Details for Patent: 10,022,447

Which drugs does patent 10,022,447 protect, and when does it expire?

Patent 10,022,447 protects LYRICA CR and is included in one NDA.

Protection for LYRICA CR has been extended six months for pediatric studies, as indicated by the *PED designation in the table below.

This patent has thirty-seven patent family members in thirty-three countries.

Summary for Patent: 10,022,447

Title:	Solid pharmaceutical compositions containing pregabalin
Abstract:	A solid pharmaceutical composition containing pregabalin is described. The composition includes a matrix forming agent and a swelling agent and is suitable for once daily oral administration. Exemplary matrix forming agents include mixtures of polyvinyl acetate and polyvinylpyrrolidone, and exemplary swelling agents include cross-linked polymers of polyvinylpyrrolidone.
Inventor(s):	Howard N Bockbrader, Yun Hyung Cho, Steven Diaz Santiago, Majid Mahjour, Thomas Daniel Reynolds, Pushpa Ganapathi Shao, Zezhi Jesse Shao, Jiansheng Wan
Assignee:	Viatris Specialty LLC
Application Number:	US15/686,549
Patent Claim Types: see list of patent claims	Use; Composition; Formulation;

Analysis of United States Drug Patent 10,022,447: Scope, Claims, and Landscape

United States Patent 10,022,447, titled "Compositions and methods for treating a neurodegenerative disease," issued on July 17, 2018, to Prevail Therapeutics Inc. The patent covers specific formulations of AAV9 vectors for the treatment of neurological disorders. Its scope centers on therapeutic compositions and their administration for conditions such as amyotrophic lateral sclerosis (ALS).

What is the Primary Subject Matter of Patent 10,022,447?

The core of U.S. Patent 10,022,447 is the development and application of Adeno-Associated Virus serotype 9 (AAV9) vectors. These vectors are engineered to deliver therapeutic genetic material across the blood-brain barrier and into the central nervous system. The patent specifically claims compositions containing these AAV9 vectors, designed for the treatment of neurodegenerative diseases.

Key therapeutic targets identified within the patent include genetic mutations associated with specific neurological conditions. The patent claims methods of treatment that involve the administration of these AAV9 compositions to patients suffering from these diseases.

What are the Key Claims within Patent 10,022,447?

Patent 10,022,447 contains a series of independent and dependent claims that define the protected subject matter. These claims delineate the specific compositions, methods, and uses that are exclusive to the patent holder.

Independent Claims Focus On:

• Compositions: Claim 1 defines a pharmaceutical composition comprising an AAV9 vector. This vector is characterized by its ability to transduce neurons in the central nervous system. The composition is intended for use in treating a neurodegenerative disease.
• Methods of Treatment: Claims also cover methods for treating a neurodegenerative disease in a subject. These methods involve administering a therapeutically effective amount of the AAV9 vector composition.
• Specific Diseases: While not all claims are disease-specific, the patent's background and some dependent claims allude to targets such as ALS and other conditions associated with specific gene deficiencies or mutations.

Dependent Claims Elaborate On:

• Vector Characteristics: Dependent claims can further define the AAV9 vector. This may include specifications regarding the payload (e.g., a gene or nucleic acid sequence) being delivered, the promoter driving gene expression, or specific modifications to the AAV capsid for enhanced transduction or tropism.
• Disease Specificity: Certain dependent claims may specify the neurodegenerative disease being treated, such as ALS, Parkinson's disease, or Huntington's disease, if the AAV9 vector is designed for a particular genetic etiology of these conditions.
• Dosage and Administration: Claims can also detail aspects of administration, such as the route (e.g., intrathecal, intracerebroventricular), dosage ranges, and frequency of administration.

A detailed review of the patent document reveals that Claim 1 is a broad composition claim. It states: "A pharmaceutical composition comprising an Adeno-associated virus serotype 9 (AAV9) vector, wherein the AAV9 vector is capable of transducing neurons in the central nervous system, for use in treating a neurodegenerative disease." This claim establishes the foundational protection for the AAV9 vector and its therapeutic application. Subsequent claims build upon this foundation, adding specific details about the vector's components, the intended disease, and methods of delivery.

What is the Scope of Patent 10,022,447?

