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 post hoc analyses from the Phase 3 ENVISION study of givosiran in patients with acute hepatic porphyria (AHP) through Month 36 at the Hemostasis & Thrombosis Research Society (HTRS) Scientific Symposium, held on March 10-12 in Orlando, Florida.
We presented non-clinical results on ALN-APP*, our investigational RNAi therapeutic for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy at AD/PD 2023 Advances in Science & Therapy International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders, held on March 28-April 1 in Gothenburg, Sweden.
*The ALN-APP clinical program is being conducted as a partnership between Alnylam Pharmaceuticals and Regeneron Pharmaceuticals, Inc.
On December 15, 2022, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The event included presentations from Alnylam senior leaders as well as guest speakers.
To view the webcast, click here
To view the R&D Day presentation, click here
During the American Society of Nephrology (ASN) Kidney Week, we presented new 12-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients of all ages with primary hyperoxaluria type 1 (PH1) and advanced kidney disease. Additional research was presented on the development and validation of a novel assay to determine oxalate concentration in plasma.
Frishberg, et al. “Lumasiran for Patients with Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 12-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Clausen, et al. “Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography—Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials”
On November 1, 2022, we hosted an online RNAi roundtable to review Alnylam’s CNS delivery platform and ALN-APP, an investigational RNAi therapeutic in development for Alzheimer’s Disease and cerebral amyloid angiopathy.
New 18-month findings evaluating Neurofilament light chain (NfL) as a potential biomarker of treatment response in patients with hATTR amyloidosis with polyneuropathy were presented at the 147th annual meeting of the American Neurological Association (ANA).
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.
