United States Patent 8,110,560: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
United States Patent 8,110,560, titled "Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences," is a crucial patent in the field of genetic therapies, particularly for the treatment of spinal muscular atrophy (SMA). This patent is associated with the drug SPINRAZA (nusinersen sodium), which has been approved by the FDA for the treatment of SMA in pediatric and adult patients.
Background
SPINRAZA is a modified antisense oligonucleotide designed to treat SMA by targeting the SMN2 gene. SMA is a genetic disorder caused by mutations in the SMN1 gene, which leads to the deficiency of the survival motor neuron (SMN) protein. The SMN2 gene, while similar, produces a truncated and non-functional SMN protein due to a splicing defect. The patent describes methods and compositions to block the inhibitory effect of newly identified intronic inhibitory sequences in the SMN2 gene, thereby increasing the production of functional SMN protein[4].
Scope of the Patent
The scope of U.S. Patent 8,110,560 encompasses several key aspects:
Methods and Compositions
The patent covers methods for treating SMA by administering antisense oligonucleotides that target specific splice site inhibitory sequences within the SMN2 gene. These oligonucleotides are designed to modify the splicing of the SMN2 pre-mRNA, leading to the inclusion of exon 7 and the production of a functional SMN protein[4].
Targeting SMN2 Splice Site Inhibitory Sequences
The patent details the identification and targeting of specific intronic inhibitory sequences that prevent the proper splicing of the SMN2 gene. By blocking these sequences, the oligonucleotides enable the correct splicing and translation of the SMN2 gene into a functional SMN protein[4].
Therapeutic Applications
The patent includes therapeutic applications for the treatment of SMA, emphasizing the potential to improve the quality of life and survival rates of patients with this genetic disorder.
Claims of the Patent
The claims of U.S. Patent 8,110,560 are critical in defining the scope of protection for the invention. Here are some key claims:
Claim 1
Claim 1 typically sets the broadest scope for the invention, often describing the general method or composition. For this patent, Claim 1 might involve the administration of an antisense oligonucleotide that targets a specific sequence within the SMN2 gene to increase the production of functional SMN protein[4].
Dependent Claims
Dependent claims further narrow down the scope by adding specific details or limitations to the independent claims. These might include the specific sequences targeted, the type of oligonucleotides used, or the methods of administration.
Patent Landscape
The patent landscape surrounding U.S. Patent 8,110,560 is complex and involves several key players and patents.
Related Patents
Other patents related to SPINRAZA and SMA treatment include U.S. Patent Nos. 7,838,657 and 8,361,977. These patents also cover aspects of the drug's development, manufacturing, and use, highlighting the extensive intellectual property portfolio associated with SPINRAZA[2].
Patent Term Extension
The FDA has determined the regulatory review period for SPINRAZA, which is crucial for patent term extension. The regulatory review period for SPINRAZA was 1,891 days, with 1,799 days in the testing phase and 92 days in the approval phase. This determination allows for potential patent term extensions under the Drug Price Competition and Patent Term Restoration Act of 1984[2].
Patent Analytics and Claim Coverage
To manage and analyze the extensive patent portfolio associated with SPINRAZA, companies often use patent analytics tools. These tools help in tracking patents by claims and scope concepts, identifying gaps or opportunities in intellectual property protection, and ensuring that all claims cover the intended technology[3].
Regulatory and Legal Aspects
The approval and regulatory review process for SPINRAZA involved significant interactions between the FDA and the USPTO. Here are some key regulatory and legal aspects:
FDA Approval
SPINRAZA was approved by the FDA on December 23, 2016, marking the first permitted commercial marketing or use of the product. This approval was a critical milestone in the regulatory review process[2].
Patent Term Restoration
The FDA's determination of the regulatory review period is essential for calculating the potential patent term extension. The applicants for U.S. Patent 8,110,560 sought various lengths of patent term extension, which were evaluated based on statutory limitations and the regulatory review period[2].
Industry Impact and Expert Insights
The approval and patent protection of SPINRAZA have had a significant impact on the treatment landscape for SMA.
Treatment Outcomes
Clinical trials and real-world data have shown that SPINRAZA significantly improves motor function and survival rates in SMA patients. This has transformed the management of SMA, offering new hope for patients and families affected by this condition.
Market Dominance
The strong patent protection surrounding SPINRAZA has contributed to its market dominance in the SMA treatment space. Biogen, the manufacturer, has leveraged this intellectual property to ensure exclusivity and protect its investment in research and development.
Key Takeaways
- Innovative Therapy: U.S. Patent 8,110,560 covers an innovative antisense oligonucleotide therapy for SMA, targeting the SMN2 gene to increase functional SMN protein production.
- Extensive Patent Portfolio: The patent is part of a broader intellectual property portfolio related to SPINRAZA, including other patents covering various aspects of the drug.
- Regulatory Approval: The FDA approval and subsequent patent term extension applications highlight the complex regulatory landscape surrounding this drug.
- Industry Impact: SPINRAZA has significantly improved treatment outcomes for SMA patients, and its patent protection has contributed to its market dominance.
FAQs
What is the primary focus of U.S. Patent 8,110,560?
The primary focus of U.S. Patent 8,110,560 is on methods and compositions for treating spinal muscular atrophy (SMA) by targeting the SMN2 gene with antisense oligonucleotides.
How does SPINRAZA work?
SPINRAZA works by administering antisense oligonucleotides that target specific splice site inhibitory sequences within the SMN2 gene, leading to the inclusion of exon 7 and the production of a functional SMN protein.
What is the significance of the FDA's determination of the regulatory review period for SPINRAZA?
The FDA's determination of the regulatory review period is crucial for calculating the potential patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984.
How does patent analytics help in managing the intellectual property related to SPINRAZA?
Patent analytics helps in tracking patents by claims and scope concepts, identifying gaps or opportunities in intellectual property protection, and ensuring that all claims cover the intended technology.
What is the impact of U.S. Patent 8,110,560 on the treatment of SMA?
U.S. Patent 8,110,560 has led to the development of SPINRAZA, which has significantly improved motor function and survival rates in SMA patients, transforming the management of this genetic disorder.
Sources
- FDA Document: FEB l 7 2017 - Regulations.gov
- Federal Register: Volume 86 Issue 208 (Monday, November 1, 2021)
- Patent Analytics: Intellectual Property Law - SLWIP
- Google Patents: US8110560B2 - Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
- Regulations.gov: Patent Extension Application for SPINRAZA® U.S. Patent No. 8,110,560
