BioTech IP Review

Application Number: PCT/US2025/017864
Filing Date: February 28, 2025
Publication Number: WO/2025/184520
Publication Date: September 4, 2025
Applicant: Intellia Therapeutics, Inc. (Cambridge, MA, USA)
Inventors: Gamze Karaca, Trisha Das, Arti Kanjolia, et al.

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What This Patent Covers

This international patent application discloses genome‑editing compositions and methods directed at the ANGPTL3 gene, which encodes angiopoietin‑like 3 — a protein that plays a central role in lipid metabolism and is a validated therapeutic target for cardiovascular and metabolic diseases:

Targeted Gene Editing:

• The described inventions include CRISPR‑based editing systems (such as guide RNAs and nuclease components) configured to introduce precise genetic modifications in the ANGPTL3 locus in cells. These modifications can disrupt or alter expression of ANGPTL3 to achieve therapeutic effects.

Therapeutic Methods:

• Methods for editing ANGPTL3 in patient cells or in vivo, potentially reducing circulating lipid levels and addressing conditions such as hyperlipidemia, atherosclerotic cardiovascular disease, and other metabolic syndromes where lowering ANGPTL3 activity is beneficial.

Compositions & Delivery:

• The application likely includes nucleotide constructs, CRISPR guide sequences, and delivery compositions (e.g., lipid nanoparticles or viral vectors) optimized to support efficient ANGPTL3 gene editing in target tissues such as the liver, which is the primary site of ANGPTL3 expression.

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Why This Patent Is Important

1) Expands Intellia’s Therapeutic Scope:
While Intellia’s lead programs (e.g., nexiguran ziclumeran and NTLA‑2002) focus on transthyretin amyloidosis and hereditary angioedema using CRISPR in vivo editing, this ANGPTL3 patent positions the company to tackle cardiovascular/metabolic disease markets, which are much larger in patient population and potential revenue than many rare genetic diseases.

2) Validated Target with Commercial Value:
Loss‑of‑function variants in ANGPTL3 are associated with lower lipid levels and reduced cardiovascular risk, which has already been validated clinically by other therapeutic modalities. Gene editing offers a one‑time genome‑level intervention that could be compelling to insurers and providers.

3) Platform Leverage:
Because it’s a method and composition patent, not tied to a single disease indication, it protects broad aspects of ANGPTL3 editing technology, giving Intellia IP that could be used in multiple product candidates and partnership/licensing deals.

4) Strategic Timing:
Filing early in 2025 and publishing by September allows Intellia to stake priority on this emerging indication ahead of competitors, securing rights that could support late‑stage development or collaborations.