United States Patent 10,781,451: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
The United States Patent 10,781,451, titled "Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof," is a significant patent in the field of genetic therapy, particularly for treating muscular dystrophy. This patent, assigned to the University of Western Australia, involves the use of antisense oligonucleotides to induce exon skipping in the dystrophin gene, a crucial approach for treating Duchenne muscular dystrophy (DMD) and other related disorders.
Background and Context
Duchenne muscular dystrophy is a severe genetic disorder caused by mutations in the dystrophin gene, leading to the absence or dysfunction of the dystrophin protein. Antisense oligonucleotides offer a promising therapeutic strategy by binding to specific sequences of the dystrophin gene, thereby skipping defective exons and allowing the production of a shorter but functional dystrophin protein[2].
Patent Scope
Inventors and Assignees
The patent was invented by Stephen Donald Wilton, Sue Fletcher, and Graham McClorey, and is assigned to the University of Western Australia. This institution has been at the forefront of research in exon skipping therapies[2].
Claims
The patent includes multiple claims that define the scope of the invention. These claims cover:
- Antisense Oligonucleotides: Specific sequences designed to bind to target sites within the dystrophin gene to induce exon skipping.
- Methods of Use: Procedures for administering these oligonucleotides to patients, including routes of administration and dosing regimens.
- Therapeutic Applications: The use of these oligonucleotides for treating muscular dystrophies, particularly DMD[2].
Target Sites and Sequences
The patent specifies numerous target sites within the dystrophin gene where the antisense oligonucleotides can bind to induce exon skipping. These sequences are detailed in the patent document, with over 200 specific sequences identified (SEQ ID NO: 1 to 202)[1].
Claims Analysis
Independent Claims
The patent includes independent claims that define the broadest scope of the invention. These claims typically cover the composition of the antisense oligonucleotides and their method of use.
Dependent Claims
Dependent claims further narrow down the scope by specifying particular aspects of the invention, such as the length of the oligonucleotides, the specific exons targeted, and the methods of administration.
Patent Landscape
Prior Art and References
The patent cites numerous prior art documents, including earlier patents and publications related to antisense oligonucleotide technology and exon skipping therapies. This extensive list of references underscores the evolutionary nature of the technology and the contributions of various researchers in the field[2].
Related Patents and Applications
The patent is part of a larger family of patents and applications related to exon skipping therapies. It is a continuation of several earlier applications, indicating a long-standing research effort in this area. Other related patents and applications cover various aspects of antisense oligonucleotide design, delivery methods, and therapeutic applications[2].
Technical and Scientific Aspects
Mechanism of Action
The antisense oligonucleotides work by binding to specific RNA sequences, preventing the inclusion of defective exons during the splicing process. This results in the production of a truncated but functional dystrophin protein, which can significantly improve muscle function in patients with DMD[2].
Chemical and Structural Details
The patent provides detailed chemical and structural information about the antisense oligonucleotides, including their sequences and modifications. These details are crucial for understanding the specificity and efficacy of the oligonucleotides[1].
Economic and Commercial Implications
Market Potential
The market potential for exon skipping therapies is significant, given the lack of effective treatments for DMD. This patent, along with others in the field, represents a major advancement in therapeutic options for patients with muscular dystrophy.
Competitive Landscape
The competitive landscape in this field is dynamic, with several pharmaceutical and biotechnology companies investing in exon skipping therapies. The University of Western Australia's patent positions it as a key player in this area, with potential for collaboration or licensing agreements with industry partners[5].
Regulatory and Legal Considerations
Patent Term and Extensions
The patent term is subject to extensions or adjustments under U.S. patent law. The patent also includes a terminal disclaimer, which can affect the duration of patent protection[2].
Global Patent System
The patent is part of the global patent system, with implications for international patent protection. Tools like the Global Dossier and Common Citation Document (CCD) facilitate the management and search of related patent applications across different jurisdictions[4].
Conclusion
United States Patent 10,781,451 is a pivotal patent in the field of genetic therapy, particularly for treating Duchenne muscular dystrophy. The detailed analysis of its scope, claims, and the broader patent landscape highlights its significance in advancing therapeutic options for patients with muscular dystrophy.
Key Takeaways
- Antisense Oligonucleotides: The patent covers specific sequences designed to induce exon skipping in the dystrophin gene.
- Therapeutic Applications: The primary use is for treating muscular dystrophies, especially DMD.
- Claims and Scope: The patent includes independent and dependent claims defining the composition and method of use of the oligonucleotides.
- Patent Landscape: Part of a larger family of patents and applications related to exon skipping therapies.
- Economic and Commercial Implications: Significant market potential and a dynamic competitive landscape.
FAQs
Q: What is the primary use of the antisense oligonucleotides described in the patent?
A: The primary use is to induce exon skipping in the dystrophin gene for treating muscular dystrophies, particularly Duchenne muscular dystrophy.
Q: Who are the inventors of the patent?
A: The inventors are Stephen Donald Wilton, Sue Fletcher, and Graham McClorey.
Q: What is the significance of the sequences listed in the patent?
A: The sequences (SEQ ID NO: 1 to 202) are specific antisense oligonucleotides designed to bind to target sites within the dystrophin gene to induce exon skipping.
Q: How does the patent fit into the broader patent landscape?
A: It is part of a larger family of patents and applications related to exon skipping therapies and is a continuation of several earlier applications.
Q: What are the economic implications of this patent?
A: The patent has significant market potential due to the lack of effective treatments for DMD and positions the University of Western Australia as a key player in the field.
Sources
- US10781451B2 - Antisense oligonucleotides for inducing exon skipping and methods of use thereof - Google Patents
- United States Patent - googleapis.com - Patent Images Storage
- Patent Claims Research Dataset - USPTO
- Search for patents - USPTO
- US-10781451-B2 · Priority Date: 2004-06-28 · Assignees: University of Western Australia - Unified Patents Portal
