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SG10202107429WA - Nucleic acid-polypeptide compositions and methods of inducing exon skipping - Google Patents

Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Info

Publication number
SG10202107429WA
SG10202107429WA SG10202107429WA SG10202107429WA SG10202107429WA SG 10202107429W A SG10202107429W A SG 10202107429WA SG 10202107429W A SG10202107429W A SG 10202107429WA SG 10202107429W A SG10202107429W A SG 10202107429WA SG 10202107429W A SG10202107429W A SG 10202107429WA
Authority
SG
Singapore
Prior art keywords
methods
nucleic acid
exon skipping
polypeptide compositions
inducing exon
Prior art date
Application number
SG10202107429WA
Inventor
Arthur A Levin
Andrew John Geall
Venkata Ramana Doppalapudi
Michael Caramian COCHRAN
Hanhua Huang
Rob Burke
Original Assignee
Avidity Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Avidity Biosciences Inc filed Critical Avidity Biosciences Inc
Publication of SG10202107429WA publication Critical patent/SG10202107429WA/en

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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003—Peptide-nucleic acids (PNAs)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/321—2'-O-R Modification
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    • C12N2310/3513—Protein; Peptide
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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SG10202107429WA 2017-01-06 2018-01-05 Nucleic acid-polypeptide compositions and methods of inducing exon skipping SG10202107429WA (en)

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US201762443514P 2017-01-06 2017-01-06
US201762561939P 2017-09-22 2017-09-22

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EP (2) EP3565577A4 (en)
JP (3) JP7573966B2 (en)
KR (2) KR20190104381A (en)
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Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
CN108929325A (en) 2017-05-25 2018-12-04 深圳明赛瑞霖药业有限公司 Pkc inhibitor solid-state form
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
KR102850887B1 (en) 2017-08-03 2025-08-28 보이저 테라퓨틱스, 인크. Compositions and methods for delivering AAV
AU2018335880B2 (en) * 2017-09-22 2025-04-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
CN111655268B (en) 2017-10-04 2024-03-26 艾维迪提生物科学公司 Nucleic acid-polypeptide compositions and uses thereof
EP3720448A4 (en) 2017-12-06 2021-11-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2020028857A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20210324101A1 (en) * 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy
JP2021533196A (en) * 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. Muscle-targeted complexes and their use to treat progressive ossifying fibrodysplasia
JP7705793B2 (en) * 2018-08-02 2025-07-10 ダイン セラピューティクス，インコーポレーテッド Muscle-targeting complexes and their uses for treating myotonic dystrophy - Patent Application 20070123333
WO2020028844A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CA3108313A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
WO2020157760A1 (en) * 2019-01-31 2020-08-06 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
CA3142283A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
WO2020247818A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Una amidites and uses thereof
WO2020247738A1 (en) * 2019-06-07 2020-12-10 Dyne Therapeutics, Inc. Methods of preparing protein-oligonucleotide complexes
EP3987029A1 (en) * 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EP4007633A4 (en) * 2019-08-02 2024-05-08 Research Institute at Nationwide Children's Hospital NUCLEIC ACIDS TARGETING EXON 44 AND RECOMBINANT ADENO-ASSOCIATED VIRUS COMPRISING SAID NUCLEIC ACIDS FOR THE TREATMENT OF DYSTROPHIN-BASED MYOPATHIES
