Adeno-associated virus (aav) vector lipid nanoparticle compositions and methods of use
AssigneeSpark Therapeutics, Inc.
InventorsAndrew William SHAW, Xavier ANGUELA
Life of this patent
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Abstract
A composition includes an adeno-associated virus (AAV) vector in a lipid nanoparticle (LNP). The AAV vector can include a heterologous nucleic acid sequence, optionally an inverted terminal repeat (ITR) positioned 5′ of the heterologous nucleic acid sequence and an ITR positioned 3′ of the heterologous nucleic acid sequence. The AAV vector can further include expression control elements (e.g., a promoter and/or enhancer), and intron, and/or or a polyadenylation signal.
Claims23
A composition comprising an adeno-associated virus (AAV) vector in a lipid nanoparticle (LNP), said AAV vector comprising a heterologous nucleic acid sequence and an inverted terminal repeat (ITR) positioned 5′ of said heterologous nucleic acid sequence and an ITR positioned 3′ of said heterologous nucleic acid sequence.
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12-13. (canceled)
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A method of making a composition comprising an AAV vector in a lipid nanoparticle (LNP), said method comprising:
- coating a vessel with one or more lipids to form a LNP;
- and
- adding said AAV vector to said lipid coated vessel, wherein said LNP further comprises polyethylene glycol (PEG) or a PEG-modified lipid in an amount from about 0.5% by weight of said LNP to about 10% by weight of said LNP.
A method of making a composition comprising an AAV vector in a lipid nanoparticle (LNP), said method comprising:
- coating a vessel with said AAV vector;
- and
- adding said LNP or one or more lipids to said AAV coated vessel, wherein said LNP further comprises polyethylene glycol (PEG) or a PEG-modified lipid in an amount from about 0.5% by weight of said LNP to about 10% by weight of said LNP.
24-29. (canceled)
Dependencies are inferred from the claim text.