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 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.
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.
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.
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.
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.
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”
Data from exploratory cardiac assessments and post-hoc analyses of safety data in the APOLLO Phase 3 study of patisiran, an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, were published in the journal Circulation.
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*.
* 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.
On August 15, 2018, we hosted an online RNAi Roundtable to review the progress with lumasiran, an RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1.
On July 24, 2018, we hosted an online RNAi Roundtable to review the progress with givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyrias.
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.
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…)
