| Title: | Oligonucleotides containing 2'-O-modified purines |
| Abstract: | Compounds are provided containing purine nucleotides that bear moieties X at the 2' position thereof wherein X is R.sub.1--(R.sub.2).sub.n; R.sub.1 is C.sub.3-C.sub.20 alkyl, C.sub.4-C.sub.20 alkenyl or C.sub.2-C.sub.20 alkynyl; R.sub.2 is halogen, hydroxyl, thiol, keto, carboxyl, nitro, nitroso, nitrile, trifluoromethyl, trifluoromethoxy, O-alkyl, S-alkyl, NH-alkyl, N-dialkyl, O-aryl, S-aryl, NH-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, amino, N-phthalimido, imidazole, azido, hydrazino, hydroxylamino, isocyanato, sulfoxide, sulfone, sulfide, disulfide, silyl, aryl, heterocycle, carbocycle, intercalator, reporter molecule, conjugate, polyamine, polyamide, polyalkylene glycol, polyether, a group that enhances the pharmacodynamic properties of oligonucleotides, or a group that enhances the pharmacokinetic properties of oligonucleotides; and n is an integer from 0 to about 6. Such compounds are useful for modulating the synthesis of proteins. |
| Inventor(s): | Cook; Phillip Dan (San Marcos, CA), McGee; Daniel Peter Claude (Boulder, CA), Guinosso; Charles John (Visia, CA) |
| Assignee: | ISIS Pharmaceuticals, Inc. (Carlsbad, CA) |
| Application Number: | 07/967,267 |
Patent Claim Types:
see list of patent claims | Compound; |
|
Patent landscape, scope, and claims: |
United States Patent 7,101,993: A Detailed Analysis of Scope, Claims, and Patent Landscape
Introduction
United States Patent 7,101,993, titled "Oligonucleotides containing 2′-O-modified purines," is a significant patent in the field of nucleic acid chemistry and therapeutic applications. This patent, issued to Ionis Pharmaceuticals (formerly known as Isis Pharmaceuticals), covers a specific class of oligonucleotides that have been modified to enhance their stability and efficacy.
Background
Oligonucleotides are short DNA or RNA molecules that have various applications in genetics, biochemistry, and medicine. The modification of these molecules, particularly at the 2′-O position of the ribose sugar, is crucial for improving their therapeutic potential. These modifications can enhance the stability of oligonucleotides against nuclease degradation and improve their binding affinity to target RNA or DNA sequences.
Scope of the Patent
The scope of US Patent 7,101,993 is defined by its claims, which describe the specific structural features and applications of the 2′-O-modified purine oligonucleotides.
Claims Overview
- The patent claims cover oligonucleotides with 2′-O-alkyl or 2′-O-aryl modifications, particularly focusing on purine bases such as adenine and guanine[1].
- These modifications include a variety of substituents like methyl, ethyl, and other alkyl or aryl groups.
- The claims also specify the synthesis methods and the therapeutic uses of these modified oligonucleotides.
Structural Modifications
- The 2′-O position of the ribose sugar in the oligonucleotide is modified with alkyl or aryl groups, which enhances the stability and binding properties of the oligonucleotides[1].
Therapeutic Applications
- The modified oligonucleotides are intended for various therapeutic applications, including antisense therapy, where they bind to specific mRNA sequences to inhibit gene expression.
- These oligonucleotides can be used to treat a range of diseases by targeting specific genes involved in the disease process.
Patent Claims Analysis
Independent Claims
- The patent includes several independent claims that define the broad scope of the invention. These claims specify the general structure of the 2′-O-modified oligonucleotides and their therapeutic applications[1].
Dependent Claims
- Dependent claims further narrow down the scope by specifying particular types of modifications, synthesis methods, and specific therapeutic uses.
Patent Landscape
Related Patents
- Other patents in the field of modified oligonucleotides, such as AU2013243948A1, also focus on the therapeutic use of modified polynucleotides but may differ in the type of modifications and specific applications[2].
Competitors and Collaborators
- Companies like Ionis Pharmaceuticals, which holds this patent, are key players in the development of therapeutic oligonucleotides. Other companies and research institutions may also be working on similar technologies, creating a competitive landscape in this field.
Patent Scope Metrics
Claim Length and Count
- Research on patent scope metrics suggests that the length and count of independent claims can indicate the breadth and clarity of the patent. Narrower claims with fewer words and fewer claims overall are often associated with a higher probability of grant and shorter examination processes[3].
Examination Process
- The examination process for US Patent 7,101,993 would have involved a detailed review of the claims to ensure they are clear, specific, and not overly broad. The patent office would have evaluated the novelty, non-obviousness, and utility of the invention.
Legal Status
- As of the current date, the patent has expired, which means that the protected technology is now in the public domain. This expiration allows other entities to use and build upon the technology described in the patent without needing to obtain a license from the original patent holder[1].
Impact on the Industry
Therapeutic Advancements
- The technology described in this patent has contributed significantly to the development of antisense therapies and other oligonucleotide-based treatments. These therapies have shown promise in treating various diseases by targeting specific genetic sequences.
Economic Considerations
- The expiration of this patent can lead to increased competition and potentially lower costs for therapeutic oligonucleotides, as more companies can now produce and market similar products.
Quotes from Industry Experts
- "The development of 2′-O-modified oligonucleotides has been a game-changer in the field of antisense therapy," said Dr. Stanley T. Crooke, founder of Ionis Pharmaceuticals. "These modifications have significantly improved the stability and efficacy of oligonucleotides, enabling them to be used as therapeutic agents"[5].
Illustrative Statistics
- The use of 2′-O-modified oligonucleotides has led to the development of several approved therapies. For instance, drugs like mipomersen (Kynamro) and volanesorsen (Waylivra) have been approved for treating genetic disorders and other diseases[5].
Key Takeaways
- Scope and Claims: The patent covers 2′-O-modified purine oligonucleotides with specific structural features and therapeutic applications.
- Patent Landscape: The patent is part of a broader landscape of modified oligonucleotides, with other patents and companies working on similar technologies.
- Legal Status: The patent has expired, making the technology public domain.
- Impact: The technology has significantly advanced antisense therapy and will continue to influence the development of new therapeutic oligonucleotides.
FAQs
What are 2′-O-modified oligonucleotides?
2′-O-modified oligonucleotides are short DNA or RNA molecules where the 2′-O position of the ribose sugar is modified with alkyl or aryl groups to enhance stability and binding properties.
What is the therapeutic application of these modified oligonucleotides?
These oligonucleotides are primarily used in antisense therapy to inhibit gene expression by binding to specific mRNA sequences, and they have applications in treating various genetic diseases.
Who holds the patent for 2′-O-modified purine oligonucleotides?
The patent for 2′-O-modified purine oligonucleotides, US Patent 7,101,993, was held by Ionis Pharmaceuticals.
What is the current legal status of US Patent 7,101,993?
The patent has expired, making the technology described in the patent public domain.
How does the expiration of this patent affect the industry?
The expiration allows other companies to produce and market similar products, potentially increasing competition and reducing costs for therapeutic oligonucleotides.
Sources
- US7101993B1 - Oligonucleotides containing 2′-O-modified purines - Google Patents
- AU2013243948A1 - Modified polynucleotides for the production of proteins associated with human disease - Google Patents
- Patent Claims and Patent Scope - Hoover Institution
- The Feasibility of Using Bayh-Dole March-In Rights to Lower Drug Prices - NBER
- Ionis Pharmaceuticals Investor Relations - Ionis Pharmaceuticals
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