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Patent Searching and Data


Title:
RECOMBINANT ADENO-ASSOCIATED VIRUS WITH MODIFIED AAV CAPSID POLYPEPTIDES
Document Type and Number:
WIPO Patent Application WO/2023/155828
Kind Code:
A1
Abstract:
The present invention relates to a modified adeno-associated virus (AAV) capsid polypeptide and a novel recombinant adeno-associated virus (rAAV) comprising the modified AAV capsid polypeptide for delivering the gene product for the therapy. The present invention also relates to a pharmaceutical composition comprising the rAAV of the present invention, and a method of treating a retinal disease comprising the administration of the rAAV or the pharmaceutical composition of the present invention to an eye of a subject in need thereof.

Inventors:
SHU YANQUN (CN)
HOU JINZHAO (CN)
LI HEPING (CN)
REN QIONGQIONG (CN)
XIAO LUMEI (CN)
KONG HUIMIN (CN)
WU BING (CN)
LI PEILU (CN)
Application Number:
PCT/CN2023/076389
Publication Date:
August 24, 2023
Filing Date:
February 16, 2023
Export Citation:
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Assignee:
SKYLINE THERAPEUTICS SHANGHAI CO LTD (CN)
SKYLINE THERAPEUTICS LTD (GB)
International Classes:
C07K14/005; A61P27/02; C12N15/86
Domestic Patent References:
WO2020180951A12020-09-10
WO2021204407A12021-10-14
WO2021084133A12021-05-06
WO2012145601A22012-10-26
WO2016134375A12016-08-25
WO2018022905A22018-02-01
Foreign References:
US20050053922A12005-03-10
US20090202490A12009-08-13
US7271002B22007-09-18
US29795807A2007-08-24
US7846730B22010-12-07
Other References:
"GenBank", Database accession no. NC_006261
ESTHER J. LEE ET AL.: "Adeno-Associated Virus (AAV) Vectors: Rational Design Strategies for Capsid Engineering", CURR OPIN BIOMED ENG., vol. 7, 2018, pages 58 - 63, XP055953373, DOI: 10.1016/j.cobme.2018.09.004
CROSSON SM ET AL.: "Helper-free Production of Laboratory Grade AAV and Purification by Iodixanol Density Gradient Centrifugation", MOL THER METHODS CLIN DEV., vol. 10, 2018, pages 1 - 7
A. GENNARO: "Remington: The Science and Practice of Pharmacy", 2000, LIPPINCOTT, WILLIAMS, & WILKINS
"Pharmaceutical Dosage Forms and Drug Delivery Systems", 1999, LIPPINCOTT, WILLIAMS, & WILKINS
PENNES ET AL.: "Animal models of age related macular degeneration", MOL ASPECTS MED, vol. 33, no. 4, 2012, pages 487 - 509
Attorney, Agent or Firm:
NTD UNIVATION INTELLECTUAL PROPERTY AGENCY LTD. (CN)
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