Capella—the Online Voice of Progress in RNAi

Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.

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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Read the paper in The New England Journal of Medicine (more…)

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.

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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”


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”


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”

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”


We presented updated positive results from the Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting, held October 3-6, 2018 in Antalya, Turkey.

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View the presentation


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”


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