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Patent landscape, scope, and claims: |
United States Patent 9,133,461: A Detailed Analysis of Scope and Claims
Introduction
United States Patent 9,133,461, titled "Compositions and methods for inhibiting expression of the ALAS1 gene," is a significant patent in the field of genetic therapy, particularly for the treatment of acute hepatic porphyria (AHP). This patent, issued on September 15, 2015, is held by Alnylam Pharmaceuticals, Inc., and the Icahn School of Medicine at Mount Sinai.
Background of the Invention
The patent addresses the inherited porphyrias, a family of disorders resulting from deficient activity of specific enzymes in the heme biosynthetic pathway. One of the key enzymes involved is 5-aminolevulinic acid synthase 1 (ALAS1), and the patent focuses on methods to inhibit its expression using double-stranded ribonucleic acid (dsRNA) compositions[1].
Scope of the Patent
The scope of this patent is broad and encompasses several key areas:
dsRNA Compositions
The patent describes dsRNA compositions that target the ALAS1 gene. These compositions are designed to direct the sequence-specific degradation of mRNA through a process known as RNA interference (RNAi)[1].
Methods of Inhibition
The methods outlined include using these dsRNA compositions to alter, specifically inhibit, the expression of the ALAS1 gene. This inhibition is crucial for treating conditions like acute intermittent porphyria (AIP) and other porphyrias by reducing the accumulation of toxic porphyrin precursors[1].
Delivery Agents
The patent also covers carbohydrate conjugates as delivery agents for oligonucleotides, ensuring the effective delivery of the dsRNA compositions to the target cells[2].
Claims of the Patent
The patent includes multiple claims that define the scope of the invention:
Independent Claims
- The patent has several independent claims that describe the dsRNA compositions, their structure, and the methods for their use in inhibiting ALAS1 gene expression.
- These claims specify the sequence and structure of the dsRNA molecules, including the length and the specific nucleotide sequences[1].
Dependent Claims
- Dependent claims further detail the specific applications, dosages, and administration methods of the dsRNA compositions.
- These claims also cover various aspects of the therapeutic use, including the treatment of specific porphyrias and the reduction of symptoms associated with these disorders[1].
Patent Landscape
The patent landscape surrounding US 9,133,461 is complex and involves several related patents and exclusivities:
Related Patents
- Other patents, such as US 10,119,143 and US 10,125,364, also relate to compositions and methods for inhibiting the ALAS1 gene, often with overlapping but distinct claims[2].
- Patents like US 8,106,022 and US 8,828,956 focus on carbohydrate conjugates as delivery agents for oligonucleotides, which are integral to the delivery mechanism of the dsRNA compositions[2].
Patent Expiration Dates
- The patent US 9,133,461 is set to expire on November 30, 2033, which is consistent with the typical 20-year term from the date of filing[2].
Exclusivities
- The FDA has granted exclusivities related to this patent, including a new chemical entity exclusivity and an exclusivity for the treatment of adults with acute hepatic porphyria (AHP), which run concurrently with the patent term[2].
Therapeutic Implications
The therapeutic implications of this patent are significant:
Givosiran
- The siRNA therapeutic givosiran, which targets ALAS1, is a direct result of the research and development covered by this patent. Givosiran is used in the prophylaxis of acute hepatic porphyria and has shown promising results in clinical trials[4].
Treatment of Porphyrias
- By inhibiting the ALAS1 gene, this therapy reduces the production of toxic porphyrin precursors, providing a novel and effective treatment for patients with acute hepatic porphyria[1].
Industry Impact
The impact of this patent on the pharmaceutical industry is substantial:
Innovation in RNAi Therapeutics
- This patent represents a milestone in the development of RNA interference (RNAi) therapeutics, demonstrating the potential of dsRNA compositions in treating genetic disorders[1].
Competitive Landscape
- The patent landscape around ALAS1 inhibition is highly competitive, with multiple players involved in developing similar therapies. This patent provides Alnylam Pharmaceuticals with a strong position in this market[2].
Legal and Regulatory Aspects
The legal and regulatory aspects surrounding this patent are crucial:
Patent Scope and Quality
- The scope and quality of this patent have been subject to scrutiny, with metrics such as independent claim length and count being used to assess patent quality. Narrower claims, as seen in this patent, are often associated with a higher probability of grant and shorter examination processes[3].
FDA Exclusivities
- The FDA exclusivities granted for this therapy ensure that Alnylam Pharmaceuticals has exclusive marketing rights for a specified period, protecting their investment in research and development[2].
Key Takeaways
- Targeted Therapy: The patent describes a targeted therapy using dsRNA compositions to inhibit the ALAS1 gene, addressing a specific genetic disorder.
- Therapeutic Impact: The therapy has significant implications for treating acute hepatic porphyria and other porphyrias.
- Patent Landscape: The patent is part of a complex landscape involving multiple related patents and exclusivities.
- Industry Impact: The patent represents a significant innovation in RNAi therapeutics and positions Alnylam Pharmaceuticals strongly in the market.
- Legal and Regulatory: The patent's scope and quality are critical, and FDA exclusivities provide market protection.
FAQs
What is the primary focus of United States Patent 9,133,461?
The primary focus is on compositions and methods for inhibiting the expression of the ALAS1 gene using double-stranded ribonucleic acid (dsRNA) compositions.
What is the therapeutic application of this patent?
The therapeutic application is in the treatment of acute hepatic porphyria and other porphyrias by reducing the production of toxic porphyrin precursors.
Who are the inventors and assignees of this patent?
The inventors include Brian Bettencourt, Kevin Fitzgerald, William Querbes, Makiko Yasuda, and Robert J. Desnick. The assignees are Alnylam Pharmaceuticals, Inc., and the Icahn School of Medicine at Mount Sinai.
What is the expiration date of this patent?
The patent is set to expire on November 30, 2033.
What is the significance of FDA exclusivities related to this patent?
FDA exclusivities provide Alnylam Pharmaceuticals with exclusive marketing rights for a specified period, protecting their investment in research and development.
Cited Sources
- United States Patent and Trademark Office, "Compositions and methods for inhibiting expression of the ALAS1 gene," US 9,631,193 B2, April 25, 2017.
- Drugs.com, "Generic Givlaari Availability," November 6, 2024.
- Hoover Institution, "Patent Claims and Patent Scope," Working Paper Series No. 16001, August 18, 2016.
- DrugBank Online, "Givosiran: Uses, Interactions, Mechanism of Action," May 20, 2019.
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