NZ747685A - Oligonucleotide analogues targeting human lmna - Google Patents

Oligonucleotide analogues targeting human lmna

Info

Publication number
NZ747685A
NZ747685A NZ747685A NZ74768517A NZ747685A NZ 747685 A NZ747685 A NZ 747685A NZ 747685 A NZ747685 A NZ 747685A NZ 74768517 A NZ74768517 A NZ 74768517A NZ 747685 A NZ747685 A NZ 747685A
Authority
NZ
New Zealand
Prior art keywords
lmna
oligonucleotides
targeting human
oligonucleotide analogues
human lmna
Prior art date
Application number
NZ747685A
Other languages
English (en)
Inventor
Michael R Erdos
Kan Cao
Ryszard Kole
Richard Keith Bestwick
Francis S Collins
Leslie B Gordon
Original Assignee
Sarepta Therapeutics Inc
Us Health
Univ Maryland
The Progeria Res Foundation
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, Us Health, Univ Maryland, The Progeria Res Foundation filed Critical Sarepta Therapeutics Inc
Publication of NZ747685A publication Critical patent/NZ747685A/en

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
    • 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
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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)
NZ747685A 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna NZ747685A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662330027P 2016-04-29 2016-04-29
PCT/US2017/030174 WO2017190041A1 (en) 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna

Publications (1)

Publication Number Publication Date
NZ747685A true NZ747685A (en) 2023-05-26

Family

ID=59285320

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ747685A NZ747685A (en) 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna

Country Status (14)

Country Link
US (2) US10822608B2 (enExample)
EP (1) EP3449000A1 (enExample)
JP (2) JP7042255B2 (enExample)
KR (1) KR102329187B1 (enExample)
CN (1) CN109477109B (enExample)
AU (2) AU2017258642B2 (enExample)
CA (1) CA3022303A1 (enExample)
CO (1) CO2018012873A2 (enExample)
EA (1) EA201892467A1 (enExample)
MA (1) MA45616A (enExample)
MX (1) MX2018013157A (enExample)
NZ (1) NZ747685A (enExample)
SG (1) SG11201809468XA (enExample)
WO (1) WO2017190041A1 (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
CA3002827A1 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
SG11201809468XA (en) * 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
JP7201153B2 (ja) 2016-08-09 2023-01-10 プレジデント アンド フェローズ オブ ハーバード カレッジ プログラム可能ｃａｓ９－リコンビナーゼ融合タンパク質およびその使用
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
MA65895B1 (fr) * 2016-12-19 2024-06-28 Sarepta Therapeutics, Inc. Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
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
US20240173430A1 (en) * 2018-09-05 2024-05-30 The Broad Institute, Inc. Base editing for treating hutchinson-gilford progeria syndrome
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
EP4146804A1 (en) 2020-05-08 2023-03-15 The Broad Institute Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022098014A1 (ko) * 2020-11-03 2022-05-12 한국생명공학연구원 유전자 교정을 이용한 조로증 및 자연노화의 예방 또는 치료용 조성물
EP4612292A1 (en) * 2022-11-02 2025-09-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate
CN119101680A (zh) * 2023-06-09 2024-12-10 良渚实验室 多核苷酸、药物组合物及其用途

Family Cites Families (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
JP3022967B2 (ja) 1985-03-15 2000-03-21 アンチバイラルズ インコーポレイテッド 立体規則性ポリヌクレオチド結合ポリマー
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
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
JPH07501204A (ja) 1991-06-28 1995-02-09 マサチューセッツ インスティテュート オブ テクノロジー 局所的オリゴヌクレオチド療法
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
CA2372085C (en) 1999-05-04 2009-10-27 Exiqon A/S L-ribo-lna analogues
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
CA2394758A1 (en) 1999-12-29 2001-07-05 A. James Mixson Histidine-containing copolymers enhance pharmaceutical agent delivery
JP2005514005A (ja) 2001-09-04 2005-05-19 エクシコン エ／エス 新規のｌｎａ組成物およびその使用
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003287464A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. 2'-fluoro substituted oligomeric compounds and compositions for use in gene modulations
CA2523672C (en) 2003-04-29 2012-07-17 Avi Biopharma, Inc. Compositions for enhancing transport of molecules into cells
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
WO2007047913A2 (en) * 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
MX2009002859A (es) * 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
EP2170363B1 (en) 2007-06-29 2018-08-08 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
CA2693742A1 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
WO2009064471A1 (en) 2007-11-15 2009-05-22 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
WO2010064146A2 (en) 2008-12-02 2010-06-10 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
EP2417257B1 (en) 2009-04-10 2016-03-09 Association Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2011034072A1 (ja) 2009-09-16 2011-03-24 株式会社キラルジェン Rna及びその誘導体合成のための新規保護基
CA3090304A1 (en) * 2010-05-13 2011-11-17 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
HUE030292T2 (en) 2010-09-30 2017-04-28 Nippon Shinyaku Co Ltd Morpholino-nucleic acid derivatives
JP5653160B2 (ja) 2010-10-15 2015-01-14 ダンロップスポーツ株式会社 ゴルフボール
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102339196B1 (ko) * 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9302014B2 (en) 2011-08-30 2016-04-05 Medical Research Council Cell-penetrating peptides having a central hydrophobic domain
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
HK1201514A1 (en) 2011-11-18 2015-09-04 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US9326992B2 (en) * 2011-12-08 2016-05-03 Sarepta Therapeutics, Inc. Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
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 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
JP6453212B2 (ja) 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
JP6774333B2 (ja) * 2013-04-16 2020-10-21 メモリアル スローン ケタリング キャンサー センター 年齢改変細胞および年齢改変細胞を作製するための方法
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
KR20230152178A (ko) 2014-01-16 2023-11-02 웨이브 라이프 사이언시스 리미티드 키랄 디자인
EP3620178A3 (en) * 2014-05-16 2020-07-22 Oregon State University Antisense antibacterial compounds and methods
US10391098B2 (en) * 2014-05-19 2019-08-27 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
WO2015179743A1 (en) * 2014-05-23 2015-11-26 Genzyme Corporation Library and screening approach for improved cell penetrating peptides
SG11201809468XA (en) * 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna

