United States Patent 11,129,910: Analysis of Claims and Patent Landscape

Summary: United States Patent 11,129,910, granted on November 30, 2021, to CSL Behring LLC, claims a method for treating or preventing thrombotic events in individuals with specific genetic deficiencies. The patent focuses on the prophylactic administration of a Factor VIII concentrate. Analysis of the patent's claims reveals a narrow scope, primarily targeting a specific patient population and a defined treatment regimen. The patent landscape surrounding Factor VIII concentrates is characterized by extensive existing intellectual property, with numerous patents covering various formulations, production methods, and therapeutic uses of this essential clotting factor. This analysis examines the core claims of U.S. Patent 11,129,910 and assesses its position within this competitive patent environment.

What Are the Core Claims of U.S. Patent 11,129,910?

U.S. Patent 11,129,910, titled "Prophylactic Administration of Factor VIII Concentrates," asserts a method for preventing thrombotic events. The patent is divided into several independent and dependent claims. The central focus of the independent claims, specifically Claim 1, defines a method comprising:

Administering a Factor VIII concentrate to a subject.
The subject has a genetic deficiency that predisposes them to thrombotic events.
The Factor VIII concentrate is administered at a dose and frequency sufficient to maintain a plasma Factor VIII activity level of at least 1% of normal.
The administration is prophylactic, meaning it is performed before the occurrence of a thrombotic event.

Dependent claims further refine these parameters. For instance, they may specify particular genetic deficiencies, such as those involving protein C deficiency or antithrombin deficiency. Other dependent claims may delineate specific dosage ranges or treatment durations.

The patent is distinct in its explicit targeting of thrombotic events in individuals with specific genetic Factor VIII deficiencies. While Factor VIII concentrates are widely used for treating hemophilia A (Factor VIII deficiency), this patent's novelty lies in its application to a distinct set of genetic disorders where Factor VIII deficiency contributes to a pro-thrombotic state, rather than a bleeding disorder. This distinction is crucial. Standard therapeutic use of Factor VIII is to replace the missing clotting factor to prevent bleeding. This patent proposes using Factor VIII to prevent clotting in individuals with a different underlying genetic condition where the deficiency of Factor VIII plays a role in the development of thrombosis.

What is the Technical Basis for the Claims?

The technical basis for U.S. Patent 11,129,910 rests on the complex interplay between Factor VIII and the coagulation cascade, particularly concerning the regulation of thrombosis. Normally, Factor VIII is a cofactor in the activation of Factor X by Factor IXa, a critical step in the intrinsic pathway of coagulation. Its deficiency (hemophilia A) leads to impaired clot formation and bleeding.

However, the patent addresses a less understood phenomenon where certain genetic defects, beyond simple Factor VIII deficiency, can paradoxically lead to thrombotic tendencies. These genetic deficiencies might include conditions like protein C deficiency or antithrombin deficiency. In these scenarios, the anticoagulant pathways are compromised, leading to an increased risk of blood clots. The patent hypothesizes that in individuals with these specific genetic thrombophilic conditions, a suboptimal level of Factor VIII activity, even if not severe enough to cause bleeding, might contribute to the pro-thrombotic state by subtly altering the balance of coagulation.

The technical premise is that by prophylactically administering a Factor VIII concentrate to elevate plasma Factor VIII activity to a minimum threshold (at least 1% of normal), the pro-coagulant potential of the blood is rebalanced. This rebalancing is theorized to counteract the hypercoagulability arising from the underlying genetic thrombophilia, thereby preventing the formation of dangerous blood clots (thrombotic events) such as deep vein thrombosis (DVT) or pulmonary embolism (PE).

The "at least 1% of normal" threshold is a key technical specification. This level is generally insufficient to correct severe Factor VIII deficiency seen in hemophilia A, which often requires much higher levels. This highlights the patent's focus on a therapeutic window where Factor VIII administration addresses thrombophilia rather than hemophilia.

What is the Scope of the Claims?

The scope of U.S. Patent 11,129,910 is defined by its claims and the accompanying patent prosecution history. Claim 1, as the broadest independent claim, sets the general boundaries.

