Capella

We presented positive complete results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, at the 1st European ATTR Amyloidosis Meeting for Patients and Doctors, held November 2-3, 2017 in Paris, France. Adams et al. – “Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy: Results from the Phase 3 APOLLO Study” Berk et al. – “Long-Term, Open-Label Clinical Experience with Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy”

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.

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