United States Patent 11,079,379: A Detailed Analysis
Overview of the Patent
The United States Patent 11,079,379, titled "Methods of treating transthyretin (TTR) mediated amyloidosis," was issued on August 3, 2021, and is assigned to Alnylam Pharmaceuticals, Inc. This patent focuses on innovative methods for treating transthyretin-mediated amyloidosis, a condition characterized by the accumulation of abnormal transthyretin (TTR) protein in various tissues, leading to severe neuropathy and other systemic issues.
Scope of the Patent
Target and Mechanism
The patent describes methods for reducing or arresting the increase in Neuropathy Impairment Score (NIS) or modified NIS (mNIS+7) in patients with TTR-mediated amyloidosis. This is achieved by administering an effective amount of a TTR-inhibiting composition, specifically RNA interference (RNAi) molecules such as small interfering RNA (siRNA) or double-stranded RNA (dsRNA)[1][2][4].
TTR-Inhibiting Compositions
The TTR-inhibiting compositions include patisiran and revusiran, both of which are siRNAs designed to target and silence the TTR gene. Patisiran is a well-known example, while revusiran is an siRNA conjugated to a Trivalent GalNAc carbohydrate cluster, enhancing its delivery and efficacy[1][2].
Claims of the Patent
Dosage and Administration
The patent claims methods where the amount of the TTR-inhibiting composition is adjusted based on the serum levels of TTR protein. If the TTR protein level is greater than 50 μg/ml, the dose is increased, and if it is below 50 μg/ml, the dose is decreased. This dynamic dosing strategy ensures optimal therapeutic efficacy while minimizing potential side effects[1].
RNAi Design
The dsRNA molecules described have an antisense strand with a region of complementarity to the TTR mRNA, typically between 19-24 nucleotides in length. These molecules can include single-stranded nucleotide overhangs to enhance stability and specificity[1].
Stability and Delivery
The patent also addresses the stability of the dsRNA molecules in cells and biological fluids, which is crucial for their therapeutic effectiveness. The methods described include modifications to improve the stability and delivery of these RNAi molecules[2].
Patent Landscape Analysis
Competitive Landscape
The patent landscape in the area of TTR-mediated amyloidosis treatment is highly competitive, with several key players, including Alnylam Pharmaceuticals, Inc. This company holds multiple patents related to TTR-inhibiting compositions and methods, indicating a strong focus on this therapeutic area[2][3].
Patent Expiration Dates
The patent 11,079,379 is set to expire on May 27, 2025. Other related patents, such as those covering compositions and methods for inhibiting TTR expression, expire on October 20, 2029. Understanding these expiration dates is crucial for strategic planning and potential generic or biosimilar development[2].
Innovation and White Space
Patent landscape analysis helps identify gaps in the current technology and potential areas for innovation. For TTR-mediated amyloidosis, there is ongoing research into new RNAi molecules and delivery systems, as well as combinations with other therapeutic approaches. Identifying these gaps can guide future R&D efforts and investment strategies[3].
Legal and Business Implications
Freedom to Operate
The patent claims and landscape analysis are essential for determining freedom to operate in this therapeutic area. Companies must ensure that their products and methods do not infringe on existing patents, and this analysis helps in navigating the complex patent landscape[3].
Licensing and Collaboration
The patent landscape can also highlight potential partners or licensees. For instance, Alnylam Pharmaceuticals, Inc. may collaborate with other companies or research institutions to further develop and commercialize TTR-inhibiting therapies[3].
Technical and Clinical Significance
Clinical Impact
The methods described in this patent have significant clinical implications, particularly for patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Reducing TTR protein levels can halt or reverse neuropathy progression, improving patient outcomes and quality of life[1][4].
Technological Advancements
The use of RNAi technology represents a major advancement in treating genetic diseases. The specificity and efficacy of these molecules make them a promising therapeutic approach for conditions that were previously difficult to treat[1][2].
Key Takeaways
- Innovative Therapeutic Approach: The patent introduces RNAi-based methods for treating TTR-mediated amyloidosis, offering a novel therapeutic strategy.
- Dynamic Dosing: The dosage adjustment based on serum TTR levels ensures optimal therapeutic efficacy and minimizes side effects.
- Competitive Landscape: Alnylam Pharmaceuticals, Inc. is a key player in this area, with multiple related patents.
- Patent Expiration: Understanding patent expiration dates is crucial for strategic planning and future development.
- Clinical Impact: The methods have significant clinical implications for patients with hATTR amyloidosis.
FAQs
What is the primary mechanism of action described in the patent?
The primary mechanism involves the use of RNA interference (RNAi) molecules, such as siRNA or dsRNA, to silence the TTR gene and reduce TTR protein levels.
Which companies are involved in the development of TTR-inhibiting therapies?
Alnylam Pharmaceuticals, Inc. is a leading company in this area, with multiple patents and products related to TTR-inhibiting compositions.
What is the significance of the dynamic dosing strategy?
The dynamic dosing strategy ensures that the TTR protein levels are maintained below a certain threshold (50 μg/ml), optimizing therapeutic efficacy and minimizing potential side effects.
When does the patent expire?
The patent 11,079,379 is set to expire on May 27, 2025.
How does this patent fit into the broader patent landscape for TTR-mediated amyloidosis?
This patent is part of a larger portfolio of patents held by Alnylam Pharmaceuticals, Inc., which collectively cover various aspects of TTR-inhibiting compositions and methods, indicating a strong focus on this therapeutic area.
Cited Sources
- US11079379B2 - Methods of treating transthyretin (TTR) mediated amyloidosis - Google Patents
- Generic Onpattro Availability - Drugs.com
- Patent Landscape Analysis – An Overview - Viện Công nghệ VinIT
- US11806360B2 - Compositions and methods for treating transthyretin (TTR) mediated amyloidosis - Google Patents