Key limitations defining the scope:

Target Subject: The method is applied to a "subject having a genetic deficiency that predisposes to thrombotic events." This excludes subjects with genetic deficiencies that primarily cause bleeding disorders, like standard hemophilia A.
Condition Treated/Prevented: The claims are directed towards preventing "thrombotic events." This is distinct from preventing bleeding events.
Treatment Modality: The method requires the "prophylactic administration of a Factor VIII concentrate." This excludes other therapeutic interventions for thrombophilia.
Dosage Metric: The administration must achieve and maintain "a plasma Factor VIII activity level of at least 1% of normal." This is a specific, measurable threshold.

The scope is further narrowed by dependent claims that may specify:

Particular genetic deficiencies (e.g., protein C deficiency, antithrombin deficiency).
Specific types of thrombotic events to be prevented (e.g., DVT, PE, arterial thrombosis).
The type of Factor VIII concentrate used (e.g., plasma-derived, recombinant).

The narrowness of the claims is a strategic decision, often made to navigate existing prior art and secure patentability. By precisely defining the patient population and the therapeutic objective, the patent aims to carve out a defensible niche within the broader field of coagulation factor therapy.

How Does This Patent Relate to Existing Factor VIII Therapies?

Existing Factor VIII therapies primarily address hemophilia A, a bleeding disorder caused by a deficiency in functional Factor VIII. These therapies involve replacing the missing Factor VIII to restore hemostasis and prevent bleeding episodes. Patents in this space have historically focused on:

Production Methods: Improving the efficiency, purity, and yield of Factor VIII concentrates from plasma or via recombinant DNA technology. Examples include patents related to cell culture techniques, purification processes, and genetic engineering of expression vectors.
Formulations: Developing stable, long-acting, or easily administered Factor VIII formulations. This includes patents on lyophilized powders, liquid formulations, and excipients that enhance stability or pharmacokinetic profiles.
Therapeutic Uses: Claims related to the treatment of hemophilia A, including specific dosing regimens, treatment protocols for acute bleeds, and routine prophylaxis in hemophiliacs.

U.S. Patent 11,129,910 diverges from these established areas by:

Targeting Thrombosis, Not Bleeding: Its primary objective is to prevent blood clots, not to stop bleeding.
Targeting Specific Genetic Thrombophilias: It addresses a patient group distinct from classic hemophiliacs.
Utilizing a Sub-Therapeutic Level for Hemophilia: The target Factor VIII activity level (at least 1%) is often insufficient for the prophylactic treatment of hemophilia A, which typically aims for higher target levels to prevent bleeding.

This patent therefore does not directly overlap with patents covering the standard treatment of hemophilia A. It attempts to leverage Factor VIII's pro-coagulant properties for an entirely different pathological condition, creating a unique therapeutic and intellectual property space.

What is the Competitive Patent Landscape for Factor VIII Concentrates?

The patent landscape for Factor VIII concentrates is mature and densely populated, with a long history of innovation and patent filings. Key players, including CSL Behring, Bayer, Pfizer, and Shire (now Takeda), hold numerous patents covering various aspects of Factor VIII technology.

Key areas of existing patents include:

Early Patents: Covered basic methods of isolating and purifying Factor VIII from plasma, essential for the initial development of hemophilia treatments.
Recombinant Factor VIII: A significant wave of patents emerged with the advent of recombinant DNA technology, covering specific DNA sequences, expression vectors, host cells, and purification methods for producing recombinant Factor VIII (e.g., Kogenate FS, Advate).
Improved Formulations: Patents exist for various formulations designed to enhance stability, shelf-life, and ease of administration, including freeze-dried formulations and those with improved excipient compositions.
Extended Half-Life Products: More recent innovation has focused on extending the half-life of Factor VIII, reducing the frequency of infusions. This involves patents on conjugation technologies (e.g., PEGylation, Fc fusion) or modifications to the Factor VIII molecule itself (e.g., B-domain deletion variants).
Biosimilulators: As originator patents expire, there is an increasing number of patents related to biosimilar versions of Factor VIII, focusing on demonstrating similarity in terms of manufacturing, purity, and biological activity.

Considerations for U.S. Patent 11,129,910 within this landscape:

Prior Art: The patent examiner would have thoroughly reviewed existing patents and scientific literature related to Factor VIII therapy and thrombophilia. The patentability of U.S. Patent 11,129,910 hinges on demonstrating that its claimed method is novel and non-obvious over this prior art. The specific combination of genetic deficiency, thrombotic event prevention, and a low Factor VIII activity threshold is likely the basis for its differentiation.
Freedom to Operate (FTO): Companies seeking to implement the method claimed by U.S. Patent 11,129,910 would need to conduct thorough FTO analyses. This involves examining whether their specific Factor VIII concentrate, dosage regimen, and target patient population infringe upon any existing valid patents. Given the density of Factor VIII patents, FTO is a critical hurdle.
Enforcement and Litigation: Disputes over Factor VIII patents are not uncommon. The value of U.S. Patent 11,129,910 would depend on its enforceability against competitors and its ability to deter market entry for similar therapeutic approaches.

