Capella

We presented new 12-month interim results from the ongoing Global Open-Label Extension (OLE) study of ONPATTRO® (patisiran), demonstrating maintained reversal of disease progression and consistent safety profile with >4 years of patient experience and >6,000 doses administered. These results were presented at the 2019 Peripheral Nerve Society (PNS) Annual Meeting, held June 22-26 in Genoa, Italy. Read our press release Polydefkis et al. – "Long-Term Safety and Efficacy of Patisiran in Patients with hATTR Amyloidosis: Global OLE Study" Lin et al. – "Efficacy of Patisiran in Patients with hATTR Amyloidosis and Prior Tafamidis Use: Analysis of APOLLO" Obici et al. – "Indirect Treatment Comparison of the Efficacy of Patisiran and Inotersen for hATTR Amyloidosis with Polyneuropathy"

We presented new advances in our RNAi therapeutics platform, including preclinical results demonstrating oral delivery of GalNAc-conjugated small interfering RNAs (siRNAs) directed to a liver target. The results were presented at the 3rd International Conference on the Long and the Short of Non-Coding RNAs, held June 18-23 in Crete, Greece. These new preclinical data are the first demonstration of functional delivery of GalNAc-siRNA conjugates via the oral route of administration, representing an important step forward in potentially advancing and expanding the clinical and commercial potential of RNAi therapeutics. We believe that this approach can be applied to existing and future liver-directed pipeline programs, potentially creating a relatively near-term opportunity for Alnylam. Read our press release View the presentation

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 56th 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"

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. Read our press release View the presentation

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) 54th Annual International Liver Congress™, held April 10 – 14, 2019 in Vienna, Austria. Read our press release 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”

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.” Read our press release Read the paper in The New England Journal of Medicine

On December 6th, 2018, we will be hosting an R&D Day in New York City where Alnylam management and key opinion leaders will discuss our late stage clinical efforts, in addition to next wave programs and platform advances.  Scroll down to see the R&D Day agenda and links to the presentations.  Learn more about our science and the science of RNAi.  Click here to see our pipeline of RNAi therapeutics. To view the webcast, click here.  A replay of the webcast will be available at that link on December 7th. To view the R&D Day Presentation, click here.

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. Read our press release 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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