SG10201700751XA - Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy - Google Patents

Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Info

Publication number
SG10201700751XA
SG10201700751XA SG10201700751XA SG10201700751XA SG10201700751XA SG 10201700751X A SG10201700751X A SG 10201700751XA SG 10201700751X A SG10201700751X A SG 10201700751XA SG 10201700751X A SG10201700751X A SG 10201700751XA SG 10201700751X A SG10201700751X A SG 10201700751XA
Authority
SG
Singapore
Prior art keywords
peptide
treatment
antisense oligonucleotides
myotonic dystrophy
morpholino antisense
Prior art date
Application number
SG10201700751XA
Other languages
English (en)
Inventor
Andrew Leger
Bruce Wentworth
Carol A Nelson
Timothy E Weeden
Nicholas Clayton
Seng Cheng
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of SG10201700751XA publication Critical patent/SG10201700751XA/en

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • 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
    • C12N2310/30Chemical 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • 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
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
SG10201700751XA 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy SG10201700751XA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201261705335P 2012-09-25 2012-09-25

Publications (1)

Publication Number Publication Date
SG10201700751XA true SG10201700751XA (en) 2017-03-30

Family

ID=49301675

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201700751XA SG10201700751XA (en) 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
SG11201501544PA SG11201501544PA (en) 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201501544PA SG11201501544PA (en) 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Country Status (15)

Country Link
US (1) US10111962B2 (ja)
EP (1) EP2900821B1 (ja)
JP (3) JP6404219B2 (ja)
KR (1) KR102240217B1 (ja)
CN (1) CN104837997A (ja)
AR (1) AR092658A1 (ja)
AU (2) AU2013323820B2 (ja)
BR (1) BR112015006049A2 (ja)
CA (1) CA2886017C (ja)
IL (1) IL237457B (ja)
MX (1) MX369519B (ja)
RU (2) RU2662962C2 (ja)
SG (2) SG10201700751XA (ja)
TW (1) TWI677350B (ja)
WO (1) WO2014052276A1 (ja)

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EP3044337B1 (en) * 2013-09-13 2020-03-18 Rutgers, the State University of New Jersey Multiplex diagnostic assay for lyme disease and other tick-borne diseases
WO2015179743A1 (en) * 2014-05-23 2015-11-26 Genzyme Corporation Library and screening approach for improved cell penetrating peptides
WO2015179742A1 (en) * 2014-05-23 2015-11-26 Genzyme Corporation Multiple oligonucleotide moieties on peptide carrier
KR20240035901A (ko) * 2015-05-19 2024-03-18 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
KR20180081600A (ko) * 2015-11-16 2018-07-16 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP2018538302A (ja) * 2015-12-15 2018-12-27 サレプタ セラピューティクス, インコーポレイテッド ペプチドオリゴヌクレオチド複合体
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
US11655470B2 (en) 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
US10450572B2 (en) * 2016-08-12 2019-10-22 Regents Of The University Of Minnesota Androgen receptor variant inhibitors and methods of using
WO2019079367A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. OLIGONUCLEOTIDE CONJUGATES-BICYCLIC PEPTIDES
JP7320500B2 (ja) * 2017-10-17 2023-08-03 サレプタ セラピューティクス, インコーポレイテッド アンチセンス送達のための細胞透過性ペプチド
US20210260206A1 (en) * 2018-03-16 2021-08-26 Sarepta Therapeutics, Inc. Chimeric peptides for antisense deliver
EP3790890A4 (en) 2018-05-09 2022-03-02 Ohio State Innovation Foundation CYCLIC PEPTIDES OF CELL PENETRATION WITH ONE OR MORE HYDROPHOBIC RESIDUES
CA3108287A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
BR112021023899A2 (pt) * 2019-05-28 2022-01-18 Astellas Pharma Inc Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
WO2021150867A1 (en) * 2020-01-24 2021-07-29 Sarepta Therapeutics, Inc. Designing antisense oligonucleotide delivery peptides by interpretable machine learning
JP7461687B2 (ja) 2021-04-30 2024-04-04 エディットフォース株式会社 筋強直性ジストロフィー1型治療薬
KR20240038967A (ko) * 2021-06-23 2024-03-26 엔트라다 테라퓨틱스, 인크. Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법
WO2024063570A1 (ko) * 2022-09-23 2024-03-28 주식회사 셀리버리 근긴장성 이영양증 단백질 키나아제를 표적화하는 세포투과성 펩티드 융합 안티센스 올리고뉴클레오티드 및 이의 용도

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Also Published As

Publication number Publication date
KR20150060738A (ko) 2015-06-03
WO2014052276A1 (en) 2014-04-03
JP2019006808A (ja) 2019-01-17
MX2015003841A (es) 2015-07-17
CN104837997A (zh) 2015-08-12
JP2020196751A (ja) 2020-12-10
AR092658A1 (es) 2015-04-29
IL237457A0 (en) 2015-04-30
AU2013323820A1 (en) 2015-04-16
EP2900821B1 (en) 2020-04-01
KR102240217B1 (ko) 2021-04-14
RU2018123569A (ru) 2019-03-07
AU2019203929A1 (en) 2019-07-11
RU2662962C2 (ru) 2018-07-31
JP2015532264A (ja) 2015-11-09
TWI677350B (zh) 2019-11-21
IL237457B (en) 2022-02-01
CA2886017A1 (en) 2014-04-03
EP2900821A1 (en) 2015-08-05
CA2886017C (en) 2022-04-05
BR112015006049A2 (pt) 2017-08-22
JP6404219B2 (ja) 2018-10-10
AU2013323820B2 (en) 2019-06-27
MX369519B (es) 2019-11-11
US20150238627A1 (en) 2015-08-27
RU2015115721A (ru) 2016-11-20
RU2018123569A3 (ja) 2021-11-09
SG11201501544PA (en) 2015-03-30
US10111962B2 (en) 2018-10-30
TW201424752A (zh) 2014-07-01

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