RNA-Programmable Endonuclease Systems and Uses Thereof

Patent Number: U.S. 12203110 B2
Assignees: CRISPR Therapeutics AG and Bayer Healthcare LLC

What This Patent Covers

This patent protects novel synthetic RNA-guided endonucleases (sRGNs) — engineered CRISPR-type enzymes that can be programmed by a guide RNA to target and edit specific DNA sequences in cells. The invention encompasses:

RNA-programmable endonuclease systems derived from small type II CRISPR Cas9 nucleases.
Methods for targeting, editing, or manipulating DNA using these engineered nucleases in cells.
Implementation of these systems in genetic engineering workflows, including therapeutic genome editing.

Essentially, the claims protect both the engineered nuclease proteins and their use with guide RNAs to achieve precise, programmable DNA edits — a core function of CRISPR genome editing.

Why This Patent Is Important

1. Core Gene-Editing Technology for Therapeutics

The sRGNs claimed in this patent are fundamental tools for CRISPR-based gene therapies, including CRISPR Therapeutics’ lead programs such as CTX001 for sickle-cell disease and beta-thalassemia (CRISPR-edited hematopoietic stem cells). Programmable endonucleases are at the heart of how CRISPR therapies edit disease-causing genes.

2. Strengthens CRISPR Therapeutics’ IP Portfolio

Although much CRISPR foundational IP originates from academic inventors like Charpentier and Doudna, this patent represents CRISPR Therapeutics’ own inventive work on engineered CRISPR systems — giving it distinct claim rights around custom nuclease systems beyond basic Cas9.

3. Enables Therapeutic & Licensing Value

Engineered endonucleases with improved specificity, efficiency, or novel design can be more effective and safer in clinical contexts. Having IP rights around such systems supports the company’s competitive differentiation, potential licensing revenue, and defense against competitors in the crowded genome-editing space (e.g., Editas, Intellia).

Summary

Patent: RNA-programmable endonuclease systems and uses thereof (U.S. 12203110 B2).
Filed: March 19, 2019 (priority).
Focus: Engineered CRISPR RNA-guided nucleases and methods to target/edit DNA.
Importance: Provides CRISPR Therapeutics with proprietary rights over custom gene-editing systems key to therapeutic development, giving both scientific utility and commercial leverage in its clinical pipeline and broader licensing landscape.

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