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)
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:
Assignee:
SKYLINE THERAPEUTICS SHANGHAI CO LTD (CN)
SKYLINE THERAPEUTICS LTD (GB)
SKYLINE THERAPEUTICS LTD (GB)
International Classes:
C07K14/005; A61P27/02; C12N15/86
Domestic Patent References:
WO2020180951A1 | 2020-09-10 | |||
WO2021204407A1 | 2021-10-14 | |||
WO2021084133A1 | 2021-05-06 | |||
WO2012145601A2 | 2012-10-26 | |||
WO2016134375A1 | 2016-08-25 | |||
WO2018022905A2 | 2018-02-01 |
Foreign References:
US20050053922A1 | 2005-03-10 | |||
US20090202490A1 | 2009-08-13 | |||
US7271002B2 | 2007-09-18 | |||
US29795807A | 2007-08-24 | |||
US7846730B2 | 2010-12-07 |
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
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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