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 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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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”

On September 11, 2018, we hosted an online RNAi Roundtable to review the progress with ONPATTRO™ (patisiran) and ALN-TTRsc02, RNAi therapeutics for the treatment of transthyretin-mediated amyloidosis*.

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* ONPATTRO is approved in the U.S. for the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, and in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. **ALN-TTRsc02 is an investigational RNAi therapeutic, in development for the treatment of ATTR amyloidosis. 


We presented new analyses from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 2018 Peripheral Nerve Society (PNS) Annual Meeting, held July 22-25, 2018 in Baltimore, MD.

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Ajroud-Driss et al. – “Impact of Patisiran on Overall Health Status in hATTR Amyloidosis: Results from the APOLLO Trial”

Polydefkis et al. – “Infusion Related Reactions in Patients with hATTR Amyloidosis Treated with Patisiran”

Dyck et al. – “Neuropathy Progression in Patients with hATTR Amyloidosis: Analysis of the APOLLO Placebo Arm”

Coelho et al. – “Longitudinal Changes in mNIS+7 are Associated with Changes in Ambulatory Status in Hereditary Transthyretin-Mediated Amyloidosis”

Coelho et al. – “Indirect Comparison of Patisiran and Tafamidis for Treatment of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy”

Gonzalez-Duarte et al. – “Changes in Neuropathy Stage in Patients with Hereditary Transthyretin-Mediated Amyloidosis Following Treatment with Patisiran, an Investigational RNAi Therapeutic: An Analysis from the Phase 3 APOLLO Study” (more…)

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 International Congress on Neuromuscular Diseases (ICNMD), held July 6-10, 2018 in Vienna, Austria.

Obici et al. – “APOLLO Phase 3 Study: Impact of Baseline Neuropathy Severity on Response to Patisiran”

Coelho et al. – “Transthyretin Reduction with Patisiran in the APOLLO Phase 3 Study”

Mauermann et al. – “Impact of Patisiran on Autonomic Neuropathy in Hereditary Transthyretin-Mediated Amyloidosis Patients”

Goel et al. – “Population Pharmacokinetics (PK) of Patisiran in Healthy Volunteers and hATTR Patients”

Zhang et al. – “Pharmacokinetics of Patisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis”


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