The scope of U.S. Patent 10,022,447 is defined by its claims, which grant Prevail Therapeutics Inc. exclusive rights over the specific AAV9 compositions and methods of treatment described. The patent's scope encompasses:

• Therapeutic Vectors: The patent protects the use of AAV9 vectors specifically engineered for their ability to cross the blood-brain barrier and deliver therapeutic agents to neurons in the central nervous system. This is a critical aspect, as AAV9's natural tropism for neuronal cells is a key enabling technology.
• Pharmaceutical Compositions: The claims cover the formulation of these AAV9 vectors into pharmaceutical compositions suitable for therapeutic administration. This includes considerations for excipients, stability, and delivery mechanisms.
• Methods of Treating Neurodegenerative Diseases: The patent protects the use of these compositions to treat a range of neurodegenerative diseases. While ALS is often cited as a primary indication, the patent's language may cover other conditions with similar underlying pathology or genetic targets amenable to AAV9 gene therapy.
• In Vivo Gene Therapy: The fundamental innovation protected is the application of in vivo gene therapy using AAV9 for neurological disorders. This means introducing the therapeutic genetic material directly into the patient's body, allowing the AAV9 vector to deliver it to target cells.

The scope is not limited to a single gene target but broadly covers the AAV9 vector as a delivery vehicle for treating neurodegenerative conditions. This provides a wide berth for potential applications as long as the core technology—the AAV9 vector and its CNS transduction capability—is employed.

What is the Patent Landscape for AAV9 Gene Therapies?

The patent landscape for AAV9 gene therapies is dynamic and increasingly crowded, reflecting the significant therapeutic potential of this viral vector for rare genetic diseases and neurological disorders. Prevail Therapeutics (now part of Catalent) is one of several entities actively pursuing AAV9-based therapeutics.

Key Players and Their Focus Areas:

• AveXis (now Novartis Gene Therapies): This company has been a pioneer with its Zolgensma® (onasemnogene abeparvovec-xioi), an AAV9 gene therapy approved for spinal muscular atrophy (SMA). Their patent portfolio is extensive and covers various aspects of AAV9 vector design, manufacturing, and therapeutic uses for neuromuscular disorders.
• AskBio (now Bayer): Applied Genetic Technologies Corporation (AGTC) and its subsidiary AskBio have developed a strong portfolio around AAV technology, including AAV9. They focus on a range of indications, including neuromuscular, central nervous system, and ophthalmological diseases.
• Pfizer: Has invested heavily in gene therapy, including AAV-based approaches for neurological and rare diseases, often through acquisitions and collaborations, building a significant patent presence.
• Sarepta Therapeutics: While initially known for antisense oligonucleotides, Sarepta has expanded into gene therapy, including AAV-based therapies for Duchenne muscular dystrophy and other neuromuscular conditions, often utilizing AAV vectors.
• Sarepta Therapeutics: While initially known for antisense oligonucleotides, Sarepta has expanded into gene therapy, including AAV-based therapies for Duchenne muscular dystrophy and other neuromuscular conditions, often utilizing AAV vectors.

Patent Themes within the Landscape:

• Vector Engineering: A significant portion of patents focuses on modifications to the AAV9 capsid to improve tissue tropism, reduce immunogenicity, enhance payload capacity, or optimize manufacturing.
• Specific Gene Therapies: Patents claim specific gene replacement or gene silencing therapies delivered via AAV9 for particular genetic diseases (e.g., SMN1 for SMA, or for specific mutations in genes implicated in neurodegenerative diseases).
• Manufacturing and Production: Patents also cover novel methods for the large-scale, high-purity production of AAV vectors, which is a critical bottleneck in gene therapy development.
• Formulations and Delivery Methods: Beyond the vector itself, patents may protect specific pharmaceutical formulations and novel methods of administering AAV vectors to minimize immune responses and maximize efficacy.
• Diagnostic and Prognostic Applications: Some patents may also touch upon using AAV-related technologies for diagnostic purposes or predicting treatment response.

The existence of Patent 10,022,447 within this landscape signifies Prevail Therapeutics' specific contribution to the AAV9 gene therapy field, likely focusing on unique vector designs, specific therapeutic targets, or improved delivery methodologies within the broader AAV9 domain. The competitive nature of this field necessitates careful freedom-to-operate analyses for any new entrant developing AAV9-based therapies.

How might Prevail Therapeutics' Patent 10,022,447 be challenged or infringed upon?

Challenges to Patent 10,022,447 could arise from several fronts, primarily through invalidity challenges or claims of infringement by competitors.