CN111020018B (en) * 2019-11-28 2021-09-14 天津金匙医学科技有限公司 Macrogenomics-based pathogenic microorganism detection method and kit
CA3159250A1 (en) * 2019-11-29 2021-06-03 Facio Intellectual Property B.V. New compounds for treatment of diseases related to dux4 expression
BR112022012114A2 (en) * 2019-12-19 2022-09-06 Nippon Shinyaku Co Ltd ANTISENSE NUCLEIC ACID THAT ALLOWS THE LEAP OF EXONS
US20230226212A1 (en) * 2020-01-10 2023-07-20 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US20230088865A1 (en) * 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2021205346A1 (en) * 2020-01-10 2022-09-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
TW202144572A (en) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 Compositions and methods of treating facioscapulohumeral muscular dystrophy
BR112022019137A2 (en) 2020-03-27 2023-01-31 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF MUSCULAR DYSTROPHY
EP4146229A4 (en) * 2020-05-05 2025-07-23 Avidity Biosciences Inc COMPOSITIONS AND METHODS FOR THE TREATMENT OF POMPE DISEASE
CA3186746A1 (en) * 2020-07-23 2022-01-27 Romesh R. Subramanian Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN111808887B (en) * 2020-09-10 2020-12-18 中国农业大学 A method for preparing double-muscle rump beef cattle similar to natural mutant Belgian blue cattle
IL303332A (en) * 2020-12-04 2023-07-01 Dyne Therapeutics Inc Antibody-oligonucleotide complexes and uses thereof
CN116916938A (en) * 2020-12-31 2023-10-20 达因疗法公司 Muscle-targeted complexes and their use in the treatment of facioscapulohumeral muscular dystrophy
US12286630B2 (en) 2021-04-30 2025-04-29 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
AU2022309028A1 (en) 2021-07-09 2024-01-25 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023121444A1 (en) 2021-12-22 2023-06-29 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
US20250250564A2 (en) 2021-12-22 2025-08-07 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders
ES3052234T3 (en) 2021-12-22 2026-01-02 Sapreme Tech Bv Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
WO2023122347A2 (en) * 2021-12-23 2023-06-29 Mirecule, Inc. Compositions for delivery of polynucleotides
CN119013401A (en) 2022-03-17 2024-11-22 萨勒普塔医疗公司 Phosphorodiamidite morpholines substituted oligomer conjugates
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CA3255934A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
JP2025533454A (en) * 2022-09-21 2025-10-07 サレプタ セラピューティクス， インコーポレイテッド DMD antisense oligonucleotide-mediated exon skipping efficiency
CN115820642B (en) * 2022-11-11 2023-10-10 昆明理工大学 A CRISPR-Cas9 system for treating Duchenne muscular dystrophy
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
AU2024308906A1 (en) 2023-06-30 2026-02-05 Avidity Biosciences, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
TW202532646A (en) * 2023-10-02 2025-08-16 美商亞維代堤生物科學公司 Antibody oligonucleotide conjugate compositions and methods of inducing dmd exon 45 skipping
US20250325693A1 (en) * 2024-04-03 2025-10-23 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 50 skipping

Family Cites Families (165)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1000075A (en) 1910-07-27 1911-08-08 William R Collins Tunneling-machine.
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
DE3687030T2 (en) 1985-03-15 1993-03-11 Eugene Stirchak STEREO-REGULAR POLYNUCLEOTID-BINDING POLYMERS.
US4828971A (en) 1988-03-24 1989-05-09 Eastman Kodak Company Thermally processable element comprising a backing layer
CA1251835A (en) 1988-04-05 1989-03-28 Wai-Cheung Tang Dielectric image-resonator multiplexer
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
DE552178T1 (en) 1990-10-12 1994-02-03 Max Planck Gesellschaft MODIFIED RIBOZYMS.