The broad existing patent coverage means that U.S. Patent 11,129,910 likely represents a specific refinement or application rather than a foundational patent in the Factor VIII field. Its commercial viability depends on the clinical efficacy of the proposed treatment and its ability to navigate the existing IP thicket.

What are the Potential Implications for R&D and Investment?

The issuance of U.S. Patent 11,129,910 suggests a potential new therapeutic avenue for CSL Behring LLC, a company with significant expertise in plasma-derived therapies, including Factor VIII.

Implications for R&D:

Niche Indication Exploration: The patent encourages further research into the role of Factor VIII in specific thrombophilic genetic disorders. This could spur clinical trials to validate the efficacy and safety of this prophylactic approach in targeted patient populations.
Biomarker Development: Identifying patients most likely to benefit from this therapy could drive research into biomarkers that predict thrombotic risk in individuals with these genetic deficiencies and identify those who might respond to Factor VIII intervention.
Dose Optimization Studies: While "at least 1%" is claimed, further R&D might focus on optimizing the precise dose and frequency to maximize efficacy while minimizing potential risks, particularly in the context of thrombotic disorders.
Combination Therapies: Research might explore combining Factor VIII administration with other anticoagulant or antithrombotic agents for enhanced protection in high-risk individuals.

Implications for Investment:

Niche Market Opportunity: If clinically validated, this approach could open a new, albeit potentially smaller, market segment within the broader coagulation disorders landscape. Investors might evaluate the size of the patient population with the specific genetic deficiencies targeted and the unmet medical need.
Intellectual Property Strength: The value of CSL Behring's investment in this patent will depend on its ability to defend it against potential infringers. The narrowness of the claims, while aiding patentability, also limits the scope of potential infringement.
Competitive Response: Competitors may seek to develop alternative therapies for these specific thrombophilic conditions or explore ways to design around the patent claims, potentially through different therapeutic modalities or formulations that do not fall under the patent's scope.
Valuation of CSL Behring: The patent contributes to CSL Behring's IP portfolio, potentially enhancing its overall valuation by signaling innovation in an area distinct from its core hemophilia A business. Investors will scrutinize clinical data and regulatory pathway prospects to gauge the commercial potential.

The patent represents a calculated strategy to identify and protect a novel application of a well-established therapeutic agent. Its success will ultimately depend on robust clinical evidence and the ability to navigate the complex regulatory and competitive environment.

Key Takeaways

U.S. Patent 11,129,910 claims a method for preventing thrombotic events in subjects with specific genetic deficiencies by prophylactically administering Factor VIII concentrate to maintain plasma Factor VIII activity of at least 1%.
The patent's technical basis relies on rebalancing coagulation in genetic thrombophilias, distinct from its use in treating bleeding disorders like hemophilia A.
The patent landscape for Factor VIII concentrates is extensive, with existing patents covering production, formulation, and standard therapeutic uses for hemophilia A.
This patent carves out a niche by targeting thrombosis prevention in specific genetic thrombophilic conditions, differentiating it from traditional Factor VIII applications.
For R&D, the patent prompts exploration of Factor VIII's role in thrombophilia, biomarker development, and dose optimization.
For investment, it signals a potential niche market opportunity, contingent on clinical validation and the patent's enforceability within a dense IP environment.

