CA3173049A1 - Antisense nucleic acid inducing skipping of exon 51 - Google Patents

Antisense nucleic acid inducing skipping of exon 51

Info

Publication number
CA3173049A1
CA3173049A1 CA3173049A CA3173049A CA3173049A1 CA 3173049 A1 CA3173049 A1 CA 3173049A1 CA 3173049 A CA3173049 A CA 3173049A CA 3173049 A CA3173049 A CA 3173049A CA 3173049 A1 CA3173049 A1 CA 3173049A1
Authority
CA
Canada
Prior art keywords
antisense oligomer
exon
base sequence
nos
seq
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3173049A
Other languages
English (en)
French (fr)
Inventor
Yu Honda
Kaname Muchima
Takahiro Fukui
Saki HASEGAWA
Shin'ichi Takeda
Yoshitsugu Aoki
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
Original Assignee
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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 Nippon Shinyaku Co Ltd, National Center of Neurology and Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of CA3173049A1 publication Critical patent/CA3173049A1/en
Pending legal-status Critical Current

Links

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3173049A 2020-02-28 2021-02-26 Antisense nucleic acid inducing skipping of exon 51 Pending CA3173049A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2020033483 2020-02-28
JP2020-033483 2020-02-28
PCT/JP2021/007286 WO2021172498A1 (ja) 2020-02-28 2021-02-26 エクソン51のスキッピングを誘導するアンチセンス核酸

Publications (1)

Publication Number Publication Date
CA3173049A1 true CA3173049A1 (en) 2021-09-02

Family

ID=77491251

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3173049A Pending CA3173049A1 (en) 2020-02-28 2021-02-26 Antisense nucleic acid inducing skipping of exon 51

Country Status (17)

Country Link
US (2) US20230140736A1 (https=)
EP (1) EP4112083A4 (https=)
JP (3) JPWO2021172498A1 (https=)
KR (1) KR20220145865A (https=)
CN (1) CN115210376A (https=)
AU (1) AU2021226089A1 (https=)
BR (1) BR112022017066A2 (https=)
CA (1) CA3173049A1 (https=)
CL (3) CL2022002279A1 (https=)
CO (1) CO2022013685A2 (https=)
EC (1) ECSP22074446A (https=)
IL (1) IL295967A (https=)
MX (1) MX2022010545A (https=)
PE (1) PE20230237A1 (https=)
TW (2) TWI901457B (https=)
WO (1) WO2021172498A1 (https=)
ZA (1) ZA202209294B (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7842395B2 (ja) * 2019-12-19 2026-04-08 日本新薬株式会社 エクソンスキッピングを可能にするアンチセンス核酸
CA3244382A1 (en) 2021-12-27 2025-02-26 Nippon Shinyaku Co., Ltd. PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
JP3398378B2 (ja) 1989-12-20 2003-04-21 アンチビラルス・インコーポレイテツド リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体,デバイス及び医薬組成物
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
JP4777777B2 (ja) 2002-11-25 2011-09-21 雅文 松尾 mRNA前駆体のスプライシングを修飾するENA核酸医薬
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
DK2735568T3 (da) 2006-05-10 2017-11-13 Sarepta Therapeutics Inc Oligonukleotidanaloger med kationiske bindinger mellem underenheder
EP3443976A1 (en) 2007-06-29 2019-02-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
CN112574988A (zh) 2008-10-24 2021-03-30 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
JP5786109B2 (ja) 2008-10-27 2015-09-30 プロセンサ テクノロジーズ ベー.フェー. Duchenne型筋ジストロフィーmRNA前駆体のエクソン45の効率的なスキッピングのための方法及び手段
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012150960A1 (en) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
CN117721110A (zh) * 2011-12-28 2024-03-19 日本新药株式会社 反义核酸
CN112251436A (zh) * 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP2870246B1 (en) * 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
JP6519842B2 (ja) 2013-10-04 2019-05-29 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸
SMT201900169T1 (it) * 2014-03-12 2019-05-10 Nippon Shinyaku Co Ltd Acido nucleico antisenso
RU2695430C2 (ru) * 2014-06-17 2019-07-23 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
EP3297649B1 (en) 2015-05-19 2023-10-11 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
SI3331891T1 (sl) 2015-08-05 2022-04-29 Eisai R&D Management Co., Ltd. Postopek priprave v bistvu diastereomerno čistega fosforodiamidatnega oligomera, fosforodiamidatni oligomer izdelan po takem postopku in farmacevtski sestavek, ki vsebuje tak fosforodiamidatni oligomer
SI3351633T1 (sl) * 2015-09-15 2020-09-30 Nippon Shinyaku Co., Ltd. Protismiselna nukleinska kislina
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
IL297528A (en) 2016-12-19 2022-12-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
SMT202200366T1 (it) * 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
SI3554554T1 (sl) 2016-12-19 2023-02-28 Sarepta Therapeutics, Inc. Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo
WO2019241385A2 (en) 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy

Also Published As

Publication number Publication date
EP4112083A1 (en) 2023-01-04
BR112022017066A2 (pt) 2022-11-16
KR20220145865A (ko) 2022-10-31
ECSP22074446A (es) 2022-10-31
IL295967A (en) 2022-10-01
JP2022180420A (ja) 2022-12-06
US20230097387A1 (en) 2023-03-30
CL2022002279A1 (es) 2023-02-10
TW202515585A (zh) 2025-04-16
JP7292636B2 (ja) 2023-06-19
PE20230237A1 (es) 2023-02-07
CL2024001472A1 (es) 2024-09-27
US11781140B2 (en) 2023-10-10
CN115210376A (zh) 2022-10-18
AU2021226089A1 (en) 2022-09-15
WO2021172498A1 (ja) 2021-09-02
US20230140736A1 (en) 2023-05-04
ZA202209294B (en) 2024-04-24
MX2022010545A (es) 2022-09-21
EP4112083A4 (en) 2024-06-26
TWI901457B (zh) 2025-10-11
CO2022013685A2 (es) 2022-10-11
CL2024001471A1 (es) 2024-09-27
TW202200162A (zh) 2022-01-01
JP2026001070A (ja) 2026-01-06
TWI868323B (zh) 2025-01-01
JPWO2021172498A1 (https=) 2021-09-02

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