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Dicerna Technology: Next Generation RNAi and RNAi Delivery

Dicerna is a leading developer of RNAi drugs targeting unmet needs in cancer. The company’s novel Dicer Substrate siRNA (DsiRNA) molecules and proprietary drug delivery technologies have the demonstrated ability to silence previously undruggable disease targets. Dicerna’s RNAi platform is comprised of two key components: Dicer Substrates (DsiRNAs), which are 25-30 asymmetric double stranded RNAs that mediate RNAi with extremely high potency, and EnCore lipid nanoparticles, which are optimized for tumor delivery of RNAi molecules. The DsiRNA platform is broadly covered by the recently issued US patent 8,084,599.

Dicerna DsiRNA and Delivery Technologies enable breakthrough cancer treatments based on previously undruggable targets

Robust in vivo anti-tumor efficacy has been achieved in multiple difficult-to-treat tumors using DsiRNA Technology against classic undruggable oncology targets, both within our own pipeline and with our partner Kyowa Kirin. The company’s most advanced program targets the MYC oncogene and is focused on Hepatocellular Carcinoma (HCC), where there still remains a significant need for new treatment options. The HCC program is available for partnering.

Recent accomplishments

·         The demonstration of profound tumor responses in multiple solid tumor xenograft models.

·         The recent announcements that our partner, Kyowa Kirin, has moved the initial oncology candidate into formal development, triggering a substantial success milestone and the achievement of the in vivo efficacy milestone for the second DsiRNA oncology candidate.

·         The characterization and optimization of our EnCore lipid nanoparticle (LNP) technology and its ability to deliver DsiRNA payloads into the cell cytoplasm and achieve efficient knockdown of targeted genes.

·         The issuance of the fundamental patent covering the DsiRNA platform.

At Dicerna we can rapidly generate DsiRNA inhibitors against any gene and optimize our EnCore delivery technology to deliver to specific tumor types or organs.  We have demonstrated these capabilities in our lead HCC program, available for partnering, and can use these capabilities to develop therapeutics against targets of interest to collaborators.  I hope you have the time to meet with Dicerna during BioNetwork West to discuss our technology and areas of possible mutual interest.

The Company is a biopharmaceutical company that is developing and commercializing innovative medicines to target unmet therapeutic needs in arthritis, pain and inflammatory diseases. The Company has two FDA approved products: DUEXIS® and RAYOS®.  DUEXIS®s is a proprietary tablet formulation containing a fixed-dose combination of ibuprofen and famotidine in a single pill.  DUEXIS is indicated for the relief of signs and symptoms of rheumatoid arthritis (“RA”) and osteoarthritis and to decrease the risk of developing upper gastrointestinal ulcers in patients who are taking ibuprofen for these indications. In June 2012, the Company began expanding its commercial organization and expects to almost double its original field force size by the end of the third quarter of 2012, to approximately 150 field sales representatives. Also in June 2012, the Company engaged Mallinckrodt LLC, the pharmaceutical business of Covidien plc, on a non-exclusive basis to co-promote DUEXIS in the U.S. and entered into an exclusive collaboration, license and supply agreement with Grünenthal S.A. for the potential commercialization of DUEXIS in Latin America.

The Company’s second product, RAYOS®, known as LODOTRA® outside the U.S., is a proprietary delayed release formulation of low-dose prednisone that is currently marketed in Europe by its distribution partner, Mundipharma International Corporation Limited (“Mundipharma”), for the treatment of moderate to severe, active RA in adults, particularly when accompanied by morning stiffness. In addition, the Company has granted to Mundipharma commercialization rights to LODOTRA in Asia and Latin America. On July 26, 2012, the FDA approved RAYOS for the treatment of a broad range of diseases, including RA, polymyalgia rheumatica (“PMR”), psoriatic arthritis (“PsA”), ankylosing spondylitis (“AS”), asthma and chronic obstructive pulmonary disease (“COPD”). The Company anticipates the launch of RAYOS in the United States in the fourth quarter of 2012. The Company’s strategy is to commercialize its products in the U.S. and to enter into licensing or additional distribution agreements for commercialization of its products outside the U.S.

Based in Toronto, Canada, DRI Capital Inc. is a life sciences industry-focused investment management firm with over US$2 billion under management that purchases drug royalties from universities, inventors, research institutes and pharmaceutical and biotechnology companies.  DRI Capital closed Drug Royalty II in April, 2010 with US$701 million in equity capital commitments, and a Co-Investment Fund in December, 2010 with an additional US$225 million in equity capital commitments.  Investors in Drug Royalty II and the Co-Investment Fund include corporate entities, public pension plans, 501(c)(3) entities, family offices and a sovereign wealth fund. Royalties recently acquired by DRI’s funds include those on Johnson and Johnson’s Remicade, Amgen and Pfizer’s Enbrel, Kyowa Kirin’s Regpara, and Biogen Idec and Elan’s Tysabri. 

AlloCure is a Boston-based biotechnology company developing AC607, an allogeneic mesenchymal stem cell therapy for the treatment acute kidney injury (AKI) with potential applications in other grievous illnesses. 

Building on the success of a phase 1 clinical trial, AlloCure has initiated ACT-AKI, a randomized, multicenter, double-blind, placebo-controlled phase 2 trial in cardiac surgery subjects with post-operative AKI. Currently, there are no available treatment options for patients with AKI other than supportive care. AC607 represents the first application of a cell therapy to alter the course of this life threatening condition.