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Title:
INTRATHECAL ADMINISTRATION OF ADENO-ASSOCIATED-VIRAL VECTORS FOR GENE THERAPY
Document Type and Number:
Japanese Patent JP2022116189
Kind Code:
A
Abstract:
To provide compositions for delivering immunoglobulin constructs, including without limitation, full-length antibodies, to the central nervous system.SOLUTION: A composition comprises at least one AAV vector formulated for intrathecal delivery for treatment of a neurodegenerative disorder such as Parkinson's disease, amyotrophic lateral sclerosis (ALS), multiple sclerosis, Alzheimer's disease, Huntington disease, wherein the composition comprises an AAV vector comprising an AAV capsid which targets a cell of the central nervous system, the capsid having packaged therein at least one AAV inverted terminal repeat sequence and sequences encoding an immunoglobulin construct operably linked to expression control sequences therefor, the composition further comprising a pharmaceutically acceptable carrier and/or excipient. The immunoglobulin construct may comprise an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn).SELECTED DRAWING: None

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Inventors:
JAMES M WILSON
CHRISTIAN HINDERER
WILLIAM THOMAS ROTHWELL
Application Number:
JP2022085777A
Publication Date:
August 09, 2022
Filing Date:
May 26, 2022
Export Citation:
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Assignee:
TRUSTEES OF THE UNIV OF PENNSYLVANIA
International Classes:
A61K35/76; A61K39/395; A61K48/00; A61P9/10; A61P17/02; A61P21/04; A61P25/00; A61P25/06; A61P25/14; A61P25/16; A61P25/28; A61P31/00; A61P37/06; A61P43/00
Other References:
THE JOURNAL OF NEUROSCIENCE, vol. 35(16), JPN6021013181, April 2015 (2015-04-01), pages 6265 - 6276, ISSN: 0005065976
BMC BIOTECHNOLOGY, vol. 9, JPN6021013180, 2009, pages 14, ISSN: 0005065977
MOLECULAR THERAPY, vol. 10, no. 2, JPN6022002691, 2004, pages 302 - 317, ISSN: 0005065978
Attorney, Agent or Firm:
Patent business corporation Odashima patent office



 
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