CA2691673A1 - Tissue specific peptide conjugates and methods - Google Patents

Tissue specific peptide conjugates and methods Download PDF

Info

Publication number
CA2691673A1
CA2691673A1 CA002691673A CA2691673A CA2691673A1 CA 2691673 A1 CA2691673 A1 CA 2691673A1 CA 002691673 A CA002691673 A CA 002691673A CA 2691673 A CA2691673 A CA 2691673A CA 2691673 A1 CA2691673 A1 CA 2691673A1
Authority
CA
Canada
Prior art keywords
peptide
cell
conjugate
seq
oligomer
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
CA002691673A
Other languages
English (en)
French (fr)
Inventor
Hong M. Moulton
Patrick L. Iversen
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.)
Sarepta Therapeutics Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2691673A1 publication Critical patent/CA2691673A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/08Drugs for disorders of the urinary system of the prostate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/16Antivirals for RNA viruses for influenza or rhinoviruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/40Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Rheumatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Vascular Medicine (AREA)
CA002691673A 2007-06-29 2008-06-30 Tissue specific peptide conjugates and methods Pending CA2691673A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US93772507P 2007-06-29 2007-06-29
US60/937,725 2007-06-29
PCT/US2008/008168 WO2009005793A2 (en) 2007-06-29 2008-06-30 Tissue specific peptide conjugates and methods

Publications (1)

Publication Number Publication Date
CA2691673A1 true CA2691673A1 (en) 2009-01-08

Family

ID=40226726

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002691673A Pending CA2691673A1 (en) 2007-06-29 2008-06-30 Tissue specific peptide conjugates and methods

Country Status (7)