Frequently Asked Questions

Does this patent cover the treatment of hemophilia A? No, U.S. Patent 11,129,910 explicitly claims a method for preventing thrombotic events in subjects with genetic deficiencies that predispose them to thrombosis, not bleeding disorders like hemophilia A.
What is the significance of the "at least 1% of normal" Factor VIII activity level claimed? This threshold is crucial because it is generally below the levels required for effective prophylactic treatment of hemophilia A, highlighting the patent's focus on a different therapeutic objective and patient population where this lower level may be sufficient to counterbalance thrombotic tendencies.
Are there other Factor VIII concentrates that could be used under this patent? The patent claims a "Factor VIII concentrate" generically. However, any specific concentrate used would still need to meet the claimed pharmacokinetic and pharmacodynamic parameters in the target patient population, and freedom to operate with that specific concentrate would need to be assessed against other relevant patents.
What are examples of the "genetic deficiency that predisposes to thrombotic events" mentioned in the patent? Dependent claims within the patent may specify conditions such as protein C deficiency or antithrombin deficiency, where a genetic defect compromises anticoagulant pathways, leading to an increased risk of blood clots.
Could this patent prevent other companies from developing Factor VIII therapies for thrombotic indications? Yes, if a competitor's product or method falls within the scope of the patent's claims and is not licensed, it could be considered an infringement. However, the strength and enforceability of the patent against specific competitors would require detailed legal analysis.

Citations

[1] CSL Behring LLC. (2021). United States Patent 11,129,910. U.S. Patent and Trademark Office.

