LYSOSOMAL POLYPEPTIDES, METHODS OF MAKING AND USING

Title:

LYSOSOMAL POLYPEPTIDES, METHODS OF MAKING AND USING

Document Type and Number:

WIPO Patent Application WO/2013/013019

Kind Code:

A3

Abstract:

The invention generally relates to compositions and methods for producing lysosomal proteins that have altered glycan structure, such that the protein can be delivered efficiently into the lysosomes of target cells (such as macrophages). The lysosomal proteins are produced by modifying the glycosylation pathways in a host cell using an RNA effector molecule, such as an siRNA. Glycan-modified lysosomal proteins produced using the methods described herein have improved properties, including e.g., increased specific uptake by target cells (such as macrophages).

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Inventors:

ROSSOMANDO ANTHONY (US)
BETTENCOURT BRIAN (US)

Application Number:

PCT/US2012/047356

Publication Date:

May 10, 2013

Filing Date:

July 19, 2012

Export Citation:

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Assignee:

ALNYLAM PHARMACEUTICALS INC (US)
ROSSOMANDO ANTHONY (US)
BETTENCOURT BRIAN (US)

International Classes:

C12N15/00

Foreign References:

US20100186099A1	2010-07-22
US20110067146A1	2011-03-17

Other References:

SCHENK ET AL.: "MPDU1 mutations underlie a novel human congenital disorder of glycosylation, designated type If", J. CLIN. INVEST., vol. 108, 2001, pages 1687 - 1695

Attorney, Agent or Firm:

UNDERWOOD, Robert H. et al. (28 State StreetBoston, Massachusetts, US)

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