United States Patent 10,781,450: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
United States Patent 10,781,450, titled "Antisense molecules and methods for treating pathologies," is a significant patent that delves into the use of antisense molecules for therapeutic applications, particularly in the treatment of genetic diseases like Duchenne Muscular Dystrophy (DMD). This analysis will explore the scope, claims, and the broader patent landscape surrounding this invention.
Background
The patent, issued to treat specific pathologies, involves the use of antisense molecules that bind to selected target sites to induce exon skipping in the dystrophin gene. This approach is crucial for treating DMD, a genetic disorder caused by mutations in the dystrophin gene[2].
Scope of the Patent
Therapeutic Applications
The patent focuses on the therapeutic use of antisense molecules, specifically designed to target and modify the dystrophin gene. This gene is critical for muscle function, and mutations in it lead to severe muscular dystrophies. The scope includes methods for treating DMD and other genetic diseases by inducing exon skipping, which allows for the production of a functional dystrophin protein despite the presence of mutations[2].
Antisense Molecules
The patent describes antisense oligonucleotides, which are short, single-stranded DNA or RNA molecules designed to bind to specific sequences of the dystrophin gene. These molecules can induce exon skipping, thereby bypassing the mutated regions and enabling the production of a truncated but functional dystrophin protein[2].
Claims of the Patent
Independent and Dependent Claims
The patent includes several claims that define the scope of the invention. These claims can be categorized into independent and dependent claims.
- Independent Claims: These claims stand alone and define the core aspects of the invention. For example, Claim 1 might describe the antisense molecule itself, its structure, and its ability to bind to a specific target site in the dystrophin gene.
- Dependent Claims: These claims build upon the independent claims and provide additional details or variations. For instance, dependent claims might specify different sequences of the antisense molecules, methods of administration, or specific formulations[2].
Key Claim Elements
- Antisense Oligonucleotides: Claims describe the composition and structure of the antisense oligonucleotides, including their length, sequence, and chemical modifications.
- Target Sites: The claims specify the target sites within the dystrophin gene where the antisense molecules bind to induce exon skipping.
- Methods of Treatment: Claims outline the methods for using these antisense molecules to treat genetic diseases, including DMD.
- Pharmaceutical Compositions: The patent also claims pharmaceutical compositions containing the antisense molecules, along with carriers and other components necessary for effective delivery[2].
Patent Landscape
Prior Art and Related Patents
The patent landscape for antisense technology is extensive, with numerous patents and publications related to the use of antisense oligonucleotides for therapeutic purposes. Prior art includes other patents and research focused on exon skipping and gene modification techniques. For example, the European Patent Office (EPO) and other international patent offices have granted patents related to similar antisense technologies[1].
Global Dossier and International Patent Offices
The Global Dossier service provided by the USPTO allows users to access the file histories of related applications from participating IP Offices, including the IP5 Offices (USPTO, EPO, JPO, KIPO, and CNIPA). This service helps in understanding the global patent family and prior art citations relevant to this invention[1].
Search Tools and Resources
To navigate the patent landscape, various search tools are available, such as the Patent Public Search tool, which replaced legacy search tools like PubEast and PubWest. Additionally, resources like the Common Citation Document (CCD) and international patent databases (e.g., WIPO's PATENTSCOPE) provide comprehensive access to prior art and related patent applications[1].
Economic and Competitive Impact
Innovation and Competitiveness
The USPTO's mission to drive innovation and global competitiveness is reflected in patents like US 10,781,450. Such patents contribute to the advancement of medical treatments and the development of new therapeutic approaches, which can significantly impact public health and the pharmaceutical industry[4].
Market and Therapeutic Implications
The approval and commercialization of treatments based on this patent can lead to significant market opportunities. Given the critical need for effective treatments for genetic diseases like DMD, this patent holds substantial therapeutic and commercial value.
Expert Insights and Statistics
Expert Opinions
Industry experts highlight the importance of antisense technology in treating genetic diseases. For example, Dr. Jerry Mendell, a leading researcher in DMD, has emphasized the potential of exon skipping therapies in improving patient outcomes.
Statistical Impact
The impact of such patents can be quantified through various metrics. For instance, the Patent Claims Research Dataset by the USPTO provides insights into the scope and claims of patents, including those related to antisense technology. This dataset can help in understanding the trends and economic implications of these inventions[3].
Challenges and Future Directions
Regulatory and Ethical Considerations
The development and approval of antisense therapies involve rigorous regulatory processes and ethical considerations. Ensuring the safety and efficacy of these treatments is crucial before they can be widely adopted.
Technological Advancements
Future directions in antisense technology include advancements in delivery methods, improved specificity of the antisense molecules, and the exploration of new target genes for various diseases.
Key Takeaways
- Therapeutic Focus: The patent is centered on the use of antisense molecules to treat genetic diseases, particularly DMD.
- Scope and Claims: The patent includes detailed claims on the structure and use of antisense oligonucleotides, methods of treatment, and pharmaceutical compositions.
- Patent Landscape: The patent is part of a broader landscape of antisense technologies, with significant prior art and related international patents.
- Economic Impact: The patent contributes to innovation and competitiveness in the pharmaceutical industry, with substantial market and therapeutic implications.
FAQs
Q: What is the primary focus of United States Patent 10,781,450?
A: The primary focus is on the use of antisense molecules to treat genetic diseases, particularly Duchenne Muscular Dystrophy (DMD), by inducing exon skipping in the dystrophin gene.
Q: What are antisense oligonucleotides?
A: Antisense oligonucleotides are short, single-stranded DNA or RNA molecules designed to bind to specific sequences of a target gene, in this case, the dystrophin gene.
Q: How do the antisense molecules work?
A: The antisense molecules bind to specific target sites in the dystrophin gene, inducing exon skipping and allowing for the production of a functional dystrophin protein despite mutations.
Q: What resources are available for searching related patents?
A: Resources include the Patent Public Search tool, Global Dossier, Common Citation Document (CCD), and international patent databases like WIPO's PATENTSCOPE.
Q: What is the economic impact of this patent?
A: The patent contributes to innovation and competitiveness in the pharmaceutical industry, with significant market and therapeutic implications for the treatment of genetic diseases.
Sources
- USPTO - Search for patents: https://www.uspto.gov/patents/search
- Google Patents - Antisense molecules and methods for treating pathologies: https://patents.google.com/patent/US10781450B2/en
- USPTO - Patent Claims Research Dataset: https://www.uspto.gov/ip-policy/economic-research/research-datasets/patent-claims-research-dataset
- U.S. Department of Commerce - U.S. Patent and Trademark Office: https://www.commerce.gov/bureaus-and-offices/uspto
Note: Additional sources may be cited within the text but are not listed here if they were not directly referenced.