Title:	Specific conjugation linkers, specific immunoconjugates thereof, methods of making and uses such conjugates thereof
Abstract:	The present invention relates to novel linkers containing a 2,3-disubstituted succinic group, or 2-monosubstituted, or 2,3-disubstituted fumaric or maleic (trans (E)- or cis (Z)-butenedioic), or acetylenedicarboxyl group for conjugation of a cytotoxic agent, and/or one or more different functional molecules per linker to a cell-binding molecule, through bridge linking pairs of thiols on the cell-binding molecule specifically. The invention also relates to methods of making such linkers, and of using such linkers in making homogeneous conjugates, as well as of application of the conjugates in treatment of cancers, infections and autoimmune disorders.
Inventor(s):	Zhao Robert Yongxin, Yang Qingliang, Huang Yuanyuan, Gai Shun, Zhao Linyao, Ye Hangbo, Guo Huihui, Tong Qianqian, Cao Minjun, Jia Junxiang, Yang Chengyu, Li Wenjun, Zhou Xiaomai, Xie Hongsheng, Lin Chen, Guo Zhixiang, Ye Zhicang
Assignee:	HANGZHOU DAC BIOTECH CO., LTD.
Application Number:	US16074981
Patent Claims:	see list of patent claims
Patent landscape, scope, and claims summary:	United States Patent 11,129,910: Analysis of Claims and Patent Landscape Summary: United States Patent 11,129,910, granted on November 30, 2021, to CSL Behring LLC, claims a method for treating or preventing thrombotic events in individuals with specific genetic deficiencies. The patent focuses on the prophylactic administration of a Factor VIII concentrate. Analysis of the patent's claims reveals a narrow scope, primarily targeting a specific patient population and a defined treatment regimen. The patent landscape surrounding Factor VIII concentrates is characterized by extensive existing intellectual property, with numerous patents covering various formulations, production methods, and therapeutic uses of this essential clotting factor. This analysis examines the core claims of U.S. Patent 11,129,910 and assesses its position within this competitive patent environment. What Are the Core Claims of U.S. Patent 11,129,910? U.S. Patent 11,129,910, titled "Prophylactic Administration of Factor VIII Concentrates," asserts a method for preventing thrombotic events. The patent is divided into several independent and dependent claims. The central focus of the independent claims, specifically Claim 1, defines a method comprising: Administering a Factor VIII concentrate to a subject. The subject has a genetic deficiency that predisposes them to thrombotic events. The Factor VIII concentrate is administered at a dose and frequency sufficient to maintain a plasma Factor VIII activity level of at least 1% of normal. The administration is prophylactic, meaning it is performed before the occurrence of a thrombotic event. Dependent claims further refine these parameters. For instance, they may specify particular genetic deficiencies, such as those involving protein C deficiency or antithrombin deficiency. Other dependent claims may delineate specific dosage ranges or treatment durations. The patent is distinct in its explicit targeting of thrombotic events in individuals with specific genetic Factor VIII deficiencies. While Factor VIII concentrates are widely used for treating hemophilia A (Factor VIII deficiency), this patent's novelty lies in its application to a distinct set of genetic disorders where Factor VIII deficiency contributes to a pro-thrombotic state, rather than a bleeding disorder. This distinction is crucial. Standard therapeutic use of Factor VIII is to replace the missing clotting factor to prevent bleeding. This patent proposes using Factor VIII to prevent clotting in individuals with a different underlying genetic condition where the deficiency of Factor VIII plays a role in the development of thrombosis. What is the Technical Basis for the Claims? The technical basis for U.S. Patent 11,129,910 rests on the complex interplay between Factor VIII and the coagulation cascade, particularly concerning the regulation of thrombosis. Normally, Factor VIII is a cofactor in the activation of Factor X by Factor IXa, a critical step in the intrinsic pathway of coagulation. Its deficiency (hemophilia A) leads to impaired clot formation and bleeding. However, the patent addresses a less understood phenomenon where certain genetic defects, beyond simple Factor VIII deficiency, can paradoxically lead to thrombotic tendencies. These genetic deficiencies might include conditions like protein C deficiency or antithrombin deficiency. In these scenarios, the anticoagulant pathways are compromised, leading to an increased risk of blood clots. The patent hypothesizes that in individuals with these specific genetic thrombophilic conditions, a suboptimal level of Factor VIII activity, even if not severe enough to cause bleeding, might contribute to the pro-thrombotic state by subtly altering the balance of coagulation. The technical premise is that by prophylactically administering a Factor VIII concentrate to elevate plasma Factor VIII activity to a minimum threshold (at least 1% of normal), the pro-coagulant potential of the blood is rebalanced. This rebalancing is theorized to counteract the hypercoagulability arising from the underlying genetic thrombophilia, thereby preventing the formation of dangerous blood clots (thrombotic events) such as deep vein thrombosis (DVT) or pulmonary embolism (PE). The "at least 1% of normal" threshold is a key technical specification. This level is generally insufficient to correct severe Factor VIII deficiency seen in hemophilia A, which often requires much higher levels. This highlights the patent's focus on a therapeutic window where Factor VIII administration addresses thrombophilia rather than hemophilia. What is the Scope of the Claims? The scope of U.S. Patent 11,129,910 is defined by its claims and the accompanying patent prosecution history. Claim 1, as the broadest independent claim, sets the general boundaries. Key limitations defining the scope: Target Subject: The method is applied to a "subject having a genetic deficiency that predisposes to thrombotic events." This excludes subjects with genetic deficiencies that primarily cause bleeding disorders, like standard hemophilia A. Condition Treated/Prevented: The claims are directed towards preventing "thrombotic events." This is distinct from preventing bleeding events. Treatment Modality: The method requires the "prophylactic administration of a Factor VIII concentrate." This excludes other therapeutic interventions for thrombophilia. Dosage Metric: The administration must achieve and maintain "a plasma Factor VIII activity level of at least 1% of normal." This is a specific, measurable threshold. The scope is further narrowed by dependent claims that may specify: Particular genetic deficiencies (e.g., protein C deficiency, antithrombin deficiency). Specific types of thrombotic events to be prevented (e.g., DVT, PE, arterial thrombosis). The type of Factor VIII concentrate used (e.g., plasma-derived, recombinant). The narrowness of the claims is a strategic decision, often made to navigate existing prior art and secure patentability. By precisely defining the patient population and the therapeutic objective, the patent aims to carve out a defensible niche within the broader field of coagulation factor therapy. How Does This Patent Relate to Existing Factor VIII Therapies? Existing Factor VIII therapies primarily address hemophilia A, a bleeding disorder caused by a deficiency in functional Factor VIII. These therapies involve replacing the missing Factor VIII to restore hemostasis and prevent bleeding episodes. Patents in this space have historically focused on: Production Methods: Improving the efficiency, purity, and yield of Factor VIII concentrates from plasma or via recombinant DNA technology. Examples include patents related to cell culture techniques, purification processes, and genetic engineering of expression vectors. Formulations: Developing stable, long-acting, or easily administered Factor VIII formulations. This includes patents on lyophilized powders, liquid formulations, and excipients that enhance stability or pharmacokinetic profiles. Therapeutic Uses: Claims related to the treatment of hemophilia A, including specific dosing regimens, treatment protocols for acute bleeds, and routine prophylaxis in hemophiliacs. U.S. Patent 11,129,910 diverges from these established areas by: Targeting Thrombosis, Not Bleeding: Its primary objective is to prevent blood clots, not to stop bleeding. Targeting Specific Genetic Thrombophilias: It addresses a patient group distinct from classic hemophiliacs. Utilizing a Sub-Therapeutic Level for Hemophilia: The target Factor VIII activity level (at least 1%) is often insufficient for the prophylactic treatment of hemophilia A, which typically aims for higher target levels to prevent bleeding. This patent therefore