IP Library › Granted Patent US 12,239,692
Granted Patent B2
US 12,239,692 · App. 17/854,286 · Granted Mar 4, 2025

Adeno-associated virus factor VIII vectors

Inventors: Peter Cameron Colosi (Novato, CA); Amit Nathwani (London, GB); Jenny McIntosh (London, GB); Edward Tuddenham (London, GB); Andrew Davidoff (Memphis, TN)
Assignees: BIOMARIN PHARMACEUTICAL INC.; UCL BUSINESS LTD; ST. JUDE CHILDREN'S RESEARCH HOSPITAL
A61K38/37A61K48/0058A61K48/0066C07K14/755C12N7/00C12N15/86C12N2750/14132C12N2750/14142C12N2750/14143C12N2750/14151C12N2800/22C12N2830/008
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Quick Facts
Patent No.
US 12,239,692
App. No.
17/854,286
Granted
Mar 4, 2025
Kind
B2
Abstract

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.

Claims (7)

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.

Assignments (1)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
Continuity (5)
Continuation 16588130 · Sep 30, 2019
Continuation 15294310 · Oct 14, 2016
Continuation 14482648 · Sep 10, 2014
Provisional Application 61877042 · Sep 12, 2013
Related Publication 20220339262A1 · Oct 27, 2022
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