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.

During the American Society of Nephrology (ASN) Kidney Week, we presented new 12-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients of all ages with primary hyperoxaluria type 1 (PH1) and advanced kidney disease. Additional research was presented on the development and validation of a novel assay to determine oxalate concentration in plasma.

Frishberg, et al. “Lumasiran for Patients with Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 12-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Clausen, et al. “Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography—Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials”

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New 18-month findings evaluating Neurofilament light chain (NfL) as a potential biomarker of treatment response in patients with hATTR amyloidosis with polyneuropathy were presented at the 147th annual meeting of the American Neurological Association (ANA).

Aldinc, et al. “NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy”

At the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

Read the press release
Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”
Kale, et al. “Exploratory Analyses from APOLLO-B, a Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy”

Results from a post-hoc analysis of patients on hemodialysis in the ongoing ILLUMINATE-C Phase 3 open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1) were presented at the International Pediatric Nephrology Association (IPNA) Congress (Sept. 7-11, 2022).

Michael, et al. “Relationship of Baseline Weight and Response to Lumasiran in Patients With Primary Hyperoxaluria Type 1 on Hemodialysis”

We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, as well as medical thought leaders, will review recent progress in a selection of our pipeline programs and platform innovations, and provide perspectives on clinical developments and unmet needs.

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During a late-breaker session at the 18th International Symposium on Amyloidosis, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

Read the press release
Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”

(more…)

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