Engineered AAV Vectors for Targeted Gene Delivery to Specific Body Parts
This patent describes specially designed adeno-associated virus (AAV) vectors that can deliver new genetic material to specific tissues in the body, such as the brain, heart, or retina, to treat diseases.
Original patent title: “Engineered adeno-associated (AAV) vectors for transgene expression”
This patent describes specially designed adeno-associated virus (AAV) vectors that can deliver new genetic material to specific tissues in the body, such as the brain, heart, or retina, to treat diseases. Granted in 2026.
Coverage
What does this patent actually cover?
This patent relates to engineered adeno-associated (AAV) vectors. These vectors are modified viruses used to carry new genes into cells. The engineering allows these AAV vectors to specifically deliver genetic material, called transgenes, to particular parts of the body. For example, the patent mentions targeting the central nervous system (CNS), peripheral nervous system (PNS), inner ear, heart, or retina. This means the vectors are designed to express a desired gene primarily in cells within these specific organs or tissues. The patent also describes methods for finding new engineered AAV vectors that can deliver genes to desired cell types.
The gap
What does this patent NOT cover?
- Does not cover gene delivery methods that use viral vectors other than adeno-associated viruses (AAV).
- Does not cover AAV vectors that are not specifically engineered for targeted transgene expression.
- Does not cover AAV vectors used for purposes other than delivering and expressing a transgene, such as direct gene editing without expression.
- Does not cover delivery of genetic material to tissues or organs not mentioned in the abstractabstractA short summary at the front of the patent describing the invention. Not legally binding.Read more →, unless they fall under the broad categories of CNS or PNS.
- Does not cover general, non-engineered AAV vectors that lack specificity for particular cell types or tissues.
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 engineering AAV vectors to specifically target certain cell types and tissues, like the brain or retina, for transgene expression. This precise targeting improves the safety and effectiveness of gene therapy by ensuring the therapeutic gene reaches the right place and avoids unintended cells.
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
Luxturna (voretigene neparvovec-rzyl) for inherited retinal disease
Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy
Gene therapies targeting specific neurological disorders
Experimental treatments for certain heart conditions
Research into therapies for hearing loss affecting the inner ear
Why it matters
The bigger picture
Targeted gene delivery is crucial for gene therapy, allowing treatments to reach only the affected cells while minimizing side effects elsewhere in the body. This approach can lead to more effective therapies for genetic diseases affecting specific organs like the eye or heart. The ability to precisely deliver genes opens doors for treating conditions that currently have limited options.
Filed
March 30, 2020
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Companies like Sarepta Therapeutics, Novartis Gene Therapies, and Spark Therapeutics are actively developing and commercializing AAV-based gene therapies. Many biotech startups and academic institutions are also focused on discovering and optimizing new engineered AAV capsids for improved tissue specificity and reduced immunogenicity, building on the principles of targeted delivery.
Market impact
The development of engineered AAV vectors has significantly expanded the potential for gene therapy, moving it from theoretical promise to approved treatments for previously untreatable diseases. This technology has enabled the creation of a new class of therapeutics, attracting substantial investment and fostering intense research and development in the biotechnology and pharmaceutical sectors. It has also spurred competition to develop safer and more effective delivery systems.
Claim 1 — Plain English
What this patent covers
This patent relates to engineered adeno-associated (AAV) vectors. These vectors are modified viruses used to carry new genes into cells. The engineering allows these AAV vectors to specifically deliver genetic material, called transgenes, to particular parts of the body. For example, the patent mentions targeting the central nervous system (CNS), peripheral nervous system (PNS), inner ear, heart, or retina. This means the vectors are designed to express a desired gene primarily in cells within these specific organs or tissues. The patent also describes methods for finding new engineered AAV vectors that can deliver genes to desired cell types.
The clever bit
The novelty lies in engineering AAV vectors to specifically target certain cell types and tissues, like the brain or retina, for transgene expression. This precise targeting improves the safety and effectiveness of gene therapy by ensuring the therapeutic gene reaches the right place and avoids unintended cells.
What it does not cover
- Does not cover gene delivery methods that use viral vectors other than adeno-associated viruses (AAV).
- Does not cover AAV vectors that are not specifically engineered for targeted transgene expression.
- Does not cover AAV vectors used for purposes other than delivering and expressing a transgene, such as direct gene editing without expression.
- Does not cover delivery of genetic material to tissues or organs not mentioned in the abstract, unless they fall under the broad categories of CNS or PNS.
- Does not cover general, non-engineered AAV vectors that lack specificity for particular cell types or tissues.
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 · 13.5 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). Engineered AAV Vectors for Targeted Gene Delivery to Specific Body Parts (U.S. Patent No. 12,735,721). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12735721/engineered-adeno-associated-aav-vectors-for-transgene-expression
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 Engineered AAV Vectors for Targeted Gene Delivery to Specific Body Parts cover?
This patent describes specially designed adeno-associated virus (AAV) vectors that can deliver new genetic material to specific tissues in the body, such as the brain, heart, or retina, to treat diseases.
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?
Targeted gene delivery is crucial for gene therapy, allowing treatments to reach only the affected cells while minimizing side effects elsewhere in the body. This approach can lead to more effective therapies for genetic diseases affecting specific organs like the eye or heart. The ability to precisely deliver genes opens doors for treating conditions that currently have limited options.
What does this patent NOT cover?
Does not cover gene delivery methods that use viral vectors other than adeno-associated viruses (AAV).
Patent monitoring