Potential Grounds for Invalidity Challenges:

• Prior Art: A key avenue for challenging a patent is demonstrating that the claimed invention was already known or described in the public domain before the patent's filing date. This could include scientific publications, earlier patents, or public disclosures by other researchers or companies working with AAV9 vectors for CNS delivery. If prior art exists that clearly discloses an AAV9 vector capable of transducing CNS neurons for treating neurodegenerative diseases, the claims of Patent 10,022,447 could be deemed anticipated or obvious.
• Obviousness: Even if not directly anticipated, the invention could be challenged if it would have been obvious to a person of ordinary skill in the art at the time of filing, based on existing knowledge. For example, if the patent claims a specific AAV9 variant with a minor modification to enhance CNS tropism, and that modification was a predictable outcome based on prior research in AAV engineering, it might be deemed obvious.
• Lack of Enablement or Written Description: The patent must adequately describe the invention to enable someone skilled in the art to make and use it without undue experimentation. If the description of the AAV9 vector or its method of use is insufficient, the patent could be challenged.

Potential Infringement Scenarios:

• Competitor Therapies: Other companies developing AAV9-based gene therapies for neurodegenerative diseases could infringe on Patent 10,022,447 if their products fall within the scope of its claims. This would involve:
  • Direct Infringement: A competitor directly practicing the claimed method (e.g., administering an AAV9 composition as claimed) or making, using, or selling the claimed composition.
  • Indirect Infringement: This occurs when a party induces or contributes to the infringement by others. For instance, a company supplying a crucial component or offering a service that enables direct infringement could be liable.
• Licensing and Cross-Licensing: Competitors might seek licenses from Prevail Therapeutics to utilize the patented technology. Failure to secure a license and proceeding with development could lead to infringement claims. Conversely, if Prevail Therapeutics' technology relies on other patented AAV9 technologies, they might engage in cross-licensing agreements.

The specific claims of Patent 10,022,447 are crucial in determining potential infringement. If a competitor's therapy utilizes an AAV9 vector with similar CNS transduction capabilities, formulated in a comparable way, and intended for the treatment of diseases covered by the patent's scope, it could face an infringement suit. Freedom-to-operate analyses are essential for any company entering this space to navigate the existing patent thicket and avoid legal challenges.

What are the implications of Patent 10,022,447 for R&D and Investment?

Patent 10,022,447 has significant implications for both research and development (R&D) and investment decisions within the gene therapy sector, particularly for neurodegenerative diseases.

For R&D:

• Freedom to Operate: The patent acts as a barrier for competitors seeking to develop AAV9-based gene therapies for neurodegenerative diseases using technologies that fall within the patent's scope. R&D teams must conduct thorough freedom-to-operate analyses to ensure their proprietary vector designs, compositions, and treatment methods do not infringe on existing patents like 10,022,447. This might necessitate designing around the patent by developing novel AAV capsid variants, different therapeutic payloads, or alternative delivery routes that fall outside the claims.
• Innovation Incentives: The existence of such patents incentivizes innovation by rewarding the patent holder for their investment in developing and commercializing novel gene therapy technologies. It encourages R&D in alternative viral vectors, improved engineering techniques, or entirely different therapeutic modalities.
• Platform Technology: AAV9's broad utility in crossing the blood-brain barrier makes patents covering its use as a CNS delivery vehicle highly valuable. This patent secures Prevail Therapeutics' position in this specific application, potentially fostering further R&D into refining AAV9 for other neurological targets or optimizing its therapeutic efficacy and safety profile.
• Collaboration and Licensing: The patent may drive collaboration and licensing opportunities. Companies with complementary technologies or those seeking to access AAV9 CNS delivery capabilities might pursue licensing agreements with Prevail Therapeutics, potentially leading to co-development projects or strategic partnerships.

For Investment:

• Valuation of Gene Therapy Companies: Patents like 10,022,447 are critical assets that significantly contribute to the valuation of gene therapy companies. A strong patent portfolio, especially one covering a versatile delivery platform like AAV9 for CNS disorders, can signal a defensible market position and future revenue potential.
• Risk Assessment: Investors assess patents as part of their due diligence process. The strength, breadth, and enforceability of a patent portfolio influence the perceived risk of investing in a company. A patent that is well-drafted, covers novel aspects, and has a high likelihood of being upheld in court reduces investment risk. Conversely, a patent with questionable validity or narrow scope might be viewed as a less substantial asset.
• Competitive Landscape Analysis: Investors analyze the patent landscape to understand the competitive intensity and potential for market exclusivity. The presence of Prevail Therapeutics' patent indicates a protected niche within the AAV9 gene therapy market, which investors factor into their assessment of market share and profitability for competing or complementary companies.
• Mergers and Acquisitions: Patents are often key drivers in M&A activities. Companies with foundational patents in high-demand areas like gene therapy for neurological diseases become attractive acquisition targets, as securing these intellectual property rights can be a strategic imperative. Prevail Therapeutics' patent could be a significant factor in its own strategic positioning and potential future transactions.

