United States Patent 9,994,851: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
The United States Patent 9,994,851, titled "Antisense oligonucleotides for inducing exon skipping and methods of use thereof," is a significant patent in the field of genetic therapy, particularly for the treatment of Duchenne muscular dystrophy (DMD). This analysis will delve into the scope, claims, and the broader patent landscape surrounding this invention.
Background of the Patent
The patent, issued on June 12, 2018, is held by Sarepta Therapeutics, Inc. and the University of Western Australia. It focuses on the use of antisense oligonucleotides to induce exon skipping in the dystrophin gene, a crucial approach for treating genetic disorders like DMD[1].
Scope of the Patent
The scope of the patent is centered around the design, use, and administration of antisense oligonucleotides that target specific sequences within the dystrophin gene. These oligonucleotides are engineered to bind to pre-mRNA sequences, thereby inhibiting the normal splicing process and causing the splicing machinery to bypass mutated exons. This approach aims to restore the production of a functional dystrophin protein in patients with DMD[1].
Targeted Sequences
The patent specifies the use of antisense molecules that target particular nucleotide sequences involved in splicing within the dystrophin gene. These sequences are detailed in the patent, including the specific base sequences and their annealing coordinates, which are critical for effective exon skipping[1].
Therapeutic Applications
The patent covers the therapeutic use of these antisense oligonucleotides for treating genetic diseases, particularly DMD. It outlines methods for administering these molecules to patients, either as a treatment to alleviate symptoms or as a prophylactic measure to prevent the onset of the disease[1].
Claims of the Patent
The patent includes several claims that define the scope of the invention:
Claim 1 and Claim 2
These claims specify the composition of the antisense oligonucleotides, requiring them to have at least 12 consecutive bases of a particular sequence (CUG AAG GUG UUC UUG UAC UUC AUC C), where uracil bases are replaced with thymine bases. This ensures that the oligonucleotides are designed to target the dystrophin gene effectively[2].
Method Claims
The patent also includes method claims that describe the process of treating patients with genetic diseases by administering these antisense oligonucleotides. These methods involve selecting appropriate antisense molecules and delivering them to patients in a form suitable for therapeutic use[1].
Patent Landscape
Litigation and Infringement
The patent has been involved in significant litigation, particularly between Sarepta Therapeutics and Nippon Shinyaku Co., Ltd., and NS Pharma, Inc. Sarepta sought summary judgment for infringement of the '851 patent and other related patents, which was granted by the U.S. District Court for the District of Delaware. This ruling underscores the importance of this patent in the field of genetic therapy[2].
Related Patents and Applications
The '851 patent is part of a broader family of patents related to antisense oligonucleotides and exon skipping. The Global Dossier service provided by the USPTO allows users to view the patent family and related applications filed at participating IP offices, highlighting the interconnected nature of these inventions[4].
Impact on Genetic Therapy
Treatment of Duchenne Muscular Dystrophy
The '851 patent has significant implications for the treatment of DMD. By enabling the skipping of mutated exons in the dystrophin gene, these antisense oligonucleotides offer a promising therapeutic approach for restoring dystrophin production in affected patients[1].
Broader Applications
The technology described in this patent can be applied to other genetic disorders where exon skipping is a viable therapeutic strategy. This expands the potential impact of the invention beyond DMD to a range of genetic diseases[1].
Metrics for Patent Scope
The scope of the '851 patent can be analyzed using metrics such as independent claim length and independent claim count. These metrics help in understanding the breadth and clarity of the patent claims, which are crucial for assessing patent quality and potential litigation risks[3].
Conclusion
The United States Patent 9,994,851 is a pivotal invention in the field of genetic therapy, particularly for the treatment of Duchenne muscular dystrophy. Its scope encompasses the design, use, and administration of antisense oligonucleotides for inducing exon skipping in the dystrophin gene. The patent's claims are specific and well-defined, and its involvement in litigation highlights its importance in the patent landscape.
Key Takeaways
- Specific Targeting: The patent involves antisense oligonucleotides that target specific sequences within the dystrophin gene to induce exon skipping.
- Therapeutic Applications: It covers the therapeutic use for treating genetic diseases, particularly DMD.
- Litigation: The patent has been involved in significant litigation regarding infringement.
- Broader Impact: The technology has potential applications beyond DMD to other genetic disorders.
- Patent Metrics: The scope can be analyzed using metrics like independent claim length and count.
FAQs
What is the primary focus of the United States Patent 9,994,851?
The primary focus is on the use of antisense oligonucleotides to induce exon skipping in the dystrophin gene for treating genetic disorders like Duchenne muscular dystrophy.
What are the specific sequences targeted by the antisense oligonucleotides?
The sequences targeted include at least 12 consecutive bases of CUG AAG GUG UUC UUG UAC UUC AUC C, where uracil bases are replaced with thymine bases.
What is the significance of the litigation involving this patent?
The litigation, which resulted in a summary judgment for infringement, highlights the importance of this patent in the field of genetic therapy and its potential impact on other companies developing similar technologies.
How does this patent impact the treatment of Duchenne muscular dystrophy?
It offers a promising therapeutic approach by enabling the skipping of mutated exons in the dystrophin gene, thereby restoring dystrophin production in affected patients.
What are the broader applications of the technology described in this patent?
The technology can be applied to other genetic disorders where exon skipping is a viable therapeutic strategy, expanding its potential impact beyond DMD.
Sources
- US9994851B2 - Antisense oligonucleotides for inducing exon skipping and methods of use thereof - Google Patents
- United States District Court for the District of Delaware - Sarepta Therapeutics, Inc. and the University of Western Australia vs. Nippon Shinyaku Co., Ltd., and NS Pharma, Inc.
- Patent Claims and Patent Scope - SSRN
- Search for patents - USPTO - United States Patent and Trademark Office
