BioTech IP Review

ANGPTL3 iRNA Compositions and Methods of Use Thereof

  • Assignee: Alnylam Pharmaceuticals, Inc.
  • Patent Number: 12,467,051
  • Filing Date: January 28, 2022
  • Patent Issued: November 4, 2025
  • Inventors: Kirk Brown, Jeffrey Zuber (among others)

What It Covers

This issued patent protects double-stranded ribonucleic acid (dsRNA) agents — a type of small interfering RNA (siRNA) — specifically designed to target the ANGPTL3 gene and methods for using these agents to inhibit ANGPTL3 expression in subjects.

Key Components of the Invention

  • ANGPTL3 iRNA agents:
    The patent claims chemically defined dsRNA molecules (siRNAs) whose antisense strand is complementary to a portion of the ANGPTL3 gene, allowing the RNAi mechanism to silence ANGPTL3 expression.
  • Methods of use:
    It includes methods of inhibiting ANGPTL3 expression by administering the dsRNA compositions, as well as methods of treating diseases associated with ANGPTL3 activity, such as lipid metabolism disorders (e.g., hyperlipidemia or hypertriglyceridemia).
  • Composition of matter claims:
    Beyond methods, the patent likely covers the structure and sequences of the dsRNA agents themselves, which form the basis of therapeutic constructs.

Why This Patent Is Important

1. Expands Alnylam’s Therapeutic Scope

ANGPTL3 is a validated target for lipid disorders: reducing ANGPTL3 levels can lower triglycerides and improve overall lipid profiles, making this target attractive for cardiovascular and metabolic disease therapies — a multi-billion-dollar opportunity in biotech.

2. Core RNAi Platform Utilization

This patent exemplifies Alnylam’s RNAi therapeutic platform — using siRNAs to silence disease-causing genes. The company’s existing marketed products (e.g., Onpattro, Amvuttra) and pipeline candidates all leverage this platform.

3. Strengthening IP Position

Adding patents on specific gene targets like ANGPTL3 increases Barriers to Entry for competitors and strengthens Alnylam’s freedom to operate in RNAi drug development. This is essential as RNAi advances to treat broader disease areas beyond rare genetic conditions.

4. Broader Patent Estate

Alnylam’s IP estate also includes numerous other issued patents and applications covering RNAi agents targeting other genes (e.g., PCSK9, F5, PNPLA3), chemical modifications, and delivery technologies — forming a defensible suite that supports clinical and commercial expansion.

Summary

U.S. Patent No. 12,467,051, issued on November 4, 2025, protects siRNA compositions targeting the ANGPTL3 gene and their therapeutic use to inhibit ANGPTL3 expression, which is directly relevant to treating lipid metabolism disorders. This patent is strategically important for Alnylam because it reinforces its leadership in RNAi therapeutics and supports commercial expansion into broader cardiovascular and metabolic disease markets — areas with significant unmet needs and high commercial value.

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