WO2024064237A3 - Dmd antisense oligonucleotide-mediated exon skipping efficiency - Google Patents

Dmd antisense oligonucleotide-mediated exon skipping efficiency Download PDF

Info

Publication number
WO2024064237A3
WO2024064237A3 PCT/US2023/033297 US2023033297W WO2024064237A3 WO 2024064237 A3 WO2024064237 A3 WO 2024064237A3 US 2023033297 W US2023033297 W US 2023033297W WO 2024064237 A3 WO2024064237 A3 WO 2024064237A3
Authority
WO
WIPO (PCT)
Prior art keywords
dmd
antisense oligonucleotide
exon skipping
skipping efficiency
mediated exon
Prior art date
Application number
PCT/US2023/033297
Other languages
French (fr)
Other versions
WO2024064237A2 (en
Inventor
Chia-Ling Wu
Frederick Joseph Schnell
Remko GOOSSENS
Nisha VERWEY
Annemieke Aartsma-Rus
Yavuz ARIYUREK
Original Assignee
Sarepta Therapeutics, 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 Sarepta Therapeutics, Inc. filed Critical Sarepta Therapeutics, Inc.
Publication of WO2024064237A2 publication Critical patent/WO2024064237A2/en
Publication of WO2024064237A3 publication Critical patent/WO2024064237A3/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Provided herein are oligonucleotides, cell-penetrating peptides, and peptide- oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein.
PCT/US2023/033297 2022-09-21 2023-09-20 Dmd antisense oligonucleotide-mediated exon skipping efficiency WO2024064237A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263408505P 2022-09-21 2022-09-21
US63/408,505 2022-09-21

Publications (2)

Publication Number Publication Date
WO2024064237A2 WO2024064237A2 (en) 2024-03-28
WO2024064237A3 true WO2024064237A3 (en) 2024-05-02

Family

ID=88412152

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/033297 WO2024064237A2 (en) 2022-09-21 2023-09-20 Dmd antisense oligonucleotide-mediated exon skipping efficiency

Country Status (1)

Country Link
WO (1) WO2024064237A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014007620A2 (en) * 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
WO2018129384A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2021108640A1 (en) * 2019-11-27 2021-06-03 Dtx Pharma, Inc. Compounds and methods for the treatment of duchenne muscular dystrophy

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
PT1178999E (en) 1999-05-04 2007-06-26 Santaris Pharma As L-ribo-lna analogues
JP2005514005A (en) 2001-09-04 2005-05-19 エクシコン エ／エス Novel LNA compositions and uses thereof
KR100464261B1 (en) 2002-01-24 2005-01-03 주식회사 파나진 A Novel Monomer For Synthesis of PNA Oligomer And A Process For Producing The Same
KR20030084444A (en) 2002-04-26 2003-11-01 주식회사 파나진 A Novel Monomer For Synthesis of PNA Oligomer And A Process For Producing The Same
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
CA2504554A1 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. 2'-substituted oligomeric compounds and compositions for use in gene modulations
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
HUE036995T2 (en) 2006-05-10 2018-08-28 Sarepta Therapeutics Inc Oligonucleotide analogs having cationic intersubunit linkages
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
MX2010004955A (en) 2007-11-15 2010-06-30 Avi Biopharma Inc Method of synthesis of morpholino oligomers.
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
JP2012523225A (en) 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー Tricyclo-DNA antisense oligonucleotides, compositions and methods for treatment of disease
BR112012000828A8 (en) 2009-07-06 2017-10-10 Ontorii Inc NEW NUCLEIC ACID PRO-DRUGS AND METHODS OF THEIR USE
JP5878758B2 (en) 2009-09-16 2016-03-08 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ Novel protecting groups for the synthesis of RNA and its derivatives
WO2011150408A2 (en) 2010-05-28 2011-12-01 Avi Biopharma, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
WO2012039448A1 (en) 2010-09-24 2012-03-29 株式会社キラルジェン Asymmetric auxiliary group
JP5831455B2 (en) 2010-09-30 2015-12-09 日本新薬株式会社 Morpholino nucleic acid derivatives
EP2704749A1 (en) 2011-05-05 2014-03-12 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2581448B1 (en) 2011-10-13 2015-01-28 Association Institut de Myologie Tricyclo-phosphorothioate DNA
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
DE102012101676A1 (en) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Method and device for processing forms with a data processing system
KR20220139425A (en) 2012-07-13 2022-10-14 웨이브 라이프 사이언시스 리미티드 Chiral control
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
WO2015108048A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
PT3094728T (en) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Chiral design
WO2016070166A2 (en) 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
MA43072A (en) 2015-07-22 2018-05-30 Wave Life Sciences Ltd COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
EP3359523A4 (en) 2015-10-09 2019-07-24 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
MA45270A (en) 2016-05-04 2017-11-09 Wave Life Sciences Ltd COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
MA45290A (en) 2016-05-04 2019-03-13 Wave Life Sciences Ltd PROCESSES AND COMPOSITIONS OF BIOLOGICALLY ACTIVE AGENTS

