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Capella

Capella—the Online Voice of Progress in RNAi

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.

07 Jul, 2023

Posted at 11:12h in Capella

At the European Association for the Study of the Liver (EASL) Congress 2023, we presented results from a Phase 1/2, 48-month, open-label extension study and a qualitative patient interview study of givosiran.

Sardh, et al. “Final Results from a Phase 1/2, 48-Month, Open-Label Extension Study of Givosiran in Patients with Acute Intermittent Porphyria
Lombardelli, et al. “Patient Experience with Acute Hepatic Porphyria before and after long-term givosiran treatment: a qualitative interview study”

27 Jun, 2023

Posted at 13:50h in Capella

We presented findings from a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic, in healthy adults and patients with nonalcoholic steatohepatitis, at the European Association for the Study of the Liver (EASL) Congress 2023.

Sanyal, et al. “Phase 1 Study of the RNA Interference Therapeutic ALN-HSD in Healthy Adults and Patients with Nonalcoholic Steatohepatitis”

27 Jun, 2023

Posted at 13:49h in Capella

We presented an overview of the preclinical development program for ALN-KHK, an investigational RNAi therapeutic for type 2 diabetes mellitus, at the American Diabetes Association (ADA) 83rd Scientific Sessions.

Noetzli, et al. “Preclinical Development of ALN-KHK, an Investigational RNAi Therapeutic for Type 2 Diabetes Mellitus”

22 Jun, 2023

Posted at 16:00h in Capella

At the European Renal Association (ERA) 2023 Meeting, we presented results from a novel X-ray-based bone oxalosis grading scale for patients with primary hyperoxaluria type 1 (PH1).

Baker, et al. “Development of an X-ray–Based Bone Oxalosis Grading Scale to Assess Oxalate Accumulation in Patients with Primary Hyperoxaluria Type 1”

22 Jun, 2023

Posted at 15:59h in Capella

We presented an overview of the lumasiran clinical development program, including data from the Phase 3 ILLUMINATE-A, ILLUMINATE-B and ILLUMINATE-C studies, in patients with primary hyperoxaluria type 1 (PH1) at the Oxalosis & Hyperoxaluria Foundation (OHF) 14th International Hyperoxaluria Workshop.

Shasha-Lavsky, et al. “Targeting Glycolate Oxidase for the Treatment of Primary Hyperoxaluria Type 1: Development and Clinical Characterization of Lumasiran, an RNAi Therapeutic.”

20 Jun, 2023

Posted at 10:14h in Capella

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.

Luigetti, et al. “Impact of Baseline Polyneuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study”

26 May, 2023

Posted at 08:48h in Capella

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.

Obici, et al. “HELIOS-A: 9-Month Results from the Randomized Treatment Extension Period of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

20 May, 2023

Posted at 10:49h in Capella

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”

28 Apr, 2023

Posted at 11:09h in Capella

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.

Michael, et al. “Efficacy and Safety of Lumasiran for Infants and Young Children with Primary Hyperoxaluria Type 1: 30-Month Analysis of the Phase 3 ILLUMINATE-B Trial”

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