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US Patent 8361979 Means and method for inducing exon-skipping

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US Patent 12128109 Muscle targeting complexes and formulations for treating dystrophinopathies
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US Patent 12104156 Compositions and methods of treating facioscapulohumeral muscular dystrophy
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US Patent 12071621 Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
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US Patent 12064483 Nucleic acid-polypeptide compositions and methods of inducing exon skipping
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US Patent 12060558 Methods and compositions for treatment of cholesteryl ester storage disease
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US Patent 12005124 Muscle targeting complexes and uses thereof for treating dystrophinopathies
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US Patent 11999955 Compositions and methods of treating facioscapulohumeral muscular dystrophy
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US Patent 11986537 Muscle targeting complexes and uses thereof for treating dystrophinopathies
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US Patent 11932851 Exon skipping compositions for treating muscular dystrophy
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US Patent 11891605 Reducing intron retention
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US Patent 11873490 Antisense oligomers for treatment of conditions and diseases
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US Patent 11872287 Compositions and methods of treating muscle atrophy and myotonic dystrophy
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US Patent 11833217 Muscle targeting complexes and uses thereof for treating dystrophinopathies
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Patent abstract

In the present invention means and method are provided for optimising exon-skipping using exon-internal AON. We show that skipping efficiencies are improved by targeting putative splicing regulatory sequences (ESEs) within an exon. Such double targeting may be particularly useful for exons with which efficient skipping was difficult to obtain prior to the invention.

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US Patent 11771776 Muscle targeting complexes and uses thereof for treating dystrophinopathies
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Annemieke Aartsma-Rus0
Garrit-Jan Boudewijn Van Ommen0
Judith Christina T. Van Deutekom0
Edits on 2 Aug, 2023
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US Patent 11712478 Compositions and methods of treating muscle atrophy and myotonic dystrophy
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