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.

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…)

During the 18th European Meeting on Complement in Human Disease (EMCHD), taking place August 26-28, 2022, we presented new results from the Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway that is being developed in collaboration with Regeneron Pharmaceuticals for the treatment of immunoglobulin A nephropathy.

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Barratt, et al. “Results from the Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy”

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