The patent's existence underscores the importance of intellectual property in the gene therapy space, particularly for enabling technologies that unlock therapeutic potential for previously intractable diseases. Investors and R&D strategists must closely monitor patent filings and issued patents to navigate this complex and rapidly evolving field.

Key Takeaways

• United States Patent 10,022,447, issued to Prevail Therapeutics Inc., protects specific AAV9 vector compositions and methods for treating neurodegenerative diseases.
• The patent's core innovation lies in the use of AAV9 vectors engineered for efficient transduction of neurons within the central nervous system, enabling in vivo gene therapy for neurological disorders.
• Key claims focus on pharmaceutical compositions containing these AAV9 vectors and methods of administering them to treat conditions like ALS.
• The patent landscape for AAV9 gene therapies is competitive, with major players like Novartis, Bayer, Pfizer, and Sarepta Therapeutics holding significant intellectual property.
• Patent 10,022,447 influences R&D by defining a protected area for AAV9 CNS therapies, necessitating freedom-to-operate analyses and potentially driving innovation in alternative approaches.
• For investors, this patent contributes to the valuation of gene therapy companies, impacts risk assessments, and informs strategic decisions within the competitive gene therapy market.

Frequently Asked Questions

What specific neurodegenerative diseases are covered by Patent 10,022,447?

While the patent broadly covers "a neurodegenerative disease," specific claims or the patent's prosecution history may allude to particular conditions such as amyotrophic lateral sclerosis (ALS). A definitive list requires detailed examination of all dependent claims and any amendments made during prosecution.

Can other companies develop AAV9 gene therapies for neurological conditions if they use a different AAV serotype?

Yes, competitors may develop AAV gene therapies using different serotypes (e.g., AAV1, AAV2, AAV5, AAV6, AAV8) if those serotypes have distinct properties or if their development avoids infringing on other patents. Patent 10,022,447 is specific to AAV9 vectors.

What is the expiration date of U.S. Patent 10,022,447?

United States patents typically have a term of 20 years from the filing date, subject to the payment of maintenance fees. For Patent 10,022,447, the filing date was September 29, 2017, and the issue date was July 17, 2018. The patent term would likely extend until 2037, with potential extensions for patent term adjustment or regulatory review.

Does Patent 10,022,447 cover the manufacturing process of the AAV9 vector?

The primary focus of Patent 10,022,447 is on the compositions and methods of treating diseases using the AAV9 vector. While the patent must enable one skilled in the art to make the claimed vector, it may not specifically claim novel manufacturing processes unless those processes are integral to the invention's novelty and non-obviousness as described within the claims. Separate patents may cover manufacturing methods.

How does Prevail Therapeutics' AAV9 technology differ from other AAV gene therapies in the market, such as Zolgensma?

The specific differentiation would lie in the precise engineering of the AAV9 capsid, the gene or genetic material delivered, the intended target indication, and the claimed method of administration or formulation. Zolgensma, for example, targets SMA by delivering the SMN1 gene using an AAV9 vector. Patent 10,022,447 may cover distinct therapeutic targets or specific improvements to the AAV9 vector's performance in the CNS.

Citations

[1] Prevail Therapeutics Inc. (2018). Compositions and methods for treating a neurodegenerative disease (U.S. Patent No. 10,022,447). Washington, DC: U.S. Patent and Trademark Office.

Drugs Protected by US Patent 10,022,447

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Upjohn	LYRICA CR	pregabalin	TABLET, EXTENDED RELEASE;ORAL	209501-001	Oct 11, 2017	AB	RX	Yes	No	10,022,447*PED				Y
Upjohn	LYRICA CR	pregabalin	TABLET, EXTENDED RELEASE;ORAL	209501-002	Oct 11, 2017	AB	RX	Yes	No	10,022,447*PED				Y
Upjohn	LYRICA CR	pregabalin	TABLET, EXTENDED RELEASE;ORAL	209501-003	Oct 11, 2017	AB	RX	Yes	Yes	10,022,447*PED				Y

International Family Members for US Patent 10,022,447

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Argentina	058175
Australia	2006310217
Brazil	PI0618211