicer Substrate Technology

    Double-stranded RNA (dsRNA) triggers a series of biochemical events that culminates in sequence-specific suppression of gene expression. Long dsRNAs were used for years as a means to modulate gene expression in plants, yeast, and C. elegans. Rapid advances in smaller, synthetic RNA molecules over the last two years have led to the initiation of several RNAi clinical trials.

    Dicerna Pharmaceuticals is working with Dicer-substrate siRNAs (DsiRNA). Dicer is a critical enzyme involved in the RNAi gene-silencing cascade. Dicer processing of double-stranded RNA oligonucleotides of 25 or more base pairs in length and transfer of the cleaved product RNA to the gene-silencing complex results in five- to tenfold more potent activity and longer duration of action.

    “Our RNAi payloads are dramatically more efficient,” says Bob D. Brown, Ph.D., senior vp of research. “The challenge across the RNAi therapeutics field has been delivery. DsiRNAs offer a flexible platform for modifications that dovetail with a wide range of delivery technologies and mechanisms. We will exploit this feature to create successful therapeutics.”

    Early last year, Dicerna secured the exclusive, worldwide right to grant sublicenses to the DsiRNA intellectual property estate in-licensed by Dicerna. Its pipeline of RNAi-targeted drugs and delivery systems is focused primarily in the therapeutic areas of oncology and metabolic diseases. In addition, it expects to use the Dicer Substrate technology in several other therapeutic areas such as inflammation, immunology, and cardiovascular diseases.


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