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 are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, as well as medical thought leaders, will review recent progress in a selection of our pipeline programs and platform innovations, and provide perspectives on clinical developments and unmet needs.
During a late-breaker session at the 18th International Symposium on Amyloidosis, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
Read the press release
Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”
During the 18th European Meeting on Complement in Human Disease (EMCHD), taking place August 26-28, 2022, we presented new results from the Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway that is being developed in collaboration with Regeneron Pharmaceuticals for the treatment of immunoglobulin A nephropathy.
Read the press release
Barratt, et al. “Results from the Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy”
Results from the 12-month analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of OXLUMO® (lumasiran) in patients less than six years of age with primary hyperoxaluria type 1 (PH1) were published online in Pediatric Nephrology.
Read the paper in the Pediatric Nephrology
Eighteen-month efficacy and safety results from the HELIOS-A Phase 3 study of AMVUTTRA™ (vutrisiran), an RNAi therapeutic recently approved in the U.S. for adults with the polyneuropathy of hereditary ATTR (hATTR) amyloidosis, were published in Amyloid.
Read the paper in Amyloid
Results from the six-month primary analysis of the ongoing ILLUMINATE-C Phase 3 study of lumasiran in patients of all ages with primary hyperoxaluria type 1 (PH1) and advanced kidney disease were published online in the American Journal of Kidney Diseases.
Read the paper in the American Journal of Kidney Diseases
New results from the six-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress (May 19-22, 2022).
Findings from a separate pooled pharmacokinetic and pharmacodynamic analysis of data from four clinical trials of lumasiran were also presented.
Read the press release
Groothoff, et al. “Lumasiran for Patients With Primary Hyperoxaluria Type 1 With Impaired Kidney Function: Data From the 6-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Gansner, et al. “Pharmacokinetics and Pharmacodynamics of Lumasiran: Analysis of Four Clinical Studies”
During the annual Heart Failure congress of the Heart Failure Association of the European Society of Cardiology, taking place May 21-24, 2022, we presented new 18-month results from the exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.
Read the press release
Garcia-Pavia, et al. “HELIOS-A: 18-Month Exploratory Cardiac Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis”
New results from the HELIOS-A study of vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented during the Peripheral Nerve Society (PNS) 2022 Annual Meeting, taking place May 14-17, 2022.
New results on Alnylam’s platform advancements enabling delivery of therapeutic short interfering RNAs (siRNAs) to the central nervous system (CNS) were presented at the TIDES USA, Oligonucleotide & Peptide Therapeutics 2022 Meeting being held in Boston on May 9-12. Additional preclinical data were presented on Alnylam’s GEMINI platform for simultaneous silencing of two transcripts within the CNS with a single siRNA entity.
Thiel CS., “Recent Advancements for the Delivery of siRNAs to the Central Nervous System”
