United States Patent 10,266,827: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
The United States Patent 10,266,827, titled "Antisense oligonucleotides for inducing exon skipping and methods of use thereof," is a crucial patent in the field of genetic therapy, particularly for the treatment of Duchenne Muscular Dystrophy (DMD). This patent, assigned to The University of Western Australia, involves the use of antisense oligonucleotides to induce exon skipping in the dystrophin gene.
Background
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 approach by binding to specific regions of the dystrophin pre-mRNA, inducing exon skipping to restore the reading frame and produce a functional dystrophin protein[4].
Patent Overview
Patent Details
- Patent Number: 10,266,827
- Issue Date: September 24, 2019
- Inventors: Stephen Donald Wilton, Sue Fletcher, and Graham McClorey
- Assignee: The University of Western Australia[4][5].
Scope of the Patent
The patent covers antisense oligonucleotides designed to bind to selected target sites within the dystrophin gene, specifically to induce skipping of exon 53. This approach is aimed at treating DMD by restoring the dystrophin protein's function.
Target Sites and Mechanism
The antisense oligonucleotides are engineered to bind to specific nucleotide sequences involved in splicing, particularly targeting the regions around exon 53. By binding to these sites, the oligonucleotides prevent the inclusion of exon 53 in the mature mRNA, thereby bypassing the mutation and allowing the production of a shorter but functional dystrophin protein[4].
Claims of the Patent
The patent includes multiple claims that define the scope of the invention:
Independent Claims
- The patent claims cover the antisense oligonucleotides themselves, their methods of use, and the therapeutic applications.
- Specific sequences (SEQ ID NO: 1 to 214) are detailed, which are capable of binding to the target sites in the dystrophin gene[4].
Dependent Claims
- These claims further specify the characteristics of the antisense oligonucleotides, such as their length, composition, and the specific regions of the dystrophin gene they target.
- Methods of administration and treatment protocols are also included[4].
Patent Landscape
Related Patents
The University of Western Australia holds several related patents, including U.S. Patent Nos. 9,024,007, 9,994,851, 10,227,590, 10,421,966, and RE47,691. These patents collectively cover various aspects of antisense oligonucleotide therapy for DMD, including different target sites and methods of use[2][5].
Litigation and Disputes
There have been legal disputes involving these patents, particularly between Nippon Shinyaku Co., Ltd., and Sarepta Therapeutics, Inc. These disputes revolve around the interpretation of claim terms and the scope of the patents, highlighting the complexity and competitive nature of the field[1].
Patent Term and Expiration
The patent term for U.S. Patent 10,266,827 is set to expire on June 28, 2025. This expiration date is significant as it will impact the availability of generic versions of the drug and the competitive landscape in the market for DMD treatments[5].
Regulatory Approval
The drug associated with this patent, VYONDYS 53, was approved by the FDA on December 12, 2019. The FDA's approval process and subsequent regulatory reviews have validated the therapeutic efficacy and safety of the antisense oligonucleotides covered by this patent[2].
Impact on Innovation and Competition
The scope and claims of this patent influence the innovation landscape in genetic therapies. Narrower claims, as seen in this patent, can lead to a higher probability of grant and a shorter examination process, but they also limit the breadth of protection, potentially allowing for more competition and innovation in the field[3].
Metrics for Measuring Patent Scope
Studies have shown that metrics such as independent claim length and count can be used to measure patent scope. These metrics indicate that narrower claims, like those in U.S. Patent 10,266,827, are associated with a higher grant probability and shorter examination times, which can foster innovation by reducing litigation and licensing costs[3].
Key Takeaways
- Therapeutic Application: The patent covers antisense oligonucleotides for treating DMD by inducing exon skipping.
- Scope and Claims: The patent includes specific sequences and methods of use, with a focus on targeting exon 53.
- Patent Landscape: The University of Western Australia holds several related patents, and there have been legal disputes over claim interpretations.
- Regulatory Approval: VYONDYS 53, associated with this patent, was FDA-approved in 2019.
- Patent Term: The patent is set to expire on June 28, 2025.
FAQs
What is the primary therapeutic target of U.S. Patent 10,266,827?
The primary therapeutic target is the dystrophin gene, specifically inducing exon skipping to treat Duchenne Muscular Dystrophy.
Who is the assignee of U.S. Patent 10,266,827?
The University of Western Australia is the assignee of this patent.
What is the expiration date of U.S. Patent 10,266,827?
The patent is set to expire on June 28, 2025.
Has a generic version of VYONDYS 53 been approved?
No, there is currently no therapeutically equivalent generic version of VYONDYS 53 available in the United States.
What is the significance of the FDA approval of VYONDYS 53?
The FDA approval validates the therapeutic efficacy and safety of the antisense oligonucleotides covered by this patent, marking a significant milestone in the treatment of DMD.
Sources
- NIPPON SHINY AKU CO., LTD., Plaintiff, SAREPTA ... - U.S. District Court for the District of Delaware[1].
- FDA U.S. FOOD & DRUG OCT 2 2 2021 - Regulations.gov[2].
- Patent Claims and Patent Scope - Hoover Institution[3].
- US10266827B2 - Antisense oligonucleotides for inducing exon skipping and methods of use thereof - Google Patents[4].
- Generic Vyondys 53 Availability - Drugs.com[5].
