SG11201808964PA - Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene - Google Patents

Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

Info

Publication number
SG11201808964PA
SG11201808964PA SG11201808964PA SG11201808964PA SG11201808964PA SG 11201808964P A SG11201808964P A SG 11201808964PA SG 11201808964P A SG11201808964P A SG 11201808964PA SG 11201808964P A SG11201808964P A SG 11201808964PA SG 11201808964P A SG11201808964P A SG 11201808964PA
Authority
SG
Singapore
Prior art keywords
international
nucleobase
pct
target region
additional
Prior art date
Application number
SG11201808964PA
Inventor
Frederick Schnell
Original Assignee
Sarepta Therapeutics Inc
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 filed Critical Sarepta Therapeutics Inc
Publication of SG11201808964PA publication Critical patent/SG11201808964PA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • 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
    • C12N2310/32—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/582—Recycling of unreacted starting or intermediate materials

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER (19) World Intellectual Property Organization International Bureau (43) International Publication Date .....0\"\" 26 October 2017 (26.10.2017) WIPO I PCT THE PATENT COOPERATION TREATY (PCT) 1111111111101110111111111111111111111 011101110111111111110111101111111111011111 (10) International Publication Number WO 2017/184529 Al — (51) (21) (22) (25) Filing Language: (26) (30) (71) (72) International Patent Classification: (74) A61K 48/00 (2006.01) C07H 21/02 (2006.01) International Application Number: PCT/US2017/028002 (81) International Filing Date: 17 April 2017 (17.04.2017) English Publication Language: English Priority Data: 62/324,185 18 April 2016 (18.04.2016) US Applicant: SAREPTA THERAPEUTICS, INC. [US/US]; 215 First Street, Cambridge, MA 02142 (US). (84) Inventor: SCHNELL, Frederick, Joseph; 1654 NW Crest PL, Corvallis, OR 97330 (US). 85004 (US). Agent: MULHOLLAND, William F.; Snell & Wilmer LLP, One Arizona Center, 400 E. Van Buren, Phoenix, AZ Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, = Title: ANTISENSE OLIGOMERS THE ACID ALPHA-GLUCOSIDASE GENE \"IT 0 as ' a ,2 .... 8 .> -5 15 = Ca c t 1, Ni cl. ' c 1 d c 0 ,0 ' 2 o 0 * A,C/ c..P„,: e,- 4,— Z 3C q l O .?. , s t.f.: cP : The present disclosure and include a targeting (GAA) gene. The least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional is internal to the target region. WITH c)1: 1-1 nucleobases ---- glucosidase 0 nucleobase slk* s? ' v`k ‘.. 11 / 4 °) c ,, ,, -- • ,..,, \ - A - 4 - ,..\.! i ? ? ' 4 C1 relates sequence complementary target region N FIG. AND dr v ? METHODS 111111 5 = CU , 43\ , (1 l5 1 \"ti 'ti 0 1 '\" 4 4‘ Aki AV , tk t v y t v ._ \" l r° ,f , ,..fo . to modified antisense includes at least one additional nucleobase compared to the targeting sequence, wherein the uM 's GP s, P.,.. _ uM OF USING THE SAME FOR TREATING DISEASES ASSOCIATED = (54) = = = = = _ _ = = 15 1 '15' 15\" cl, 15' 15 * , IP 1 \"ti \", \"ti 'ti \"ti - ti N '', -' na \" .b?' P P .t.\" slx P .0 >'` ,A % . 2/ ,„'\ \" cS 0 0 0 § 3 N..... NJ. v. S' NS\" N S ' '\" N S N sf 2 4 r ° -f , .10 4c , 4. c ° -.3 c -4 c ° 1. z ..N ii ..? 1 oligonucleotides. The nucleotides described herein are of 10 to 40 to a target region within intron 1 of a pre-mRNA of the human alpha _ = = 1-1 c:7 kin 7r (57) IN at ei o [Continued on next page] WO 2017/184529 Al MIDEDIM01101 DIDIRMEM3111111111111111111111111111011111111111111111 MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event amendments (Rule 48.2(h)) of receipt of
SG11201808964PA 2016-04-18 2017-04-17 Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene SG11201808964PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662324185P 2016-04-18 2016-04-18
PCT/US2017/028002 WO2017184529A1 (en) 2016-04-18 2017-04-17 Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

Publications (1)

Publication Number Publication Date
SG11201808964PA true SG11201808964PA (en) 2018-11-29

Family

ID=60117051

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201808964PA SG11201808964PA (en) 2016-04-18 2017-04-17 Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

Country Status (14)

