Patent · US Active

Adeno associated virus vectors for the treatment of hunter disease

US11981912B2 · kind B2 · utility

0Cited by
7References
17Claims
0Family size

Assignee

Inventors

Key dates

Filing dateDec 9, 2020
Grant dateMay 14, 2024
Priority date—
Expiry dateJun 21, 2041

Classification

  • Technology area (CPC C)Chemistry; Metallurgy
  • CPC primaryC12Y301/06013
  • WIPO fieldPharmaceuticals
  • WIPO sectorChemistry

Abstract

The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.