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US 2026/0295085 A1Published application
Gene therapy for treating mucopolysaccharidosis type ii
AssigneeThe Trustees of the University of Pennsylvania
InventorsChristian Hinderer, James M. Wilson
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Abstract
A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a hIDS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
Claims1
A pharmaceutical composition suitable for intrathecal administration in human subjects, comprising a suspension of replication deficient recombinant adeno-associated virus (rAAV) in a formulation buffer, wherein:
- (a) the rAAV comprises a heterologous nucleic acid encoding human iduronate-2-sulfatase (hIDS) packaged in an AAV9 capsid;
- (b) the formulation buffer comprises a physiologically compatible aqueous buffer, and optional surfactants and excipients;
- and
- (c) (i) the rAAV Genome Copy (GC) titer is at least 1.0×1013 GC/ml (+/−20%);
- and/or
- (ii) the rAAV Empty/Full particle ratio is at least about 80% free of empty capsids;
- and/or
- (iii) a dose of at least about 2.5×1010 GC/g brain mass to about 3.6×1011 GC/g brain mass of the rAAV suspension has potency.
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Dependencies are inferred from the claim text.