Country Link
US (1) US20090099066A1 (cg-RX-API-DMAC7.html)
EP (2) EP3443976A1 (cg-RX-API-DMAC7.html)
JP (3) JP5864100B2 (cg-RX-API-DMAC7.html)
AU (1) AU2008271050B2 (cg-RX-API-DMAC7.html)
CA (1) CA2691673A1 (cg-RX-API-DMAC7.html)
ES (1) ES2694726T3 (cg-RX-API-DMAC7.html)
WO (1) WO2009005793A2 (cg-RX-API-DMAC7.html)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11801311B2 (en) * 2014-05-23 2023-10-31 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003225410A1 (en) * 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
DE602004028930D1 (de) * 2003-04-29 2010-10-14 Avi Biopharma Inc Zusammensetzungen zur verbesserung der antisense-wirksamkeit und des transports von nukleinsäureanalog in zellen
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
AU2005208710A1 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
EP2206781B1 (en) 2004-06-28 2015-12-02 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
EP1855694B1 (en) * 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8168181B2 (en) * 2006-02-13 2012-05-01 Alethia Biotherapeutics, Inc. Methods of impairing osteoclast differentiation using antibodies that bind siglec-15
EP2438962A3 (en) 2006-02-13 2012-07-18 Alethia Biotherapeutics Inc. Polynucleotides and polypeptide sequences involved in the process of bone remodeling
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
EP2235034B1 (en) * 2007-12-28 2016-11-23 Sarepta Therapeutics, Inc. Immunomodulatory agents and methods of use
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
US8231895B2 (en) * 2008-05-22 2012-07-31 Universidade De Coimbra Targeted delivery to human diseases and disorders
US20110130346A1 (en) * 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
EP2283029A1 (en) 2008-06-04 2011-02-16 Medical Research Council Peptides
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
EP2376633A1 (en) * 2008-12-17 2011-10-19 AVI BioPharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
WO2010123369A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
PL2499249T3 (pl) 2009-11-12 2019-03-29 Univ Western Australia Cząsteczki antysensowne i sposoby leczenia patologii
CN102712928B (zh) 2009-11-13 2017-08-04 萨雷普塔治疗公司 反义抗病毒化合物及治疗流感病毒感染的方法
JP5677454B2 (ja) * 2009-12-11 2015-02-25 グワンジュ・インスティテュート・オブ・サイエンス・アンド・テクノロジー 細胞内ターゲット結合用二座ペプチドバインダー
WO2011127210A1 (en) * 2010-04-06 2011-10-13 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
KR101900770B1 (ko) * 2010-07-19 2018-09-20 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
CN103619356B (zh) * 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
EP2751128B1 (en) 2011-08-30 2021-03-03 United Kingdom Research and Innovation Cell-penetrating peptides having a central hydrophobic domain
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2535654T3 (es) * 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
US9066967B2 (en) 2011-12-08 2015-06-30 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human LMNA
CN118581086A (zh) * 2012-01-27 2024-09-03 比奥马林技术公司 用于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
CA2873779A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating mecp2 expression
KR20150030205A (ko) * 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
CN104583401A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节atp2a2表达的组合物和方法
EP2850185A4 (en) * 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING UTRN GENE EXPRESSION
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
KR101329411B1 (ko) 2012-05-31 2013-11-14 주식회사 엘지생활건강 피부투과성 펩타이드
EP2875051B1 (en) 2012-07-19 2019-02-20 Daiichi Sankyo Company, Limited Anti-siglec-15 antibodies
CN116271053A (zh) 2012-08-13 2023-06-23 洛克菲勒大学 治疗和诊断黑素瘤
JP6404219B2 (ja) 2012-09-25 2018-10-10 ジェンザイム・コーポレーション 筋強直性ジストロフィーを治療するためのペプチド結合モルホリノアンチセンスオリゴヌクレオチド
ES2724853T3 (es) 2012-11-15 2019-09-16 Roche Innovation Ct Copenhagen As Conjugados de oligonucleótidos
CN103981147B (zh) 2013-02-08 2017-11-10 中国科学院上海生命科学研究院 一种新的制备肝实质细胞的方法
BR122020016865B1 (pt) 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
EP3065783A4 (en) 2013-11-06 2017-06-21 Merck Sharp & Dohme Corp. Dual molecular delivery of oligonucleotides and peptide containing conjugates
JP6901966B2 (ja) 2014-05-16 2021-07-14 オレゴン ステート ユニバーシティ アンチセンス抗菌化合物および方法
JP6687542B2 (ja) 2014-05-19 2020-04-22 オレゴン ステート ユニバーシティ アンチセンス抗菌化合物および方法
CA2949104C (en) 2014-05-23 2023-09-26 Genzyme Corporation Multiple oligonucleotide moieties on peptide carrier
HK1246303A1 (zh) * 2014-12-31 2018-09-07 Oregon State University 反义抗菌化合物和方法
MA41795A (fr) * 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
BR112018007066A2 (pt) * 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
EP3394261A4 (en) 2015-12-23 2019-11-27 Oregon State University ANTIBACTERIAL ANTISENSE COMPOUNDS AND METHOD
EP3394262B1 (en) * 2015-12-23 2024-08-28 Oregon State University Antisense antibacterial compounds and methods
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
KR102329187B1 (ko) 2016-04-29 2021-11-22 사렙타 쎄러퓨틱스, 인코퍼레이티드 인간 lmna를 표적화하는 올리고뉴클레오타이드 유사체
MY205600A (en) 2016-12-19 2024-10-29 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
US10800817B2 (en) 2016-12-19 2020-10-13 Morehouse School Of Medicine Compositions and methods for treating diseases by inhibiting exosome release
PL3554552T3 (pl) 2016-12-19 2022-11-21 Sarepta Therapeutics, Inc. Koniugaty oligomerów do pomijania egzonów dla dystrofii mięśniowej
SMT202200366T1 (it) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
SG11201906200WA (en) 2017-01-06 2019-08-27 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US20180207293A1 (en) * 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
EP3687547A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3697422A4 (en) 2017-10-17 2021-08-11 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENS ADMINISTRATION
EP3713575A4 (en) 2017-11-21 2021-08-25 Rgenix, Inc. POLYMORPHS AND THEIR USES
JP7013626B2 (ja) * 2018-01-05 2022-02-01 国立医薬品食品衛生研究所長 細胞膜透過ペプチド、構築物、及び、カーゴ分子を細胞内に輸送する方法
US11987647B2 (en) 2018-05-09 2024-05-21 Ohio State Innovation Foundation Cyclic cell-penetrating peptides with one or more hydrophobic residues
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AU2019314538B2 (en) * 2018-08-02 2025-03-13 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
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
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
BR112021010982A2 (pt) 2018-12-07 2021-08-31 Oxford University Innovation Limited Ligantes
IL318474A (en) 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
KR102270700B1 (ko) * 2019-11-01 2021-06-30 한국과학기술연구원 폐 섬유증의 예방 또는 치료를 위한 올리고뉴클레오티드 중합체와 생체 적합성 양이온성 펩타이드로 이루어지는 폐 특이적 약물 전달체 및 이의 이용
EP4073025B1 (en) 2019-12-13 2024-03-27 Inspirna, Inc. Metal salts and uses thereof
CA3165961A1 (en) 2019-12-26 2021-07-01 Nippon Shinyaku Co., Ltd. Antisense nucleic acid that induces skipping of exon 50
CA3173049A1 (en) 2020-02-28 2021-09-02 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
JP2024501800A (ja) 2020-12-23 2024-01-16 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを治療するためのエクソンスキッピングオリゴヌクレオチドコンジュゲートを含む組成物
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
CA3224782A1 (en) 2021-06-23 2022-12-29 Nippon Shinyaku Co., Ltd. Combination of antisense oligomers
EP4368186A4 (en) 2021-07-08 2025-06-25 Nippon Shinyaku Co., Ltd. NEPHROTOXICITY REDUCING AGENT
IL310001A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Precipitation suppressing agent
US20240285547A1 (en) 2021-07-08 2024-08-29 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2023034515A2 (en) * 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
CN119183457A (zh) 2022-04-15 2024-12-24 达因疗法公司 用于治疗强直性肌营养不良的肌肉靶向复合物和制剂
WO2023205451A1 (en) 2022-04-22 2023-10-26 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
WO2024091824A1 (en) 2022-10-26 2024-05-02 Ada Forsyth Institute, Inc. Differentiation and reprogramming of chondrocyte
WO2024097822A1 (en) 2022-11-02 2024-05-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate
EP4634373A1 (en) 2022-12-16 2025-10-22 University of Rochester Repairment of barrier dysfunction in esophagus
WO2025085868A2 (en) 2023-10-20 2025-04-24 Radiation Control Technologies, Inc. Cd47-targeting morpholinos

