14 Jun, 2019 Case Study of a Healthy Human with Mutations in HAO1 Gene, a Validated Target for the Treatment of PH1, and Diagnostic Journey Research on PH1

We presented a case study of a healthy human with mutations in the HAO1 gene, a validated target for the treatment of primary hyperoxaluria type 1 (PH1), as well as results from research on the diagnostic journey of PH1 at the 56^th Congress of the European Renal Association (ERA) and European Dialysis and Transplant Association (EDTA) held on June 13-16 in Budapest, Hungary.

Erbe et al. – “Identification and Phenotyping of a Healthy Human with Mutations in HAO1 Supports Glycolate Oxidase Knockdown as a Potential Approach to Therapy for Primary Hyperoxaluria Type 1”

Danese et al. – “The Importance of Evaluating Potential Underlying Causes of Kidney Stones: A Survey of Physician Experiences in Diagnosing Primary Hyperoxaluria Type 1”

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15 Apr, 2019 New Positive Results from Phase 2 Open-Label Extension Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1

We presented new results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1) at the International Society of Nephrology (ISN) 2019 Annual Meeting held on April 13-16 in Melbourne, Australia.

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12 Apr, 2019 ENVISION Phase 3 Results for Givosiran Presented at EASL

We presented positive complete results from the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria (AHP), at the European Association for the Study of the Liver (EASL) 54^th Annual International Liver Congress™, held April 10 – 14, 2019 in Vienna, Austria.

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Balwani et al. – “ENVISION, a Phase 3 Study to Evaluate the Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1, in Acute Hepatic Porphyria Patients”

Sardh et al. – “Management of Recurrent Acute Hepatic Porphyria (AHP) Attacks in Europe and the United States: EXPLORE International, Prospective, Natural History Study”

Ventura et al. – “Disease Manifestations of Patients with Recurrent Acute Hepatic Porphyria (AHP) and Daily Life Impacts in EXPLORE International, Prospective, Natural History Study”

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29 Mar, 2019 Pre-Clinical Data on Delivery of RNAi Therapeutics to the CNS and Eye

We presented new pre-clinical data on our efforts in CNS and ocular delivery of novel siRNA conjugates in rats and non-human primates at the sixth annual Cold Spring Harbor Conference on RNA and Oligonucleotide Therapeutics, held March 27 to 30 in Cold Spring Harbor, NY.

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06 Feb, 2019 Phase 1 Results for Givosiran Published in “The New England Journal of Medicine”

We published results from the Phase 1 study of givosiran, our investigational RNAi therapeutic for the treatment of acute hepatic porphyria (AHP), in The New England Journal of Medicine (NEJM) in a paper titled “Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.”

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09 Nov, 2018 Updated Phase 1/2 OLE Results for Givosiran and EXPLORE Natural History Study Data at AASLD 2018

We presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), at The Liver Meeting® 2018 of the American Association for the Study of Liver Diseases (AASLD), held November 9-13, 2018 in San Francisco, CA.

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Anderson et al. – “Phase 1/2 and Open Label Extension Studies of Givosiran, an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Acute Intermittent Porphyria”

Bonkovsky et al. – “EXPLORE: A Prospective, Multinational, Natural History Study of Acute Hepatic Porphyrias (AHP) Patients with Recurrent Attacks”

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25 Oct, 2018 New Clinical Results from Phase 1/2 and Phase 2 OLE Studies of Lumasiran at ASN 2018 Annual Meeting

We presented new results from the Phase 1/2 and Phase 2 open-label extension (OLE) studies of lumasiran at the American Society of Nephrology (ASN) 2018 Annual Meeting, held October 23-28, 2018 in San Diego, CA.

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Hulton et al. – “Safety and Efficacy Study of Lumasiran (ALN-GO1), an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Primary Hyperoxaluria Type 1”

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11 Oct, 2018 New Clinical Results from APOLLO OLE Study at 2018 AANEM Annual Meeting

We presented new 12-month efficacy and safety data with patisiran from the APOLLO Global Open Label Extension (OLE) Study at the 2018 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, held October 10-13, 2018 in Washington, DC.

Gonzalez-Duarte et al. – “Long-Term Use of Patisiran, an Investigational RNAi Therpaeutic, in Patients with Hereditary Transthyretin-Mediated Amyloidosis: 12 Month Efficacy and Safety Data from Global Open Label Extension (OLE) Study”

08 Oct, 2018 New Research on Acute Hepatic Porphyrias at ACG 2018 Annual Meeting

We presented new research on the diagnostic patterns, clinical experience and healthcare utilization of patients with acute hepatic porphyrias at the American College of Gastroenterology 2018 Annual Scientific Meeting, held October 5-10, 2018 in Phildelphia, PA.

Ko et al. – “Real-World Analysis of Symptoms, Diagnostic Patterns, and Provider Perspective on Acute Hepatic Porphyrias”

Rudnick et al. – “The Patient Odyssey to Confirmed Acute Hepatic Porphyria Diagnosis: Clinical Characteristics and Healthcare Utilization of Patients Preceding Diagnosis of Acute Hepatic Porphyria”