Capella

We are hosting a series of online "RNAi Roundtables" from June through September, at which Alnylam scientists, clinical collaborators, and patients or patient advocates review recent progress in many of the Company's late-stage pipeline programs and discuss the related disease areas.

We presented additional results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 4th Congress of the European Academy of Neurology (EAN), held June 16-19, 2018 in Lisbon, Portugal. Coelho et al. –...

We presented new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the European Society of Cardiology Heart Failure 2018 Congress, held May 26-29, 2018 in Vienna, Austria. Slama et al. - "Analysis of NT-proBNP Baseline Levels in APOLLO as a Predictor of Survival in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis" Merlini et al. - "Impact of Patisiran on Norfolk Quality of Life Questionairre Diabetic Neuropathy in Patients with Hereditary Transthyretin-Mediated Amyloidosis: Results from the Cardiac Subpopulation in the Phase 3 APOLLO Study"

One of the main features of our ESC GalNAc-siRNA conjugates platform is potent and durable target knockdown in the liver sustained for several months in humans. In some cases, RNAi therapeutics may benefit from a technology that enables reversal of target mRNA knockdown and provides finer control over pharmacology, a desired attribute for therapeutic entities. REVERSIRTM molecules are GalNAc-conjugated, short, single-stranded, high affinity oligonucleotide constructs designed to recognize and bind to the complementary, RISC-bound antisense strand of siRNAs, thereby leading to rapid and complete reversal of RNAi-mediated target gene silencing activity in vivo. Read article in Nature Biotech

We presented new positive results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the American Academy of Neurology (AAN) 2018 Annual Meeting, being held April 21-27, 2018 in Los Angeles. Read our press release Adams et al. – “Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis: Results from the Phase 3 APOLLO Study” Adams et al. – “Evaluation of Quality of Life and Disability in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Following Treatment with Patisiran, an Investigational RNAi Therapeutic: Results from the Phase 3 APOLLO Study” Mora et al. – “Utility of Genetic Testing to Identify Individuals Suspected of Having Hereditary ATTR (hATTR) Amyloidosis”

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