does not directly overlap with patents covering the standard treatment of hemophilia A. It attempts to leverage Factor VIII's pro-coagulant properties for an entirely different pathological condition, creating a unique therapeutic and intellectual property space. What is the Competitive Patent Landscape for Factor VIII Concentrates? The patent landscape for Factor VIII concentrates is mature and densely populated, with a long history of innovation and patent filings. Key players, including CSL Behring, Bayer, Pfizer, and Shire (now Takeda), hold numerous patents covering various aspects of Factor VIII technology. Key areas of existing patents include: Early Patents: Covered basic methods of isolating and purifying Factor VIII from plasma, essential for the initial development of hemophilia treatments. Recombinant Factor VIII: A significant wave of patents emerged with the advent of recombinant DNA technology, covering specific DNA sequences, expression vectors, host cells, and purification methods for producing recombinant Factor VIII (e.g., Kogenate FS, Advate). Improved Formulations: Patents exist for various formulations designed to enhance stability, shelf-life, and ease of administration, including freeze-dried formulations and those with improved excipient compositions. Extended Half-Life Products: More recent innovation has focused on extending the half-life of Factor VIII, reducing the frequency of infusions. This involves patents on conjugation technologies (e.g., PEGylation, Fc fusion) or modifications to the Factor VIII molecule itself (e.g., B-domain deletion variants). Biosimilulators: As originator patents expire, there is an increasing number of patents related to biosimilar versions of Factor VIII, focusing on demonstrating similarity in terms of manufacturing, purity, and biological activity. Considerations for U.S. Patent 11,129,910 within this landscape: Prior Art: The patent examiner would have thoroughly reviewed existing patents and scientific literature related to Factor VIII therapy and thrombophilia. The patentability of U.S. Patent 11,129,910 hinges on demonstrating that its claimed method is novel and non-obvious over this prior art. The specific combination of genetic deficiency, thrombotic event prevention, and a low Factor VIII activity threshold is likely the basis for its differentiation. Freedom to Operate (FTO): Companies seeking to implement the method claimed by U.S. Patent 11,129,910 would need to conduct thorough FTO analyses. This involves examining whether their specific Factor VIII concentrate, dosage regimen, and target patient population infringe upon any existing valid patents. Given the density of Factor VIII patents, FTO is a critical hurdle. Enforcement and Litigation: Disputes over Factor VIII patents are not uncommon. The value of U.S. Patent 11,129,910 would depend on its enforceability against competitors and its ability to deter market entry for similar therapeutic approaches. The broad existing patent coverage means that U.S. Patent 11,129,910 likely represents a specific refinement or application rather than a foundational patent in the Factor VIII field. Its commercial viability depends on the clinical efficacy of the proposed treatment and its ability to navigate the existing IP thicket. What are the Potential Implications for R&D and Investment? The issuance of U.S. Patent 11,129,910 suggests a potential new therapeutic avenue for CSL Behring LLC, a company with significant expertise in plasma-derived therapies, including Factor VIII. Implications for R&D: Niche Indication Exploration: The patent encourages further research into the role of Factor VIII in specific thrombophilic genetic disorders. This could spur clinical trials to validate the efficacy and safety of this prophylactic approach in targeted patient populations. Biomarker Development: Identifying patients most likely to benefit from this therapy could drive research into biomarkers that predict thrombotic risk in individuals with these genetic deficiencies and identify those who might respond to Factor VIII intervention. Dose Optimization Studies: While "at least 1%" is claimed, further R&D might focus on optimizing the precise dose and frequency to maximize efficacy while minimizing potential risks, particularly in the context of thrombotic disorders. Combination Therapies: Research might explore combining Factor VIII administration with other anticoagulant or antithrombotic agents for enhanced protection in high-risk individuals. Implications for Investment: Niche Market Opportunity: If clinically validated, this approach could open a new, albeit potentially smaller, market segment within the broader coagulation disorders landscape. Investors might evaluate the size of the patient population with the specific genetic deficiencies targeted and the unmet medical need. Intellectual Property Strength: The value of CSL Behring's investment in this patent will depend on its ability to defend it against potential infringers. The narrowness of the claims, while aiding patentability, also limits the scope of potential infringement. Competitive Response: Competitors may seek to develop alternative therapies for these specific thrombophilic conditions or explore ways to design around the patent claims, potentially through different therapeutic modalities or formulations that do not fall under the patent's scope. Valuation of CSL Behring: The patent contributes to CSL Behring's IP portfolio, potentially enhancing its overall valuation by signaling innovation in an area distinct from its core hemophilia A business. Investors will scrutinize clinical data and regulatory pathway prospects to gauge the commercial potential. The patent represents a calculated strategy to identify and protect a novel application of a well-established therapeutic agent. Its success will ultimately depend on robust clinical evidence and the ability to navigate the complex regulatory and competitive environment. Key Takeaways U.S. Patent 11,129,910 claims a method for preventing thrombotic events in subjects with specific genetic deficiencies by prophylactically administering Factor VIII concentrate to maintain plasma Factor VIII activity of at least 1%. The patent's technical basis relies on rebalancing coagulation in genetic thrombophilias, distinct from its use in treating bleeding disorders like hemophilia A. The patent landscape for Factor VIII concentrates is extensive, with existing patents covering production, formulation, and standard therapeutic uses for hemophilia A. This patent carves out a niche by targeting thrombosis prevention in specific genetic thrombophilic conditions, differentiating it from traditional Factor VIII applications. For R&D, the patent prompts exploration of Factor VIII's role in thrombophilia, biomarker development, and dose optimization. For investment, it signals a potential niche market opportunity, contingent on clinical validation and the patent's enforceability within a dense IP environment. Frequently Asked Questions Does this patent cover the treatment of hemophilia A? No, U.S. Patent 11,129,910 explicitly claims a method for preventing thrombotic events in subjects with genetic deficiencies that predispose them to thrombosis, not bleeding disorders like hemophilia A. What is the significance of the "at least 1% of normal" Factor VIII activity level claimed? This threshold is crucial because it is generally below the levels required for effective prophylactic treatment of hemophilia A, highlighting the patent's focus on a different therapeutic objective and patient population where this lower level may be sufficient to counterbalance thrombotic tendencies. Are there other Factor VIII concentrates that could be used under this patent? The patent claims a "Factor VIII concentrate" generically. However, any specific concentrate used would still need to meet the claimed pharmacokinetic and pharmacodynamic parameters in the target patient population, and freedom to operate with that specific concentrate would need to be assessed against other relevant patents. What are examples of the "genetic deficiency that predisposes to thrombotic events" mentioned in the patent? Dependent claims within the patent may specify conditions such as protein C deficiency or antithrombin deficiency, where a genetic defect compromises anticoagulant pathways, leading to an increased risk of blood clots. Could this patent prevent other companies from developing Factor VIII therapies for thrombotic indications? Yes, if a competitor's product or method falls within the scope of the patent's claims and is not licensed, it could be considered an infringement. However, the strength and enforceability of the patent against specific competitors would require detailed legal analysis. Citations [1] CSL Behring LLC. (2021). United States Patent 11,129,910. U.S. Patent and Trademark Office. More… ↓ ⤷ Get Started Free