DE4216134A1 (en) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog SYNTHETIC CATALYTIC OLIGONUCLEOTIDE STRUCTURES
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
NZ288223A (en) 1994-06-17 1997-11-24 Hoffmann La Roche N,n'-bis(quinolin-4-yl)-diamine derivatives; antimalarial medicaments
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
ATE373672T1 (en) 1996-01-16 2007-10-15 Sirna Therapeutics Inc SYNTHESIS OF METHOXYNUCLEOSIDES AND ENZYMATIC NUCLEIC ACID MOLECULES
CA2249195A1 (en) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Immunoglobin-like domains with increased half lives
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
ATE375365T1 (en) 1998-04-02 2007-10-15 Genentech Inc ANTIBODIES VARIANTS AND FRAGMENTS THEREOF
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
DK1242438T3 (en) 1999-12-29 2007-02-12 Immunogen Inc Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
KR20020097241A (en) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 Splice-region antisense composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
WO2003071872A1 (en) 2002-02-22 2003-09-04 University Of Maryland, Baltimore Novel treatment of neurodegenerative diseases by altering levels of trkb isoforms and/or trkc isoforms
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
BRPI0313202A8 (en) 2002-08-05 2016-08-16 Atugen Ag ADDITIONAL WAYS TO INTERFERE WITH RNA MOLECULES
US7956176B2 (en) 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
CA2507125C (en) 2002-11-25 2014-04-22 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
US8084582B2 (en) 2003-03-03 2011-12-27 Xencor, Inc. Optimized anti-CD20 monoclonal antibodies having Fc variants
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
PT2489364E (en) 2003-11-06 2015-04-16 Seattle Genetics Inc Monomethylvaline compounds conjugated to antibodies
SI1766010T1 (en) 2004-06-28 2011-06-30 Univ Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2874384B1 (en) 2004-08-17 2010-07-30 Genethon ADENO-ASSOCIATED VIRAL VECTOR FOR PRODUCING EXON JUMP IN A GENE ENCODING A PROTEIN WITH DISPENSABLE DOMAINS
CA2586909A1 (en) 2004-11-12 2006-12-14 Seattle Genetics, Inc. Auristatins having an aminobenzoic acid unit at the n terminus
US7429482B2 (en) 2005-01-13 2008-09-30 United States Of America As Represented By The Department Of Veterans Affairs Screening tools for discovery of novel anabolic agents
CA2605512A1 (en) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure.
US7736647B2 (en) 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
EP1937312B1 (en) 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
EP2735568B8 (en) 2006-05-10 2017-09-27 Sarepta Therapeutics, Inc. Oligonucleotide analogs having cationic intersubunit linkages
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
US9063129B2 (en) * 2007-06-15 2015-06-23 Idexx Laboratories, Inc. Device, kit and method for hookworm antigen capture and detection
US20090099066A1 (en) 2007-06-29 2009-04-16 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
PL2203173T3 (en) 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
PL2265283T3 (en) 2008-03-18 2015-03-31 Seattle Genetics Inc Auristatin drug linker conjugates
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
WO2009147368A1 (en) 2008-06-04 2009-12-10 Medical Research Council Peptides
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
CA3066050A1 (en) 2008-10-24 2010-04-29 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
AU2013204438B2 (en) 2008-10-24 2016-12-22 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2741629C (en) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
KR101602431B1 (en) 2008-11-05 2016-03-10 삼성전자 주식회사 Refrigerator
EP2370451B1 (en) 2008-12-02 2016-11-16 Wave Life Sciences Japan, Inc. Method for the synthesis of phosphorus atom modified nucleic acids
CA2757354A1 (en) 2009-04-02 2010-10-07 Laura P.W. Ranum Nucleotide repeat expansion-associated polypeptides and uses thereof
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US8604184B2 (en) 2009-05-05 2013-12-10 The United States Of America As Represented By The Secretary Of The Air Force Chemically programmable immunity
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US20120172415A1 (en) * 2009-08-31 2012-07-05 Thomas Voit Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy
PL2486141T3 (en) 2009-10-07 2018-07-31 Macrogenics, Inc. Fc region-containing polypeptides that exhibit improved effector function due to alterations of the extent of fucosylation, and methods for their use
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
US9145556B2 (en) 2010-04-13 2015-09-29 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
JP5870400B2 (en) 2010-04-15 2016-03-01 シアトル ジェネティクス，インコーポレーテッド Targeted pyrrolobenzodiazepine conjugates
NZ602933A (en) 2010-04-15 2014-09-26 Seattle Genetics Inc Pyrrolobenzodiazepines used to treat proliferative diseases
TWI620756B (en) 2010-05-28 2018-04-11 薩羅塔治療公司 Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
IT1400425B1 (en) * 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv MODIFIED SNRNAS FOR USE IN THERAPY.
