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 Findings From Open-Label Extension Study and Qualitative Patient Interview Study of GIVLAARI® (givosiran) Presented at European Association for the Study of the Liver (EASL) Congress 2023

Posted at 11:12:41 h

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”

Read More
27 Jun, 2023 Findings from Phase 1 Study of ALN-HSD Presented at EASL Congress 2023

Posted at 13:50:22 h

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”

Read More
27 Jun, 2023 Overview of ALN-KHK Preclinical Development Program Presented at American Diabetes Association 83rd Scientific Sessions

Posted at 13:49:00 h

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”

Read More
22 Jun, 2023 Findings From Novel X-ray-Based Bone Oxalosis Grading Scale for PH1 Presented at European Renal Association (ERA) 2023 Meeting

Posted at 16:00:41 h

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”

Read More
22 Jun, 2023 Overview of Lumasiran Clinical Development Program Presented at the Oxalosis & Hyperoxaluria Foundation (OHF) 14th International Hyperoxaluria Workshop

Posted at 15:59:04 h

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.”

Read More
20 Jun, 2023 Results from HELIOS-A Phase 3 Study Presented at 2023 PNS Annual Meeting

Posted at 10:14:13 h

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”

Read More
26 May, 2023 Results from HELIOS-A RTE Period Presented during Annual Meeting of the ASNP

Posted at 08:48:02 h

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”

Read More
20 May, 2023 New Results from APOLLO-B Phase 3 Study of Patisiran Presented during Heart Failure 2023

Posted at 10:49:38 h

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”

Read More
28 Apr, 2023 Alnylam Presents 30-Month Results from ILLUMINATE-B Phase 3 Study of OXLUMO® (lumasiran) at the Pediatric Academic Societies (PAS) 2023 Meeting

Posted at 11:09:35 h

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”

Read More
26 Apr, 2023 New Results for RNAi Mediated Lowering of HTT Presented at 2023 CHDI Conference

Posted at 12:29:07 h

We presented new results on our C16-siRNA conjugate approach to lowering Huntingtin in the CNS at the CHDI Foundation’s 18^th 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

Read More

Capella

Capella—the Online Voice of Progress in RNAi

07 Jul, 2023 Findings From Open-Label Extension Study and Qualitative Patient Interview Study of GIVLAARI® (givosiran) Presented at European Association for the Study of the Liver (EASL) Congress 2023

27 Jun, 2023 Findings from Phase 1 Study of ALN-HSD Presented at EASL Congress 2023

27 Jun, 2023 Overview of ALN-KHK Preclinical Development Program Presented at American Diabetes Association 83rd Scientific Sessions

22 Jun, 2023 Findings From Novel X-ray-Based Bone Oxalosis Grading Scale for PH1 Presented at European Renal Association (ERA) 2023 Meeting

22 Jun, 2023 Overview of Lumasiran Clinical Development Program Presented at the Oxalosis & Hyperoxaluria Foundation (OHF) 14th International Hyperoxaluria Workshop

20 Jun, 2023 Results from HELIOS-A Phase 3 Study Presented at 2023 PNS Annual Meeting

26 May, 2023 Results from HELIOS-A RTE Period Presented during Annual Meeting of the ASNP

20 May, 2023 New Results from APOLLO-B Phase 3 Study of Patisiran Presented during Heart Failure 2023

28 Apr, 2023 Alnylam Presents 30-Month Results from ILLUMINATE-B Phase 3 Study of OXLUMO® (lumasiran) at the Pediatric Academic Societies (PAS) 2023 Meeting

26 Apr, 2023 New Results for RNAi Mediated Lowering of HTT Presented at 2023 CHDI Conference

FOLLOW US