02 Oct, 2018 New Platform Innovations for RNAi Therapeutics at OTS 2018 Annual Meeting

We presented further progress on our platform efforts in extrahepatic delivery of novel siRNA conjugates, including CNS and ocular delivery in rat and non-human primates, at the Oligonucleotide Therapeutics Society (OTS) 2018 Annual Meeting, held September 30 to October 3 in Seattle, WA.

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Maier et al. – “ESC+ Design Minimizes the Off-Target Potential and Further Maximizes the Therapeutic Index of GalNAc-siRNA Conjugates”

Janas et al. – “Safety Evaluation of 2′-Deoxy-2′-Fluoro-Modified Nucleotides in GalNAc-siRNA Conjugates”

Milstein et al. – “Robust and Durable Target Silencing in the CNS with siRNA Conjugates”

Agarwal et al. – “Mechanisms of Rat Hepatotoxicity of GalNAc-siRNA Conjugates”

Theile et al. – “Bis-RNAi™ Conjugates for Simultaneous Silencing of Two Different Gene Transcripts”

Nair et al. – “Efficient and Durable Ocular Gene Silencing of TTR After Single Intravitreal Administration of siRNA Conjugates”

Rocca et al. – “The Influence of GalNAc Valency on the Pharmacokinetic and Pharmacodynamic Parameters of siRNA in Rats”