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US946496A (en) 1909-08-21 1910-01-11 Henry J Cox Fulcrum-block.
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
AU5698186A (en) 1985-03-15 1986-10-13 Summerton, J. Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
US5525465A (en) * 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
US5674980A (en) * 1989-12-21 1997-10-07 Biogen Inc Fusion protein comprising tat-derived transport moiety
ATE208634T1 (de) * 1993-01-07 2001-11-15 Univ Jefferson Modulation der proliferation von glatten muskelzellen durch antisense inhibition von c-myc
US6686338B1 (en) 1996-02-23 2004-02-03 The Board Of Regents Of The University Of Nebraska Enzyme inhibitors for metabolic redirection
US5849727A (en) * 1996-06-28 1998-12-15 Board Of Regents Of The University Of Nebraska Compositions and methods for altering the biodistribution of biological agents
AU734827B2 (en) * 1997-05-21 2001-06-21 Board Of Trustees Of The Leland Stanford Junior University Composition and method for enhancing transport across biological membranes
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
AUPP398498A0 (en) 1998-06-09 1998-07-02 Silverbrook Research Pty Ltd A method of manufacture of an image creation apparatus (ijm44)
WO2000002588A1 (en) 1998-07-13 2000-01-20 The Board Of Regents Of The University Of Nebraska Targeted site specific drug delivery compositions and method of use
JP2002535015A (ja) * 1999-01-29 2002-10-22 エイブイアイ バイオファーマ, インコーポレイテッド 標的rnaを検出するための非侵襲性方法
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
WO2000061810A1 (en) * 1999-04-08 2000-10-19 Oasis Biosciences, Inc. Antisense oligonucleotides comprising universal and/or degenerate bases
US6303573B1 (en) * 1999-06-07 2001-10-16 The Burnham Institute Heart homing peptides and methods of using same
US6669951B2 (en) * 1999-08-24 2003-12-30 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
JP2004523465A (ja) 2000-05-17 2004-08-05 エイブイアイ バイオファーマ, インコーポレイテッド 薬物の薬物動態を改善するためのアンチセンス酵素インヒビター
US20040170955A1 (en) * 2000-09-08 2004-09-02 Wadih Arap Human and mouse targeting peptides identified by phage display
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
WO2002063280A1 (en) * 2001-02-06 2002-08-15 Auburn University Ligand sensor devices and uses thereof
WO2002065986A2 (en) * 2001-02-16 2002-08-29 Cellgate, Inc. Transporters comprising spaced arginine moieties
NZ530101A (en) * 2001-05-17 2007-01-26 Avi Biopharma Inc Methods for treating cancer by administering an oligomer antisense to c-myc together with a cancer chemotherapeutic agent
JP3735292B2 (ja) * 2001-07-26 2006-01-18 三菱重工業株式会社 ダイエット効果のある健康食品および製剤
US6645974B2 (en) * 2001-07-31 2003-11-11 Merck & Co., Inc. Androgen receptor modulators and methods for use thereof
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
JP2005538035A (ja) * 2001-12-11 2005-12-15 ザ ボード オブ トラスティーズ オブ ザ リーランド スタンフォード ジュニア ユニバーシティ グアニジニウム輸送試薬および結合体
US20030207907A1 (en) 2002-05-03 2003-11-06 Iversen Patrick L. Delivery of microparticle-conjugated drugs for inhibition of stenosis
DE602004028930D1 (de) * 2003-04-29 2010-10-14 Avi Biopharma Inc Zusammensetzungen zur verbesserung der antisense-wirksamkeit und des transports von nukleinsäureanalog in zellen
US20050203041A1 (en) 2003-09-23 2005-09-15 Mourich Dan V. Antisense compound and method for selectively killing activated T cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US7786151B2 (en) * 2004-01-09 2010-08-31 Kinopharma, Inc. Therapeutic composition of treating abnormal splicing caused by the excessive kinase induction
AU2005208710A1 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20060078542A1 (en) * 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
US20060014712A1 (en) * 2004-05-30 2006-01-19 Cemines, Inc. Controlled delivery of therapeutic compounds
EP2206781B1 (en) 2004-06-28 2015-12-02 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7489981B2 (en) * 2004-09-01 2009-02-10 Honda Motor Co., Ltd. Assembly line control system and immobilizer data protocol and communication process flow
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
NZ538097A (en) * 2005-02-07 2006-07-28 Ovita Ltd Method and compositions for improving wound healing
EP1855694B1 (en) * 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
US7790694B2 (en) * 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
CA2621964A1 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
WO2007056466A2 (en) * 2005-11-08 2007-05-18 Avi Biopharma, Inc. Immunosuppression compound and treatment method
US8501704B2 (en) * 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
WO2007103529A2 (en) * 2006-03-07 2007-09-13 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
EP2024499B1 (en) 2006-05-10 2017-10-25 Sarepta Therapeutics, Inc. Oligonucleotide analogs having cationic intersubunit linkages
EP2235034B1 (en) * 2007-12-28 2016-11-23 Sarepta Therapeutics, Inc. Immunomodulatory agents and methods of use
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
EP2376633A1 (en) * 2008-12-17 2011-10-19 AVI BioPharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11801311B2 (en) * 2014-05-23 2023-10-31 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

