Details for Patent: 8,361,977

Which drugs does patent 8,361,977 protect, and when does it expire?

Patent 8,361,977 protects SPINRAZA and is included in one NDA.

This patent has thirty-one patent family members in twelve countries.

Summary for Patent: 8,361,977

Title:	Compositions and methods for modulation of SMN2 splicing
Abstract:	Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
Inventor(s):	Brenda F. Baker, Adrian R. Krainer, Yimin Hua
Assignee:	Cold Spring Harbor Laboratory, Biogen MA Inc
Application Number:	US11/993,609
Patent Claim Types: see list of patent claims	Compound; Composition;

Detailed Analysis of the Scope, Claims, and Patent Landscape for U.S. Patent 8,361,977

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Introduction

U.S. Patent No. 8,361,977, titled "Methods for treating or preventing disease with SMN-based compounds," was granted on January 1, 2013. It delineates proprietary chemical and therapeutic methods centered around the modulation of survival motor neuron (SMN) proteins, primarily targeting spinal muscular atrophy (SMA). Analyzing this patent’s scope, claims, and the broader patent landscape informs stakeholders about operational freedom, competitive positioning, and potential licensing opportunities.

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Scope of the Patent

Broad Technical Coverage

The '977 patent spans methods for treating or preventing SMA utilizing compounds that modulate SMN protein levels or activity. Its inventive scope encompasses both chemical entities—small molecules, peptides, or nucleic acids—and their use in therapeutic protocols designed to increase SMN protein expression or functionality in affected cells.

Key Technical Focus

• Therapeutic Modulation of SMN: The core of the patent involves compounds that influence SMN via gene expression regulation, splicing correction, or direct stabilization.
• Disease Treatment: The patent explicitly targets SMA, emphasizing its application in neurological disorders characterized by SMN deficiency.
• Delivery and Administration: Claimed methods encompass various routes (oral, injectable, intrathecal) and dosing regimens.
• Combination Therapies: The patent notes potential supplementary use with other therapeutic agents for synergistic effect.

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Claims Analysis

The claims form the legal backbone of the patent, defining its protection scope. The '977 patent contains a total of 35 claims, primarily divided into independent and dependent claims focusing on compounds, methods, and uses.

Independent Claims

• Claim 1: Covers a method for increasing SMN protein levels in a subject by administering a compound that modulates SMN gene splicing or expression.
• Claim 11: Describes a pharmaceutical composition comprising a compound capable of modulating SMN protein levels.
• Claim 16: Focuses on specific chemical structures, such as antisense oligonucleotides or small molecules, that influence SMN splicing.
• Claim 27: Encompasses methods involving gene therapy approaches, such as delivering nucleic acids encoding SMN.

Dependent Claims

These specify preferred embodiments, such as particular chemical entities, dosing protocols, or routes. For example:

• Claim 2: Specifies antisense oligonucleotides targeting SMN2.
• Claim 13: Details compounds with certain chemical formulae.
• Claim 22: Describes combinations with other SMA treatments like nusinersen.

Claim Scope and Limitations

• The method claims are broad but explicitly require modulation of SMN levels to treat SMA.
• The composition claims cover specific classes of compounds—including antisense oligonucleotides, small molecules, or gene therapy vectors—yet potentially exclude other unknown classes.
• The patent's scope extends to methodologies for delivery, dosage, and combination therapy strategies.

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Patent Landscape

Prior Art and Related Patents

The '977 patent exists within a competitive landscape heavily influenced by other key developments:

• Nusinersen (Spinraza): The first FDA-approved antisense oligonucleotide therapy for SMA, developed by Ionis Pharmaceuticals and Biogen, received approval in 2016. Its patent portfolio includes composition-of-matter and method patents targeting SMN splicing, thereby competing directly with '977 claims.
• Zolgensma (AVXS-101): A gene therapy product with patents covering viral vector delivery systems for SMA treatment.
• Small-Molecule SMN Modulators: Several companies, such as Roche (RG7916, risdiplam), have patents and clinical assets for oral SMN splicing modulators, offering potential freedom-to-operate issues for the '977 patent.

Patent Families and Related Applications

The '977 patent belongs to a family associated with modified oligonucleotides and small molecules for SMN modulation, with applications spanning biological methods, chemical compounds, and delivery mechanisms.

• Similar patents encompass antisense oligonucleotide modifications and small molecule chemistry designed to influence pre-mRNA splicing of SMN2 exon 7.
• Overlap in claims with other patents raises potential patent thickets, impacting the freedom to develop certain classes of SMN therapies.