US8936910B2 (en) 2010-06-11 2015-01-20 Antisense Pharma Gmbh Method for selective oligonucleotide modification
EP3031920B1 (en) 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX2013009191A (en) 2011-02-08 2013-11-04 Charlotte Mecklenburg Hospital Antisense oligonucleotides.
KR101614195B1 (en) 2011-03-29 2016-04-20 로슈 글리카트 아게 Antibody fc variants
US20140128449A1 (en) 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2708560A4 (en) 2011-05-09 2015-01-14 Perseus Proteomics Inc ANTIBODIES SPECIFICALLY RECEIVING A TRANSFERRIN RECEPTOR
CA2839593A1 (en) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
CN103998458B (en) 2011-08-30 2018-10-09 医学研究理事会 Cell-penetrating peptides with central hydrophibic domain
JP6393617B2 (en) 2011-10-14 2018-09-19 シアトル ジェネティクス，インコーポレーテッド Pyrrolobenzodiazepine and target conjugates
LT3366775T (en) 2011-11-18 2022-08-10 Alnylam Pharmaceuticals, Inc. Modified rnai agents
BR112014018427B1 (en) 2012-01-27 2021-11-03 Biomarin Technologies B.V. RNA MODULATOR OLIGONUCLEOTIDES WITH IMPROVED FEATURES FOR THE TREATMENT OF DUCHENNE AND BECKER'S MUSCULAR DYSTROPHY
WO2013121175A1 (en) 2012-02-16 2013-08-22 Ucl Business Plc Lysosome-cleavable linker
AR090905A1 (en) 2012-05-02 2014-12-17 Merck Sharp & Dohme CONJUGATES CONTAINING TETRAGALNAC AND PEPTIDES AND PROCEDURES FOR THE ADMINISTRATION OF OLIGONUCLEOTIDES, PHARMACEUTICAL COMPOSITION
CN104662000B (en) 2012-05-15 2018-08-17 索伦托医疗有限公司 Drug conjugates and its coupling method and purposes
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
CA2877644A1 (en) 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
CN104661664B (en) 2012-07-13 2020-07-03 波涛生命科学有限公司 Chiral control
JP5993093B2 (en) 2012-10-12 2016-09-14 メドイミューン・リミテッドＭｅｄＩｍｍｕｎｅ Ｌｉｍｉｔｅｄ Pyrrolobenzodiazepines and their complexes
JP6133431B2 (en) 2012-11-24 2017-05-24 ハンジョウ ディーエーシー バイオテック シーオー．，エルティディ．Ｈａｎｇｚｈｏｕ Ｄａｃ Ｂｉｏｔｅｃｈ Ｃｏ．，Ｌｔｄ． Use of hydrophilic conjugates and conjugation reactions between drug molecules and cell binding molecules
KR20200143739A (en) 2012-12-20 2020-12-24 사렙타 쎄러퓨틱스 인코퍼레이티드 Improved exon skipping compositions for treating muscular dystrophy
TWI716339B (en) 2012-12-21 2021-01-21 荷蘭商台醫（有限合夥）公司 Hydrophilic self-immolative linkers and conjugates thereof
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
US9319764B2 (en) 2013-03-08 2016-04-19 Merry Electronics Co., Ltd. MEMS microphone packaging structure
MX366485B (en) 2013-03-14 2019-07-10 Sarepta Therapeutics Inc COMPOSITIONS FOR EXON JUMPING FOR THE TREATMENT OF MUSCULAR DYSTROPHY.
