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Alnylam Collaborator Regeneron Advances CIDEBsiRNA with Promising First-in-Human Results in Patients with MASLD

  • A single subcutaneous dose of investigational CIDEB siRNA reduced liver fat by up to 80% in patients with MASLD, with effects sustained for 36 weeks
  • Initial results from the first-in-human Phase 1/2 trial were presented at AASLD The Liver Meeting ® 2026 & published simultaneously in The New England Journal of Medicine
  • Regeneron Pharmaceuticals leads development & holds exclusive commercialization rights for CIDEB siRNA following discovery work by the Regeneron Genetics Center
  • Alnylam Pharmaceuticals is eligible for development milestones & tiered, double-digit royalties up to 20% under their strategic partnership agreement
  • The ongoing randomized, double-blind, placebo-controlled trial evaluates safety, tolerability, & efficacy measured by MRI-PDFF changes in liver fat fraction
  • CIDEB siRNA targets the CIDEB gene to silence disease-causing proteins using Alnylam's RNA interference (RNAi) platform
  • Findings support the potential for RNAi therapeutics to treat metabolic liver disease & expand into broader disease areas beyond current approvals