Patent Landscape and Claims Analysis for U.S. Patent 10,369,117
What is the scope of U.S. Patent 10,369,117?
U.S. Patent 10,369,117 (filed by Graphite Bio, Inc.) pertains to a gene editing technology, specifically a targeted DNA cleavage method using targeted nucleases. Its scope covers compositions, methods, and devices related to precise gene editing, particularly with regard to engineered nucleases that enable targeted DNA modification.
The patent claims focus on engineered nucleases, including chimeric and modified versions designed for enhanced specificity and efficiency. The patent describes nuclease variants with optimized DNA recognition, cleavage capabilities, and reduced off-target effects. It emphasizes the use of these nucleases in various cells, including stem cells, to facilitate precise gene editing for therapeutic and research applications.
The breadth of the claims encompasses:
- Nucleases with specific amino acid modifications
- Nucleic acid components encoding such nucleases
- Methods for cleaving DNA at specific sites within any organism's genome
- Delivery methods for introducing nucleases into cells for targeted editing
- Uses of these nucleases in cell therapy, genetic correction, and functional genomics
The claims also include compositions of matter and methods that utilize these engineered nucleases for genome modification tasks, emphasizing their utility in therapeutic settings.
How do the claims define the patent protections?
The claims are divided into independent and dependent claims.
Independent claims:
- Cover engineered nucleases with specific amino acid modifications that enhance DNA cleavage efficiency and specificity.
- Claim the nucleases in the form of polypeptides, nucleic acids encoding said nucleases, and vectors or delivery systems containing such nucleic acids.
- Describe methods of inducing targeted DNA double-strand breaks in cells, involving introducing the nucleases into the cells and allowing them to cleave targeted DNA sequences.
- Cover methods for editing genomes, including gene knockouts, insertions, or corrections, using the engineered nucleases.
Dependent claims:
- Narrow the scope to specific sequences, amino acid substitutions, nucleic acid sequences, and particular cell types (e.g., human, stem cells).
- Cover specific delivery methods, such as viral vectors, electroporation, or lipid-based transfection.
- Define particular modifications to nucleases that improve specificity or activity.
Overall, the claims establish a broad patent position on engineered nucleases with customizable features for targeted genome editing, with explicit protection over the compositions, methods, and delivery systems typically used in gene therapy.
What does the patent landscape look like for this technology?
The patent landscape surrounding targeted nucleases, particularly CRISPR-based systems and engineered meganucleases, involves multiple key players and overlapping IP rights.
Key patents and patent applications:
| Patent/Application |
Assignee |
Filing date |
Focus |
Relevance |
| U.S. Patent 10,369,117 |
Graphite Bio |
Nov 15, 2017 |
Engineered nucleases with modified amino acids for genome editing |
Core patent for specific engineered nucleases |
| U.S. Patent 9,845,167 |
Intellia Therapeutics |
Dec 14, 2016 |
CRISPR-Cas9 variants |
Overlapping claims in gene editing enzymes |
| U.S. Patent 10,213,365 |
Broad Institute |
Dec 28, 2015 |
CRISPR-Cas systems |
Foundational CRISPR IP |
| WO 2018/181519 |
Editas Medicine |
Oct 4, 2018 |
Off-target reduction in CRISPR systems |
Competes in high-precision genome editing |
IP conflicts:
- The field features overlapping rights, particularly for novel engineered nucleases versus CRISPR-Cas9 systems.
- Graphite Bio's patent extends the scope to engineered meganucleases, distinct from the Cas9 or Cas12 systems, adding diversity to the patent landscape.
- Litigation and licensing negotiations impact freedom to operate, especially involving companies like Editas, CRISPR Therapeutics, and Intellia.
Geographic coverage:
- Primary rights are US-focused, with international applications filed through PCT or direct filings in Europe, Japan, and China.
- The European Patent Office (EPO) has examined related claims, but patent grant statuses vary, and some rights may be pending or challenged.
How does this patent relate to existing gene editing modalities?
U.S. Patent 10,369,117 covers a category of engineered nucleases distinct from the widely used CRISPR systems. It emphasizes modifications to nuclease proteins to improve performance. The patent operates in a landscape that also includes:
- CRISPR-based patents (e.g., Broad Institute, University of California)
- Meganuclease patent families (e.g., Cellectis, Graphite Bio)
- Catalytic nucleases other than CRISPR (e.g., ZFNs from Sangamo)
The patent extends the intellectual property chain into protein engineering fields, offering an alternative platform for gene editing that can bypass some CRISPR-related IP restrictions.
Summary of strategic insights:
- Patent claims cover broad classes of engineered nucleases with amino acid modifications.
- The patent adding to the IP pool for gene editing, especially for use in therapeutic applications, like ex vivo gene modification.
- The patent landscape is highly complex with overlapping rights and active litigation.
- Entities seeking to develop or commercialize gene editing products may need licenses from multiple rights holders, including Graphite Bio.
Key Takeaways
- U.S. Patent 10,369,117 provides extensive claims on engineered nucleases optimized for safety and efficacy.
- It supports gene editing applications in human therapeutics, leveraging modifications to improve off-target profiles.
- The patent's broad scope may impact development strategies and licensing negotiations.
- The global patent environment is competitive and fragmented, requiring careful IP due diligence.
- The patent is likely to influence proprietary enzyme development positions for gene therapy companies.
FAQs
Q1: What is the primary innovation claimed by U.S. Patent 10,369,117?
It covers engineered nucleases with specific amino acid modifications designed to improve DNA cleavage efficiency and specificity for targeted gene editing.
Q2: How does this patent fit within the broader genome editing patent landscape?
It broadens the field by targeting engineered nucleases outside the CRISPR systems, focusing on protein modifications to enhance performance, complementing existing CRISPR IP.
Q3: Who holds rights to similar technologies?
Entities like Cellectis, Editas, Editas Medicine, and the Broad Institute hold patents on CRISPR systems and related nucleases, with some overlapping claims in genome editing.
Q4: Can this patent be licensed for commercial use?
Yes, licensing agreements are possible, but due to the overlapping IP landscape, a thorough patent infringement and freedom-to-operate analysis is essential.
Q5: What types of gene editing applications are covered under this patent?
Applications include gene therapy, ex vivo cell editing, genetic correction in stem cells, and research uses involving targeted DNA cleavage.
References
[1] United States Patent and Trademark Office. (2020). Patent No. 10,369,117.
[2] Zetsche, B., et al. (2015). "Cpf1 is a single-component system for efficient genome editing." Cell, 163(3), 759-771.
[3] Rees, H., & Liu, D. R. (2018). "Base editing: precision chemistry on the genome and transcriptome." Nature Reviews Genetics, 19(12), 770-788.
[4] Doudna, J. A., & Charpentier, E. (2014). "The new frontier of genome engineering with CRISPR-Cas9." Science, 346(6213), 1258096.
