Enzymatic replacement therapy and antisense therapy for Pompe disease

Number of patents in Portfolio can not be more than 2000

United States of America

PATENT NO 11286483
APP PUB NO 20200138846A1
SERIAL NO

16622670

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Abstract

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The invention relates to method for repairing aberrant; splicing, wherein such aberrant: splicing is caused by the presence of a natural pseudo exon, comprising blocking of either the natural cryptic 3′ splice site or the natural cryptic 5′ splice site of said natural pseudo exon with an antisense oligomeric compound (AON). Further, the invention comprises an antisense oligomeric compound targeting SEQ ID NO: 1 or SEQ ID NO: 171, preferably selected from the sequences of SEQ ID NO: 267-2040, sequences that are complementary to said sequences or sequences that have an identity of 80% with said sequences or the complementary sequences. The invention further envisages the use of two antisense oligomeric compounds, a first AON targeting SEQ ID NO: 1 and a second targeting AON or SEQ ID NO: 171. These AONs are specifically for use in the treatment of Pompe disease. It is an aspect of the invention that antisense therapy using the above AONs or combinations thereof is used in combination with ERT.

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Patent Owner(s)

Patent OwnerAddress
ERASMUS UNIVERSITY MEDICAL CENTER ROTTERDAMDR MOLEWATERPLEIN 50 ROTTERDAM 3015 GE

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Bergsma, Atze Jacobus Utrecht, NL 7 8
Pijnappel, Wilhelmus Wenceslaus Matthias Vleuten, NL 12 11
Van, Der Ploeg Antje Tjitske Poortugaal, NL 11 10
Van, Der Wal Erik Schiedam, NL 11 10

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