MX369519B - Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. - Google Patents

Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.

Info

Publication number
MX369519B
MX369519B MX2015003841A MX2015003841A MX369519B MX 369519 B MX369519 B MX 369519B MX 2015003841 A MX2015003841 A MX 2015003841A MX 2015003841 A MX2015003841 A MX 2015003841A MX 369519 B MX369519 B MX 369519B
Authority
MX
Mexico
Prior art keywords
peptide
treatment
antisense oligonucleotides
linked morpholino
myotonic dystrophy
Prior art date
Application number
MX2015003841A
Other languages
English (en)
Other versions
MX2015003841A (es
Inventor
Cheng Seng
E Weeden Timothy
Leger Andrew
Wentworth Bruce
A Nelson Carol
Clayton Nicholas
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 MX2015003841A publication Critical patent/MX2015003841A/es
Publication of MX369519B publication Critical patent/MX369519B/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/62—Medicinal 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
    • A61K47/64—Drug-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C12N2320/32—Special 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (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)

Abstract

La presente invención se refiere a oligonucleótidos antisentido morfolino unidos a péptido (PPMO) que están dirigidos a la extensión de repetición poli CUG en la región no traducida 3' del gen que codifica la proteína quinasa de la distrofia miotónica (DMPK) y métodos para la administración sistémica del mismo para el tratamiento de la distrofia miotónica tipo 1 (DM1).
MX2015003841A 2012-09-25 2013-09-24 Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica. MX369519B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261705335P 2012-09-25 2012-09-25
PCT/US2013/061320 WO2014052276A1 (en) 2012-09-25 2013-09-24 Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy

Publications (2)

Publication Number Publication Date
MX2015003841A MX2015003841A (es) 2015-07-17
MX369519B true MX369519B (es) 2019-11-11

Family

ID=49301675

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015003841A MX369519B (es) 2012-09-25 2013-09-24 Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.

Country Status (15)

Country Link
US (1) US10111962B2 (es)
EP (1) EP2900821B1 (es)
JP (3) JP6404219B2 (es)
KR (1) KR102240217B1 (es)
CN (1) CN104837997A (es)
AR (1) AR092658A1 (es)
AU (2) AU2013323820B2 (es)
BR (1) BR112015006049A2 (es)
CA (1) CA2886017C (es)
IL (1) IL237457B (es)
MX (1) MX369519B (es)
RU (2) RU2018123569A (es)
SG (2) SG11201501544PA (es)
TW (1) TWI677350B (es)
WO (1) WO2014052276A1 (es)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
RU2739987C2 (ru) * 2014-05-23 2020-12-30 Джензим Корпорейшн Множественные олигонуклеотидные фрагменты на пептидном носителе
JP6954843B2 (ja) * 2015-05-19 2021-10-27 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コンジュゲート
EP3377042A4 (en) * 2015-11-16 2019-05-29 The Research Institute at Nationwide Children's Hospital SUBSTANCES AND METHODS FOR THE TREATMENT OF MYOPATHIES BASED ON TITINE AND OTHER TITINOPATHIES
MX2018007307A (es) * 2015-12-15 2019-03-14 Sarepta Therapeutics Inc Conjugados de peptidos y oligonucleotidos.
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
WO2018009547A1 (en) 2016-07-05 2018-01-11 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
MA50834A (fr) * 2017-10-17 2020-08-26 Massachusetts Inst Technology Peptides de pénétration cellulaire pour administration antisens
EP3700537A4 (en) * 2017-10-17 2021-08-04 Sarepta Therapeutics, Inc. OLIGONUCLEOTIDES-BICYCLIC PEPTIDES CONJUGATES
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド
US11987647B2 (en) 2018-05-09 2024-05-21 Ohio State Innovation Foundation Cyclic cell-penetrating peptides with one or more hydrophobic residues
JP2021533199A (ja) * 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. 筋強直症ジストロフィーを処置するための筋標的化複合体およびそれらの使用
MX2021014478A (es) * 2019-05-28 2022-01-06 Astellas Pharma Inc Metodo para tratar distrofia muscular por direccionamiento del gen dmpk.
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
EP4093441A1 (en) * 2020-01-24 2022-11-30 Sarepta Therapeutics, Inc. Designing antisense oligonucleotide delivery peptides by interpretable machine learning
IL308029A (en) 2021-04-30 2023-12-01 Editforce Inc Therapeutic drug for myotonic dystrophy type 1
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

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
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
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
JPH0796558B2 (ja) * 1988-03-31 1995-10-18 協和醗酵工業株式会社 修飾ポリペプチド
CA1340810C (en) 1988-03-31 1999-11-02 Motoo Yamasaki Polypeptide derivatives of human granulocyte colony stimulating factor
JP2745351B2 (ja) 1991-02-14 1998-04-28 富士写真フイルム株式会社 ペプチド誘導体及びその用途
US20090286693A1 (en) 2001-12-12 2009-11-19 New York University, Nyu Medical Center, Department Of Industrial Liaison Microarrays of tagged combinatorial triazine libraries
GB0415263D0 (en) * 2004-07-07 2004-08-11 Norwegian Radium Hospital Res Method
HUE036995T2 (hu) 2006-05-10 2018-08-28 Sarepta Therapeutics Inc Az alegységek között kationos kötéssel rendelkezõ oligonukleotid analógok
AU2008271050B2 (en) 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases

Also Published As

Publication number Publication date
BR112015006049A2 (pt) 2017-08-22
JP2015532264A (ja) 2015-11-09
TWI677350B (zh) 2019-11-21
KR20150060738A (ko) 2015-06-03
AU2019203929A1 (en) 2019-07-11
EP2900821B1 (en) 2020-04-01
TW201424752A (zh) 2014-07-01
US20150238627A1 (en) 2015-08-27
RU2662962C2 (ru) 2018-07-31
MX2015003841A (es) 2015-07-17
AR092658A1 (es) 2015-04-29
IL237457A0 (en) 2015-04-30
SG10201700751XA (en) 2017-03-30
RU2015115721A (ru) 2016-11-20
JP2019006808A (ja) 2019-01-17
CA2886017A1 (en) 2014-04-03
IL237457B (en) 2022-02-01
EP2900821A1 (en) 2015-08-05
AU2013323820A1 (en) 2015-04-16
WO2014052276A1 (en) 2014-04-03
JP2020196751A (ja) 2020-12-10
AU2013323820B2 (en) 2019-06-27
US10111962B2 (en) 2018-10-30
SG11201501544PA (en) 2015-03-30
CA2886017C (en) 2022-04-05
RU2018123569A3 (es) 2021-11-09
CN104837997A (zh) 2015-08-12
KR102240217B1 (ko) 2021-04-14
RU2018123569A (ru) 2019-03-07
JP6404219B2 (ja) 2018-10-10

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