Optimised coding sequence and promoter
US-9393323-B2 · Jul 19, 2016 · US
Adeno-associated virus factor VIII vectors
US12239692B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12239692-B2 |
| Application number | US-202217854286-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jun 30, 2022 |
| Priority date | Sep 12, 2013 |
| Publication date | Mar 4, 2025 |
| Grant date | Mar 4, 2025 |
How to read this patent
A practical reading order for non-experts. Skip the full description unless you need deep technical detail.
- Title
What the patent document calls the invention.
- Abstract
A short plain-language summary of the technical disclosure.
- Assignees and inventors
Who owns or filed the patent and who is credited as inventor.
- Key dates
Filing, priority, publication, and grant dates set the timeline.
- First independent claim
The legal scope of protection — read this for what is actually claimed.
- CPC / IPC classifications
Technology tags used to group this patent with similar filings.
- Citations and related patents
Prior art links and similar publications in this corpus.
Abstract
Official abstract text for this publication.
The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
First claim
Opening claim text (preview).
What is claimed: 1. An adeno-associated virus (AAV) vector comprising a 5′ inverted terminal repeat (ITR), a liver-specific transcription regulatory region operably linked to a codon-optimized nucleic acid sequence encoding a functionally active Factor VIII (FVIII) protein having the A3 and B domain deleted, and a 3′ ITR, wherein the nucleic acid sequence comprises nucleotides 646-4611 of SEQ ID NO: 5. 2. A method of producing a recombinant adeno-associated virus (AAV) particle comprising A) culturing a cell that has been transfected with the AAV vector of claim 1 and B) recovering recombinant AAV particle from the supernatant of the transfected cell. 3. A viral particle comprising the AAV vector of claim 1 . 4. An isolated host cell comprising the AAV vector of claim 1 . 5. The AAV vector of claim 1 wherein the nucleic acid sequence comprises nucleotide sequence of SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7, or SEQ ID NO: 8.
Assignees
Inventors
Classifications
Methods of production or purification of viral material · CPC title
Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent · CPC title
Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct · CPC title
cell type or tissue specific enhancer/promoter combination · CPC title
Vectors comprising a coding region that has been codon optimised for expression in a respective host · CPC title
Patent family
Related publications grouped by family.
External sources
Frequently asked questions
Answers are generated from the same data shown on this page.
- What does patent US12239692B2 cover?
- The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
- Who is the assignee on this patent?
- Biomarin Pharm Inc, Ucl Business Ltd, St Jude Childrens Res Hospital
- What technology area does this patent fall under?
- Primary CPC classification C12N15/86. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Mar 04 2025 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 1 related publication on this page (citations in our corpus or others sharing the same primary CPC).