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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.

25 Oct, 2023

Posted at 11:06h in Capella

We presented additional positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy, in a late-breaker session at the 16th Clinical Trials on Alzheimer’s Disease conference.

Mummery, et al. “Phase 1 Safety, Tolerability, and Pharmacological Results of ALN-APP, the First Investigational RNA Interference Therapeutic in Development for Early-Onset Alzheimer’s Disease”

08 Oct, 2023

Posted at 08:37h in Capella

New 24-month results from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting (ASM) 2023. Findings from a post-hoc analysis of the APOLLO-B Phase 3 study that further characterize the impact of patisiran on health status and quality of life in patients with the cardiomyopathy of ATTR amyloidosis were also presented, as well as an overview of baseline characteristics of patients who enrolled in the expanded access program for patisiran.

Maurer, et al. “Primary Results from APOLLO-B Open-label Extension Study of Patisiran in Patients with Transthyretin Cardiac Amyloidosis”
Shah, et al. “Impact of Patisiran on Health Status and Quality of Life in Patients with Transthyretin Cardiac Amyloidosis”
Saha, et al. “Baseline Characteristics of Patients with Transthyretin Cardiac Amyloidosis Enrolled in the Patisiran Expanded Access Program”

10 Sep, 2023

Posted at 16:11h in Capella

New results from Part D of the Phase 1 study of zilebesiran, assessing the safety and pharmacology of sequential doses of zilebesiran in patients with Class II or III obesity, were presented during the American Heart Association Hypertension Scientific Sessions 2023.

Taubel, et al. “Safety and Tolerability of Zilebesiran, an RNAi Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Obese Patients with Hypertension”

26 Aug, 2023

Posted at 11:33h in Capella

New results from a post hoc analysis of the APOLLO-B Phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy evaluating disease progression following 12 months of treatment with patisiran vs. placebo were presented during the European Society of Cardiology (ESC) Congress 2023.

Damy, et al. “Evaluation of Disease Progression in Patients with ATTR Amyloidosis with Cardiomyopathy Following Treatment with Patisiran: Post hoc Analysis of the APOLLO-B Study”

17 Jul, 2023

Posted at 02:23h in Capella

We presented updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA), at the 2023 Alzheimer’s Association International Conference (AAIC).

Cohen et al. “Interim Phase 1 Part A Results for ALN-APP, the First Investigational RNAi Therapeutic in Development for Alzheimer’s Disease”

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”

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