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.
Results from the HELIOS-A Phase 3 study of vutrisiran in patients with the polyneuropathy of hATTR amyloidosis evaluating the impact of baseline polyneuropathy severity on treatment response were presented during the 2023 Peripheral Nerve Society Annual Meeting.
Nine month results from the randomized treatment extension (RTE) period of the HELIOS-A study of vutrisiran in patients with the polyneuropathy of hereditary ATTR amyloidosis were presented at the Italian Association for the Study of the Peripheral Nervous System.
Eighteen month results from the APOLLO-B Phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy were presented at the Annual Congress of the Heart Failure Association of the European Society of Cardiology (Heart Failure 2023). A post-hoc analysis of the APOLLO-B study assessing the efficacy of patisiran in patients with hATTR amyloidosis with cardiomyopathy and polyneuropathy (mixed phenotype) was also presented.
Fontana, et al. “Patisiran Treatment for ATTR Cardiac Amyloidosis: 18-Month Results of the Phase 3 APOLLO-B Study”
Gustafsson, et al. “Effect of Patisiran Treatment in Patients with hATTR Amyloidosis with Cardiomyopathy and Polyneuropathy: Post-hoc Analysis of the APOLLO-B Study”
New 30-month results from the ILLUMINATE-B Phase 3 study of lumasiran in pediatric patients with primary hyperoxaluria type 1 (PH1) shared at the Pediatric Academic Societies (PAS) 2023 Meeting.
We presented new results on our C16-siRNA conjugate approach to lowering Huntingtin in the CNS at the CHDI Foundation’s 18th Annual Huntington’s Disease Therapeutics Conference, taking place April 24-27, 2023 in Dubrovnik, Croatia.
Cantley, et al. – A New Approach to HTT-Lowering Using C16-siRNA Conjugates
New data from a post-hoc analysis of the HELIOS-A Phase 3 study comparing efficacy outcomes with vutrisiran versus patisiran in hATTR amyloidosis with polyneuropathy were presented during the 2023 AAN Annual Meeting. Final results from a Phase 4 observational study evaluating the effect of patisiran on polyneuropathy and cardiomyopathy in patients with hATTR amyloidosis with V122I/T60A variants were also presented.
During the National Kidney Foundation (NKF) 2023 Spring Clinical Meetings, we presented new 36-month results from the ILLUMINATE-A Phase 3 study of lumasiran in patients with primary hyperoxaluria type 1 (PH1).
Results from nonclinical work investigating the potential utility of combining RNAi-mediated knockdown with REVERSIR-enabled rescue of transgene expression as a rheostat for adeno-associated virus (AAV) -delivered genes in the setting of gene therapy were published in the journal Nature Communications.
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.