Also Published As

Publication number Publication date
EP2170363A4 (en) 2010-12-29
EP2170363B1 (en) 2018-08-08
AU2008271050B2 (en) 2014-11-06
US20090099066A1 (en) 2009-04-16
EP3443976A1 (en) 2019-02-20
JP2016047050A (ja) 2016-04-07
WO2009005793A3 (en) 2009-05-28
EP2170363A2 (en) 2010-04-07
JP2010532168A (ja) 2010-10-07
JP5864100B2 (ja) 2016-02-17
AU2008271050A1 (en) 2009-01-08
WO2009005793A2 (en) 2009-01-08
JP2018171066A (ja) 2018-11-08
JP6584888B2 (ja) 2019-10-02
ES2694726T3 (es) 2018-12-26

Similar Documents

Publication Publication Date Title
AU2008271050B2 (en) Tissue specific peptide conjugates and methods
US11236329B2 (en) Compound and method for treating myotonic dystrophy
US10927378B2 (en) Compound and method for treating myotonic dystrophy
Lebleu et al. Cell penetrating peptide conjugates of steric block oligonucleotides
US8877725B2 (en) Peptide conjugated, inosine-substituted antisense oligomer compound and method
US20140051646A1 (en) Cell penetrating peptides
AU2008273094A1 (en) Molecules for targeting compounds to various selected organs, tissues or tumor cells
TW202346591A (zh) 連結有載體胜肽的核酸
JP2020510612A (ja) 多様な選択された臓器又は組織をターゲティングするための物質
Soudah et al. AntimiR-155 cyclic peptide–PNA conjugate: synthesis, cellular uptake, and biological activity
Soudah et al. CLIP6-PNA-peptide conjugates: non-endosomal delivery of splice switching oligonucleotides
AU2017202166B2 (en) Tissue specific peptide conjugates and methods
HK40004164A (en) Tissue specific peptide conjugates and methods
Class et al. Patent application title: COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY Inventors: Hong M. Moulton (Corvallis, OR, US) Ryszard Kole (Corvallis, OR, US) Ryszard Kole (Corvallis, OR, US) Assignees: AVI BIOPHARMA, INC.
Haque et al. DG9, a Versatile Cell-Penetrating Peptide to Enhance Delivery of Antisense Oligonucleotide-Based Therapeutics
Turner et al. 18 Peptide Conjugates of Oligonucleotide Analogs and siRNA for Gene Expression Modulation
HK1101314B (en) Peptide conjugated, inosine-substituted antisense oligomer compound and method

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425

EEER Examination request

Effective date: 20130425