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.
On October 21, 2022, we hosted an online RNAi roundtable to review Alnylam’s engine for sustainable innovation, including leadership in our RNAi platform and human genetics.
We presented on platform and clinical results at the Oligonucleotide Therapeutics Society (OTS) 2022 18th Annual Meeting held in Phoenix, Arizona on October 2-5, 2022.
Human Genetics as an Enabler of RNAi Therapeutics – Paul Nioi
Phase 3 study, HELIOS-A, in hATTR Patients Evaluating a Single Dose Regimen – Gabriel Robbie
Toxicology and DMPK Assessment of siRNA Therapeutics – Joe Dybowski
At the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022, 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”
Kale, et al. “Exploratory Analyses from APOLLO-B, a Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy”
On September 27, 2022, we hosted an online RNAi Roundtable to review the progress with cemdisiran, an investigational RNAi therapeutic in development for the treatment of IgA nephropathy (IgAN) and other complement-mediated diseases.
Results from a post-hoc analysis of patients on hemodialysis in the ongoing ILLUMINATE-C Phase 3 open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1) were presented at the International Pediatric Nephrology Association (IPNA) Congress (Sept. 7-11, 2022).
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