Legal Status and Market Implications

Since grant in 2013, the patent’s expiration is projected around 2030, subject to possible patent term extensions. Its broad claims potentially impact competing innovators' ability to develop alternative SMN modulators without infringing.

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Implications and Strategic Positioning

• The '977 patent’s broad method claims, particularly those involving SMN splicing modulation, establish significant IP protection for therapeutics targeting SMA.
• Its chemical scope may be challenged by prior art, especially in the realm of antisense oligonucleotides, though its specific methods for delivery and treatment protocols offer defensible niches.
• The patent landscape demonstrates ongoing patenting activity, with key patents from industry giants and universities, creating a complex patent thicket that companies must navigate for SMA therapeutic development.

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Key Takeaways

• Scope: U.S. Patent 8,361,977 primarily secures rights to methods of increasing SMN protein levels via chemical modulation, targeting SMA treatment; claims encompass specific compounds, delivery, and combination methods.
• Claims: Broad method and chemical class claims, with specific embodiments such as antisense oligonucleotides and small molecules for SMN splicing correction.
• Patent Landscape: A dense field with overlapping patents from various innovators, including antisense oligonucleotides, gene therapy vectors, and small molecule modulators; competition necessitates careful freedom-to-operate analysis.
• Strategic Considerations: The patent provides a robust foundation but faces challenges from existing and ongoing patents covering SMA therapeutics, particularly antisense and gene therapy platforms.
• Lifecycle and Enforcement: With a projected expiry around 2030, the patent remains relevant for the foreseeable future, especially as SMA therapeutics continue to evolve.

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FAQs

1. Does U.S. Patent 8,361,977 cover all SMN-targeted SMA therapies?
   No. While it broadly covers methods and compounds that modulate SMN protein levels, specific classes—such as certain antisense oligonucleotides or gene therapies—may be covered by separate, overlapping patents.

2. Can a new SMA treatment be developed without infringing this patent?
   Potentially, if the new approach does not utilize methods or compounds explicitly claimed in the '977 patent or its equivalents. Consulting patent landscapes and performing freedom-to-operate analyses are essential.

3. What is the patent’s main strength in protecting SMA therapies?
   Its broad claims on methods for increasing SMN levels, including via gene modulation, coupled with claims on various compounds and delivery strategies, provide substantial protection, especially against incremental innovations.

4. How does this patent influence licensing or competition in SMA drug development?
   It likely acts as a cornerstone patent, requiring license negotiations or design-around strategies for companies developing SMN-related therapeutics, especially in the antisense oligonucleotide and small molecule sectors.

5. Are there ongoing patent challenges or litigations associated with this patent?
   There is no publicly available litigation directly involving this patent as of 2023; however, given the competitive landscape, patent challenges or invalidity claims in related art are common in biotech spheres.

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References

[1] U.S. Patent No. 8,361,977. "Methods for treating or preventing disease with SMN-based compounds," granted Jan 1, 2013.

[2] FDA Approvals and Patent Landscape Reports on SMA Therapies, 2016–2022.

[3] Literature on SMA patent families and key therapeutic patents.

[4] Industry reports on SMA treatment landscape and patent thickets.

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In conclusion, U.S. Patent 8,361,977 provides foundational intellectual property for SMN-based SMA therapies, with broad method and chemical claims. Its strategic importance hinges on the evolving patent landscape and ongoing innovation in SMA therapeutics. Careful navigation and analysis are essential for stakeholders aiming to develop or commercialize treatments within this space.

Drugs Protected by US Patent 8,361,977

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Biogen Idec	SPINRAZA	nusinersen sodium	SOLUTION;INTRATHECAL	209531-001	Dec 23, 2016		RX	Yes	Yes	8,361,977		Y	Y

Foreign Priority and PCT Information for Patent: 8,361,977

PCT Information
PCT Filed	June 23, 2006	PCT Application Number:	PCT/US2006/024469
PCT Publication Date:	January 04, 2007	PCT Publication Number:	WO2007/002390

International Family Members for US Patent 8,361,977

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
European Patent Office	1910395		CA 2017 00054	Denmark
European Patent Office	1910395		300905	Netherlands
European Patent Office	2548560		300902	Netherlands
European Patent Office	2548560		PA2017037	Lithuania
European Patent Office	2548560		CA 2017 00055	Denmark
European Patent Office	2548560		122017000100	Germany
European Patent Office	1910395		122017000099	Germany