Gene therapy for treating phenylketonuria
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Jenny Agnes Sidrane, Scott Ashley
Life of this patent
Estimating the term…
Abstract
Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
Claims23
A recombinant adeno-associated virus (rAAV) useful as a liver-directed therapeutic for phenylketonuria (PKU), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising:
- (a) an AAV 5′ inverted terminal repeat (ITR) sequence;
- (b) a promoter;
- (c) a codon optimized sequence encoding a human phenylalanine hydroxylase (PAH);
- (d) an AAV 3′ ITR.
An aqueous suspension suitable for administration to a phenylketonuria patient, said suspension comprising an aqueous suspending liquid and about 1×1012 GC/mL to about 1×1014 GC/mL of a recombinant adeno-associated virus (rAAV) useful as a liver-directed therapeutic for phenylketonuria, said rAAV having an AAV capsid, and having packaged therein a vector genome comprising:
- (a) an AAV 5′ inverted terminal repeat (ITR) sequence;
- (b) a promoter;
- (c) a coding sequence encoding a human phenylalanine hydroxylase (PAH);
- and
- (d) an AAV 3′ ITR.
(canceled)
Dependencies are inferred from the claim text.