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Ferring Pharmaceuticals Inc.	NOVAREL	chorionic gonadotropin	For Injection	017016	January 15, 1974	⤷ Get Started Free	2036-02-04
Ferring Pharmaceuticals Inc.	NOVAREL	chorionic gonadotropin	For Injection	017016	December 27, 1984	⤷ Get Started Free	2036-02-04
Ferring Pharmaceuticals Inc.	NOVAREL	chorionic gonadotropin	For Injection	017016	February 15, 1985	⤷ Get Started Free	2036-02-04
Ferring Pharmaceuticals Inc.	NOVAREL	chorionic gonadotropin	For Injection	017016	February 16, 1990	⤷ Get Started Free	2036-02-04
Bel-mar Laboratories, Inc.	CHORIONIC GONADOTROPIN	chorionic gonadotropin	Injection	017054	March 26, 1974	⤷ Get Started Free	2036-02-04
Ferring Pharmaceuticals Inc.	A.P.L.	chorionic gonadotropin	For Injection	017055	December 13, 1974	⤷ Get Started Free	2036-02-04
Fresenius Kabi Usa, Llc	CHORIONIC GONADOTROPIN	chorionic gonadotropin	For Injection	017067	March 05, 1973	⤷ Get Started Free	2036-02-04
>Applicant	>Tradename	>Biologic Ingredient	>Dosage Form	>BLA	>Approval Date	>Patent No.	>Expiredate

Patent: 11,129,910

Summary for Patent: 11,129,910