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.

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”

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”

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”

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”

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”

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.”

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”

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”

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