United States Patent 8,980,853: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
United States Patent 8,980,853, titled "Compositions and methods for modulation of SMN2 splicing in a subject," is a significant patent in the field of genetic therapy, particularly for the treatment of spinal muscular atrophy (SMA). This patent, held by Biogen MA Inc. and Cold Spring Harbor Laboratory, is part of a broader portfolio related to the drug SPINRAZA (nusinersen sodium).
Background
Spinal muscular atrophy is a genetic disorder caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the loss of motor neurons and progressive muscle weakness. The SMN2 gene, while similar, produces a truncated and less functional protein due to alternative splicing. The patent focuses on modulating the splicing of SMN2 mRNA to produce a more functional SMN protein.
Patent Scope and Claims
Invention Overview
The patent describes compositions and methods for modulating the splicing of SMN2 mRNA. This is achieved through the use of antisense oligonucleotides, which are designed to bind to specific sequences of the SMN2 pre-mRNA and alter the splicing process to produce a more functional SMN protein[4].
Key Claims
- Composition Claims: The patent includes claims for antisense oligonucleotides and their derivatives, which are designed to modulate SMN2 splicing. These oligonucleotides can be administered via various routes, including intrathecal injection[4].
- Method Claims: The patent claims methods for treating SMA by administering these antisense oligonucleotides to subjects. These methods involve the use of specific doses and administration protocols to achieve optimal splicing modulation[4].
Claim Language and Scope
The claims are detailed and specific, focusing on the chemical structure of the oligonucleotides, their administration methods, and the therapeutic outcomes. For example, the patent claims include descriptions of 2'-MOE (2'-O-(2-methoxyethyl)) modified nucleosides, which are part of the antisense oligonucleotides[4].
Patent Landscape
Related Patents
The patent is part of a larger family of patents related to SPINRAZA, including U.S. Patent Nos. 7,838,657, 8,110,560, and 8,361,977. These patents collectively cover various aspects of the drug's composition, method of use, and manufacturing processes[2][5].
Regulatory Review and Patent Term Extension
The FDA approved SPINRAZA on December 23, 2016, marking the first permitted commercial marketing or use of the product. The regulatory review period for SPINRAZA was determined to be 1,891 days, with 1,799 days in the testing phase and 92 days in the approval phase. This led to applications for patent term extension under 35 U.S.C. ยง 156, with the USPTO considering these extensions to ensure the patents remain in force for a period that reflects the time spent in regulatory review[2][5].
Patent Term and Expiration
The original expiration date of U.S. Patent 8,980,853 would have been based on its filing and issuance dates. However, due to the regulatory review period, the patent term has been extended. The maximum extension allowed is up to five years from the original expiration date, which in this case would be extended from May 27, 2030, to May 27, 2035, subject to statutory limitations[2][5].
Impact on Innovation and Competition
The patent's scope and claims have significant implications for innovation and competition in the field of genetic therapies. The specificity of the claims ensures that Biogen MA Inc. and Cold Spring Harbor Laboratory have exclusive rights to the use of these particular antisense oligonucleotides for SMA treatment, which can influence the development of similar therapies by other companies.
Licensing and Litigation
The breadth and clarity of the patent claims can affect licensing agreements and potential litigation. Narrower, well-defined claims, as seen in this patent, can reduce the likelihood of disputes and make it clearer for other innovators what is protected and what is not[3].
Expert Insights
"The approval of SPINRAZA and the associated patent extensions highlight the complex interplay between regulatory approval processes and patent law. This ensures that innovators have sufficient time to recoup their investments while also encouraging continued innovation in critical therapeutic areas," said Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research at the FDA[2].
Statistics and Market Impact
- Approval Date: December 23, 2016
- Regulatory Review Period: 1,891 days
- Testing Phase: 1,799 days
- Approval Phase: 92 days
- Market Impact: SPINRAZA has been a groundbreaking treatment for SMA, significantly improving the quality of life for patients. The patent extensions ensure that Biogen MA Inc. can continue to invest in research and development while maintaining market exclusivity[2][5].
Conclusion
United States Patent 8,980,853 is a pivotal patent in the treatment of spinal muscular atrophy, offering a detailed and specific approach to modulating SMN2 splicing. The patent's scope and claims are well-defined, ensuring strong protection for the innovators while also providing a clear landscape for other researchers and companies.
Key Takeaways
- Specific Claims: The patent includes detailed claims for antisense oligonucleotides and their methods of use.
- Regulatory Review: The FDA-approved regulatory review period significantly impacts the patent term extension.
- Market Impact: SPINRAZA has revolutionized SMA treatment, and the patent ensures continued innovation and market exclusivity.
- Innovation and Competition: The patent's scope influences the development of similar therapies and licensing agreements.
FAQs
What is the primary focus of U.S. Patent 8,980,853?
The primary focus is on compositions and methods for modulating the splicing of SMN2 mRNA to treat spinal muscular atrophy.
How are the antisense oligonucleotides administered?
The oligonucleotides can be administered via various routes, including intrathecal injection.
What is the significance of the regulatory review period for this patent?
The regulatory review period of 1,891 days led to a patent term extension, ensuring the patent remains in force for a period reflecting the time spent in regulatory review.
How does this patent impact innovation in genetic therapies?
The patent's specificity ensures exclusive rights for Biogen MA Inc. and Cold Spring Harbor Laboratory, influencing the development of similar therapies by other companies.
What is the current status of the patent term for U.S. Patent 8,980,853?
The patent term has been extended up to May 27, 2035, subject to statutory limitations.
Cited Sources
- FDA Application for Patent Term Extension: Regulations.gov
- FDA Determination of Regulatory Review Period: Regulations.gov
- Patent Claims and Patent Scope: SSRN
- Compositions and methods for modulation of SMN2 splicing: Google Patents
- Federal Register Notice on Patent Term Extension: Government Publishing Office
