How Oligonucleotides Target C9orf72 for Brain Diseases
This patent describes using special genetic molecules called oligonucleotides to treat brain diseases like ALS and FTD by targeting a specific gene called C9orf72.
Original patent title: “Oligonucleotide compositions and methods of use thereof”
This patent describes using special genetic molecules called oligonucleotides to treat brain diseases like ALS and FTD by targeting a specific gene called C9orf72. Granted in 2026.
Coverage
What does this patent actually cover?
The patent describes specific oligonucleotide compositions designed to interact with the C9orf72 gene. These oligonucleotides are intended to be used in methods for treating conditions linked to this gene, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). For example, a patient with C9orf72-associated ALS could receive a composition containing these oligonucleotides to potentially reduce the harmful effects of the gene.
The gap
What does this patent NOT cover?
- Does not cover treatments for ALS or FTD that are not linked to the C9orf72 gene mutation.
- Does not cover therapies for C9orf72-associated conditions that use non-oligonucleotide approaches, such as small molecule drugs or gene editing tools like CRISPR.
- Does not cover diagnostic methods for identifying the C9orf72 mutation.
- Does not cover oligonucleotides designed to target genes other than C9orf72.
These exclusions are unique to PatentBrief — derived from the actual claim language, not patent-office boilerplate.
Key facts
What made this novel
The clever bit is precisely designing oligonucleotide molecules to specifically interfere with the C9orf72 gene, which is known to cause certain forms of ALS and FTD. This targeted approach aims to correct or mitigate the genetic defect rather than just treating symptoms.
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
Investigational oligonucleotide therapies for C9orf72-ALS
Antisense oligonucleotide drugs in development
Gene-targeted treatments for neurodegenerative disorders
Why it matters
The bigger picture
This patent addresses severe neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which currently have limited treatment options. The C9orf72 gene mutation is a major genetic cause for both conditions, making targeted therapies crucial. Developing specific oligonucleotides to modulate this gene could offer a new therapeutic approach for patients suffering from these debilitating diseases.
Filed
October 5, 2020
Granted
September 15, 2026
Market context
Who's building on this
Companies in this space
Companies like Biogen, Ionis Pharmaceuticals, and Wave Life Sciences are actively developing oligonucleotide therapies for neurodegenerative diseases, including those targeting C9orf72. These companies are investing heavily in research and clinical trials to bring such treatments to patients with conditions like ALS and FTD.
Market impact
As a recently filed and granted patent, its market impact is still developing. However, if successful, therapies based on this technology could create a new category of targeted treatments for C9orf72-associated neurodegenerative diseases, potentially offering hope where few options currently exist and attracting significant investment in the gene therapy space.
Claim 1 — Plain English
What this patent covers
The patent describes specific oligonucleotide compositions designed to interact with the C9orf72 gene. These oligonucleotides are intended to be used in methods for treating conditions linked to this gene, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). For example, a patient with C9orf72-associated ALS could receive a composition containing these oligonucleotides to potentially reduce the harmful effects of the gene.
The clever bit
The clever bit is precisely designing oligonucleotide molecules to specifically interfere with the C9orf72 gene, which is known to cause certain forms of ALS and FTD. This targeted approach aims to correct or mitigate the genetic defect rather than just treating symptoms.
What it does not cover
- Does not cover treatments for ALS or FTD that are not linked to the C9orf72 gene mutation.
- Does not cover therapies for C9orf72-associated conditions that use non-oligonucleotide approaches, such as small molecule drugs or gene editing tools like CRISPR.
- Does not cover diagnostic methods for identifying the C9orf72 mutation.
- Does not cover oligonucleotides designed to target genes other than C9orf72.
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 · 14.0 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). How Oligonucleotides Target C9orf72 for Brain Diseases (U.S. Patent No. 12,735,703). U.S. Patent and Trademark Office. https://patentbrief.org/patent/us/12735703/oligonucleotide-compositions-and-methods-of-use-thereof
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 How Oligonucleotides Target C9orf72 for Brain Diseases cover?
This patent describes using special genetic molecules called oligonucleotides to treat brain diseases like ALS and FTD by targeting a specific gene called C9orf72.
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?
This patent addresses severe neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which currently have limited treatment options. The C9orf72 gene mutation is a major genetic cause for both conditions, making targeted therapies crucial. Developing specific oligonucleotides to modulate this gene could offer a new therapeutic approach for patients suffering from these debilitating diseases.
What does this patent NOT cover?
Does not cover treatments for ALS or FTD that are not linked to the C9orf72 gene mutation.
Patent monitoring