Treating Muscular Dystrophy with a Self-Complementary AAV Gene Therapy
This patent describes a method to treat muscular dystrophy by delivering a specific gene using a specially designed, self-complementary adeno-associated virus (AAV) vector.
Original patent title: “Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy”
This patent describes a method to treat muscular dystrophy by delivering a specific gene using a specially designed, self-complementary adeno-associated virus (AAV) vector. Granted in 2026.
Coverage
What does this patent actually cover?
This patent describes methods for treating muscular dystrophy by introducing a specific gene into a patient's cells. It uses a self-complementary recombinant adeno-associated virus (rAAV) vector, specifically named scAAVrh74.tMCK.hSGCA, as a delivery vehicle. This vector is designed to express the alpha-sarcoglycan gene (hSGCA) in the patient's body, which is crucial for muscle function. The patent also covers ways to create this rAAV and pharmaceutical mixtures containing it. For example, a patient with a type of muscular dystrophy caused by a faulty alpha-sarcoglycan gene could receive this vector to help their muscles produce the correct protein.
The gap
What does this patent NOT cover?
- Does not cover gene therapies for muscular dystrophy that use different AAV serotypes (like AAV9 or AAV8) instead of the specified scAAVrh74 vector.
- Does not cover gene therapies for muscular dystrophy that deliver genes other than alpha-sarcoglycan (hSGCA), such as the dystrophin gene for Duchenne muscular dystrophy.
- Does not cover non-viral methods of gene delivery, such as lipid nanoparticles or electroporation, for treating muscular dystrophy.
- Does not cover treatments for muscular dystrophy that do not involve gene therapy, such as physical therapy or steroid medications.
- Does not cover the use of this specific scAAVrh74.tMCK.hSGCA vector for treating diseases other than muscular dystrophy.
These exclusions are unique to PatentBrief — derived from the actual claim language, not patent-office boilerplate.
Key facts
What made this novel
The noveltynoveltyThe requirement that an invention be different from anything publicly known before its priority date.Read more → lies in using a 'self-complementary' AAV vector, which means the viral DNA can quickly form a double-stranded template inside the cell. This speeds up gene expression compared to traditional AAVs, making the therapy potentially more effective and faster-acting for muscle diseases where rapid protein production is critical.
Schematic visualization of the patent's claim structure. Hand-drawn diagrams in progress for each landmark patent.
Where you've seen this
Real-world examples
Gene therapies for Limb-Girdle Muscular Dystrophy (LGMD)
Investigational gene therapies targeting sarcoglycan deficiencies
Sarepta Therapeutics' gene therapy programs for muscular dystrophies
Why it matters
The bigger picture
Muscular dystrophy is a group of genetic diseases that cause progressive weakness and loss of muscle mass, often leading to severe disability. Gene therapy offers a promising approach to correct the underlying genetic defects, potentially slowing or halting disease progression. This patent focuses on a specific gene and delivery method, contributing to the development of targeted treatments for these debilitating conditions.
Filed
November 30, 2022
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Companies like Sarepta Therapeutics, Pfizer, and Solid Biosciences are actively developing gene therapies for various forms of muscular dystrophy, including those targeting specific sarcoglycan deficiencies. The assigneeassigneeThe entity that owns the patent — usually the inventor's employer or a company.Read more → of this patent, though unknown, is operating in a competitive and rapidly evolving field where novel AAV vectors and gene targets are continuously being explored.
Market impact
The development of gene therapies for muscular dystrophy has created a new, high-value market segment within rare disease treatments. These therapies aim to offer long-term or curative solutions, shifting the paradigm from symptom management to addressing the root cause. This has led to significant investment in biotech and pharmaceutical research, fostering intense competition and driving innovation in viral vector design and gene delivery technologies.
Claim 1 — Plain English
What this patent covers
This patent describes methods for treating muscular dystrophy by introducing a specific gene into a patient's cells. It uses a self-complementary recombinant adeno-associated virus (rAAV) vector, specifically named scAAVrh74.tMCK.hSGCA, as a delivery vehicle. This vector is designed to express the alpha-sarcoglycan gene (hSGCA) in the patient's body, which is crucial for muscle function. The patent also covers ways to create this rAAV and pharmaceutical mixtures containing it. For example, a patient with a type of muscular dystrophy caused by a faulty alpha-sarcoglycan gene could receive this vector to help their muscles produce the correct protein.
The clever bit
The novelty lies in using a 'self-complementary' AAV vector, which means the viral DNA can quickly form a double-stranded template inside the cell. This speeds up gene expression compared to traditional AAVs, making the therapy potentially more effective and faster-acting for muscle diseases where rapid protein production is critical.
What it does not cover
- Does not cover gene therapies for muscular dystrophy that use different AAV serotypes (like AAV9 or AAV8) instead of the specified scAAVrh74 vector.
- Does not cover gene therapies for muscular dystrophy that deliver genes other than alpha-sarcoglycan (hSGCA), such as the dystrophin gene for Duchenne muscular dystrophy.
- Does not cover non-viral methods of gene delivery, such as lipid nanoparticles or electroporation, for treating muscular dystrophy.
- Does not cover treatments for muscular dystrophy that do not involve gene therapy, such as physical therapy or steroid medications.
- Does not cover the use of this specific scAAVrh74.tMCK.hSGCA vector for treating diseases other than muscular dystrophy.
Patent timeline
Application submitted to the patent office
Patent officially issued
PatentBrief Score
Impact Score
Early stage
Citation count
0/40
No citations yet
Claim breadth
0/20
Narrow claimsclaimsThe numbered statements at the end of a patent that legally define what the inventor owns.Read more →
Recency
20/20
Granted within 5 years
Assignee scale
0/20
Independent or smaller assigneeassigneeThe entity that owns the patent — usually the inventor's employer or a company.Read more →
PatentBrief Impact Score — based on citation count, claim breadth, recency, and assignee scale. Not a legal assessment.
Heuristic Value Estimate
What this patent might be worth
$36K – $115K
Midpoint $72K · 16.1 yr remaining · industry ×3.0
Heuristic only — blends forward/backward citation counts, claim scope, time remaining, litigation history, and CPC-derived industry baseline. Real valuations need a professional appraisal.
Claim text not yet imported for this patent
Concepts involved
Cite this patent
(2026). Treating Muscular Dystrophy with a Self-Complementary AAV Gene Therapy (U.S. Patent No. 12,735,723). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12735723/self-complementary-adeno-associated-virus-vector-and-its-use-in-treatment-of
Auto-generated from the patent record. Double-check author order and the issue date against the official USPTO document before submitting.
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Common Questions
Frequently Asked Questions
What does Treating Muscular Dystrophy with a Self-Complementary AAV Gene Therapy cover?
This patent describes a method to treat muscular dystrophy by delivering a specific gene using a specially designed, self-complementary adeno-associated virus (AAV) vector.
When does this patent expire?
This patent is expected to expire on September 15, 2046, when the invention enters the public domain.
What problem does this patent solve?
Muscular dystrophy is a group of genetic diseases that cause progressive weakness and loss of muscle mass, often leading to severe disability. Gene therapy offers a promising approach to correct the underlying genetic defects, potentially slowing or halting disease progression. This patent focuses on a specific gene and delivery method, contributing to the development of targeted treatments for these debilitating conditions.
What does this patent NOT cover?
Does not cover gene therapies for muscular dystrophy that use different AAV serotypes (like AAV9 or AAV8) instead of the specified scAAVrh74 vector.
Patent monitoring