Also Published As

Publication number Publication date
JP7042255B2 (ja) 2022-03-25
JP2019514426A (ja) 2019-06-06
JP2022046725A (ja) 2022-03-23
AU2017258642B2 (en) 2023-08-31
US20190127735A1 (en) 2019-05-02
EA201892467A1 (ru) 2019-05-31
BR112018072279A2 (pt) 2019-02-12
CN109477109A (zh) 2019-03-15
CN109477109B (zh) 2022-09-23
MX2018013157A (es) 2019-05-23
MA45616A (fr) 2019-03-06
CA3022303A1 (en) 2017-11-02
SG11201809468XA (en) 2018-11-29
AU2023274161A1 (en) 2023-12-21
AU2017258642A1 (en) 2018-11-15
CO2018012873A2 (es) 2020-06-19
US20210010001A1 (en) 2021-01-14
KR102329187B1 (ko) 2021-11-22
AU2017258642A8 (en) 2018-11-29
US11802283B2 (en) 2023-10-31
KR20190025821A (ko) 2019-03-12
EP3449000A1 (en) 2019-03-06
WO2017190041A1 (en) 2017-11-02
US10822608B2 (en) 2020-11-03

Similar Documents

Publication Publication Date Title
NZ747685A (en) Oligonucleotide analogues targeting human lmna
MX2025007819A (es) Composiciones para usarse en el tratamiento de un sujeto que tiene hemofilia
NZ733882A (en) Compositions for modulating c9orf72 expression
EP4494706A3 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
ZA201802966B (en) Peptides and peptidomimetics in combination with t cell activating and/or checkpoint inhibiting agents for cancer treatment
MX2024001378A (es) Oligonucleotidos multi-conjugados definidos.
PH12017550069A1 (en) Tau antisense oligomers and uses thereof
MX391815B (es) AGENTES DE iARN, COMPOSICIONES Y METODOS DE LOS MISMOS PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES ASOCIADAS CON TRANSTITERINA (TTR).
MX382225B (es) Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).
MX358603B (es) Tratamiento de enfermedades relacionadas con un gen supresor de tumor mediante inhibicion del transcrito antisentido natural para el gen.
MX376916B (es) Composiciones para el tratamiento de espasmos gastrointestinales.
MX2021006326A (es) Inhibidores de pcna.
MY185390A (en) Antisense nucleic acids
EP4285912A3 (en) Compositions and methods for modulating ataxin 3 expression
EA201792103A1 (ru) Композиции и способы для ингибирования экспрессии генов hif2альфа
EP4585228A3 (en) Dosing regimens and related compositions and methods
MX381946B (es) Restos oligonucleotidicos multiples sobre soporte peptidico.
MX2021005980A (es) Adenovirus y metodos para usar adenovirus.
EP4349363A3 (en) Dosing regimens
MX2022012146A (es) Combinaciones para el tratamiento de neoplasias utilizando localizacion de celulas quiescentes e inhibidores de la mitosis.
BR112019023948A2 (pt) tratamento de cistos epiteliais por injeção intracística de partículas antineoplásicas
MX392348B (es) Anticuerpo anti cd43 y uso del mismo para tratamiento del cáncer.
EA201792406A1 (ru) Антисмысловые олигонуклеотиды для лечения дистрофического буллезного эпидермолиза
WO2019022586A3 (ko) 간암의 예방 또는 치료용 약학적 조성물
WO2016107933A3 (en) Materials and methods for the treatment of cancers

Legal Events

Date Code Title Description
PSEA Patent sealed
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 28 APR 2025 BY DENNEMEYER AND CO. SARL

Effective date: 20240415

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 28 APR 2026 BY DENNEMEYER + CO. SARL

Effective date: 20250422