EP2968593B1 (en) 2013-03-15 2024-11-20 Regeneron Pharmaceuticals, Inc. Biologically active molecules, conjugates thereof, and therapeutic uses
EP2777714A1 (en) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Method of producing an immunoligand/payload conjugate by means of a sequence-specific transpeptidase enzyme
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
US9849066B2 (en) 2013-04-24 2017-12-26 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
GB2529356B (en) 2013-04-28 2020-12-23 Genequantum Healthcare Co Ltd Novel linkers, coupling intermediates, conjugates, preparation method and application thereof
PL3594240T3 (en) 2013-05-20 2024-04-02 F. Hoffmann-La Roche Ag Anti-transferrin receptor antibodies and methods of use
ES2736326T3 (en) 2013-05-31 2019-12-27 Zymeworks Inc Heteromultimers with reduced or silenced effector function
ES2987399T3 (en) 2013-06-05 2024-11-14 Univ Duke RNA-guided gene editing and gene regulation
WO2014197854A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Novel linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
TW201536329A (en) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc Compound and method for regulating the manifestation of dystrophic myotonic protein kinase (DMPK)
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
US9982260B2 (en) 2013-09-30 2018-05-29 The Regents Of The University Of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
US10836821B2 (en) 2013-10-15 2020-11-17 Sorrento Therapeutics, Inc. Drug-conjugates with a targeting molecule and two different drugs
MX2021008464A (en) 2013-10-15 2023-03-03 Seattle Genetics Inc Pegylated drug-linkers for improved ligand-drug conjugate pharmacokinetics.
US10239957B2 (en) 2013-11-06 2019-03-26 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
WO2015075747A1 (en) 2013-11-19 2015-05-28 Council Of Scientific And Industrial Research Cell-penetrating peptide for biomolecule delivery
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
BR112016020618B1 (en) 2014-03-12 2023-04-04 Nippon Shinyaku Co., Ltd. ANTISENSE OLIGOMER, PHARMACEUTICAL COMPOSITION, AND, USE OF AN ANTISENSE OLIGOMER
RS59764B1 (en) 2014-06-17 2020-02-28 Nippon Shinyaku Co Ltd Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
AU2016264471B2 (en) * 2015-05-19 2022-01-27 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
DK3313879T3 (en) 2015-06-24 2022-03-14 Hoffmann La Roche Anti-transferrin receptor antibodies with adapted affinity
TWI741988B (en) * 2015-07-31 2021-10-11 日商新力股份有限公司 Stacked lens structure, method of manufacturing the same, and electronic apparatus
EP3653216A1 (en) 2015-09-30 2020-05-20 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
JP2019515874A (en) 2016-03-01 2019-06-13 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト Obintuzumab variants with altered cell death induction
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
MA45290A (en) 2016-05-04 2019-03-13 Wave Life Sciences Ltd PROCESSES AND COMPOSITIONS OF BIOLOGICALLY ACTIVE AGENTS
CN109310765A (en) 2016-06-20 2019-02-05 领先基因生物技术股份有限公司 Antibody-drug conjugates
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
US10074359B2 (en) 2016-11-01 2018-09-11 Google Llc Dynamic text-to-speech provisioning
LT3554553T (en) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
AU2018335880B2 (en) 2017-09-22 2025-04-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
EP3720448A4 (en) 2017-12-06 2021-11-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
WO2019232302A1 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Optoelectronic memristor devices
US12371694B2 (en) 2018-11-14 2025-07-29 University Of Hawaii Compositions and methods for the synthesis and identification of covalent aptamers
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
WO2021003573A1 (en) 2019-07-09 2021-01-14 The Governors Of The University Of Alberta Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene
US20230226212A1 (en) 2020-01-10 2023-07-20 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US20250302979A1 (en) 2021-04-02 2025-10-02 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate and antibody-peptide-oligonucleotide conjugate compositions and methods of inducing exon skipping
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

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