US2023279430A1PendingUtilityA1
Gene therapy for mucopolysaccharidosis iiia
Est. expiryNov 30, 2037(~11.3 yrs left)· nominal 20-yr term from priority
A61K 35/76C12N 7/00A61K 39/12C12N 2750/14122C12N 15/86A61P 19/00A61K 48/005C12N 9/14C12Y 310/01001C12N 2750/14143A01K 2217/075A01K 2227/105A01K 2267/0306A61P 27/00
68
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Claims
Abstract
Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional hSGSH, a regulatory sequence which direct expression of hSGSH in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIA.
Claims
exact text as granted — not AI-modified1 . A recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional N-sulfoglycosamine sulfohydrolase (hSGSH), a regulatory sequence which directs expression of hSGSH in a target cell, and an AAV 3′ ITR, wherein the hSGSH coding sequence is at least 95% identical to SEQ ID NO: 1.
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