We and our collaborators presented new pre-clinical data highlighting our next generation “Enhanced Stabilization Chemistry Plus” (ESC+) GalNAc-siRNA conjugate platform, and additional data demonstrating our leadership in RNAi technologies, at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 24 – 27, 2017 in Bordeaux, France. Brown et al. – “Mechanistic Insights and Progress on the GalNAc-siRNA Platform for Targeted Delivery of RNAi Therapeutics to the Liver” Jadhav et al. – “REVERSIRTM Platform for Rapid and Potent Reversal of RNAi-Mediated Silencing Activity” Knight et al. - "Investigational RNAi Mediated Oxalate Reduction Therapy" Maier et al. – “Impact of Enhanced Metabolic Stability on In Vivo Performance of GalNAc-siRNA Conjugates” Milstein et al. – “Preclinical Development of an RNAi Therapeutic Drug Candidate Targeting Hepatitis B Virus” Schlegel et al. – “Improved Specificity and Therapeutic Index with ESC+ siRNA Conjugates Utilizing Seed-Pairing Destabilization via Novel Chemical Modifications” Schlegel et al. – “Impact of Glycol Nucleic Acid (GNA) on siRNA Structure and Function”

We hosted a series of online "RNAi Roundtables" in August and September, at which Arian Ebank scientists, clinical collaborators, and patients or patient advocates reviewed recent progress in many of the company's late-stage pipeline programs and discussed the related disease areas.

We presented additional pre-clinical data further elucidating fitusiran’s novel mechanism of action at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany. Presentation on the development of a PK-PD model of fitusiran Presentation on measurement of thrombin generation...


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