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014007620A2 (en) * 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
WO2018129384A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2021108640A1 (en) * 2019-11-27 2021-06-03 Dtx Pharma, Inc. Compounds and methods for the treatment of duchenne muscular dystrophy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ADAMS ABBIE M ET AL: "Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries", BMC MOLECULAR BIOLOGY, BIOMED CENTRAL LTD, GB, vol. 8, no. 1, 2 July 2007 (2007-07-02), pages 57, XP021028227, ISSN: 1471-2199, DOI: 10.1186/1471-2199-8-57 *
STEVE D WILTON ET AL: "Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 15, no. 7, 1 July 2007 (2007-07-01), pages 1288 - 1296, XP002625829, ISSN: 1525-0024, [retrieved on 20070206], DOI: 10.1038/SJ.MT.6300095 *

Also Published As

Publication number Publication date
WO2024064237A2 (en) 2024-03-28

Similar Documents

Publication Publication Date Title
EP4306538A3 (en) Peptide oligonucleotide conjugates
MXPA05007822A (en) Combination therapy for treating protein deficiency disorders.
DK1857122T3 (en) Stabilized mRNA with increased G / C content, encoding a viral antigen
EP1728863A3 (en) C-class oligonucleotide analogs with enhanced immunostimulatory potency
MX2012004625A (en) Peptides used in the treatment and/or care of the skin, mucous membranes and/or hair and its use in cosmetic or pharmaceutical compositions.
UA105210C2 (en) Anti-cancer vaccine and use thereof
NZ601677A (en) Improved cancer therapy based on tumor associated antigens derived from cyclin d1
EP2628749A3 (en) Antimicrobial kinocidin compostions and methods of use
AU8180001A (en) Human growth hormone to stimulate mobilization of pluripotent hematopoietic stemcells
WO2021222476A3 (en) High efficiency gene delivery system
MX2020009150A (en) Cpg amphiphiles and uses thereof.
MX2022000143A (en) Novel methods.
MX2020010071A (en) Antimicrobial, bacteriophage-derived polypeptides and their use against gram-negative bacteria.
DK1334119T3 (en) Compositions and Methods for Modulating RSV Infection and Immunity
WO2022010938A3 (en) Catalysis deactivated angiotensin-converting enzyme 2 (ace2) variants and their uses
CA2530900A1 (en) Peptides and peptidomimetics having immune-modulating, anti-inflammatory, and anti-viral activity
WO2024064237A3 (en) Dmd antisense oligonucleotide-mediated exon skipping efficiency
MX2018007307A (en) Peptide oligonucleotide conjugates.
WO2020154661A8 (en) Flow modifying implants
PT1603584E (en) Aplidine for multiple myeloma treatment
EP4108263A3 (en) Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
CA2755897A1 (en) Treatment of cancers with immunostimulatory hiv tat derivative polypeptides
WO2004033479A3 (en) Retroyclins: antiviral and antimicrobial peptides
MX2012005497A (en) Tivozanib and temsirolimus in combination.
MX2022008036A (en) New use of bcg immunogenic formulation expressing a respiratory syncitial virus protein against hmpv.

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 23789830

Country of ref document: EP

Kind code of ref document: A2