Country Link
US (2) US11060089B2 (en)
EP (1) EP3445405A4 (en)
JP (3) JP7033547B2 (en)
KR (1) KR102522059B1 (en)
CN (1) CN109414511B (en)
AU (1) AU2017254106A1 (en)
BR (1) BR112018071477A2 (en)
CA (1) CA3021267A1 (en)
EA (1) EA201892366A1 (en)
MX (1) MX2018012695A (en)
SA (1) SA518400251B1 (en)
SG (1) SG11201808964PA (en)
TW (1) TWI766861B (en)
WO (1) WO2017184529A1 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3445405A4 (en) 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
JP7394753B2 (en) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド antisense oligomer compounds
CA3108289A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EA202190417A1 (en) * 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF PUMP'S DISEASE
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023055774A1 (en) * 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2024016003A2 (en) 2022-07-14 2024-01-18 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
WO2024073404A1 (en) * 2022-09-28 2024-04-04 Sarepta Therapeutics, Inc. Pompe disease mouse model generation, characterization and methods of use

Family Cites Families (113)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4426330A (en) 1981-07-20 1984-01-17 Lipid Specialties, Inc. Synthetic phospholipid compounds
US4534899A (en) 1981-07-20 1985-08-13 Lipid Specialties, Inc. Synthetic phospholipid compounds
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
DE3650699T2 (en) 1985-03-15 1999-04-15 Antivirals Inc Immunoassay for polynucleotide and methods
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group 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
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5227170A (en) 1989-06-22 1993-07-13 Vestar, Inc. Encapsulation process
US5527528A (en) 1989-10-20 1996-06-18 Sequus Pharmaceuticals, Inc. Solid-tumor treatment method
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5356633A (en) 1989-10-20 1994-10-18 Liposome Technology, Inc. Method of treatment of inflamed tissues
US5469854A (en) 1989-12-22 1995-11-28 Imarx Pharmaceutical Corp. Methods of preparing gas-filled liposomes
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
DE4021108A1 (en) 1990-07-03 1992-01-09 Bayer Ag OPTICALLY ACTIVE N- (ALPHA) -FLUORACRYLOYL-AMINO ACID DERIVATIVES, THEIR PRODUCTION, THE OPTICALLY ACTIVE POLYMERS MADE THEREOF AND THE USE THEREOF FOR THE CLEAVING OF RACEMATS
JP3220180B2 (en) 1991-05-23 2001-10-22 三菱化学株式会社 Drug-containing protein-bound liposomes
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
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
JPH07501204A (en) 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 Topical oligonucleotide therapy
NZ244306A (en) 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5521291A (en) 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
FR2692265B1 (en) 1992-05-25 1996-11-08 Centre Nat Rech Scient BIOLOGICALLY ACTIVE COMPOUNDS OF THE PHOSPHOTRIESTER TYPE.
US5583020A (en) 1992-11-24 1996-12-10 Ribozyme Pharmaceuticals, Inc. Permeability enhancers for negatively charged polynucleotides
JP3351476B2 (en) 1993-01-22 2002-11-25 三菱化学株式会社 Phospholipid derivatives and liposomes containing the same
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
US5462854A (en) 1993-04-19 1995-10-31 Beckman Instruments, Inc. Inverse linkage oligonucleotides for chemical and enzymatic processes
FR2705099B1 (en) 1993-05-12 1995-08-04 Centre Nat Rech Scient Phosphorothioate triester oligonucleotides and process for their preparation.
US5534259A (en) 1993-07-08 1996-07-09 Liposome Technology, Inc. Polymer compound and coated particle composition
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5417978A (en) 1993-07-29 1995-05-23 Board Of Regents, The University Of Texas System Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5591721A (en) 1994-10-25 1997-01-07 Hybridon, Inc. Method of down-regulating gene expression
US5512295A (en) 1994-11-10 1996-04-30 The Board Of Trustees Of The Leland Stanford Junior University Synthetic liposomes for enhanced uptake and delivery
AU717660B2 (en) 1995-12-18 2000-03-30 Angiodevice International Gmbh Crosslinked polymer compositions and methods for their use
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
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
ATE356824T1 (en) 1999-05-04 2007-04-15 Santaris Pharma As L-RIBO-LNA ANALOGUE
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
KR20020097241A (en) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 Splice-region antisense composition and method
KR100408844B1 (en) 2000-07-29 2003-12-06 한국산업기술평가원 Expression vector using for animal cell
US20040049022A1 (en) 2001-04-24 2004-03-11 Nyce Jonathan W. Composition & methods for treatment and screening
AU2002334307A1 (en) 2001-09-04 2003-03-18 Exiqon A/S Novel lna compositions and uses thereof
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
KR100464261B1 (en) 2002-01-24 2005-01-03 주식회사 파나진 A Novel Monomer For Synthesis of PNA Oligomer And A Process For Producing The Same
KR20030084444A (en) 2002-04-26 2003-11-01 주식회사 파나진 A Novel Monomer For Synthesis of PNA Oligomer And A Process For Producing The Same
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004043978A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
CN1922313B (en) 2004-02-10 2011-10-26 生物马林医药公司 Acid alpha-glucosidase and fragments thereof
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2397113T3 (en) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Compositions and procedures to modulate the splicing of SMN2
WO2007022470A2 (en) 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
HUE035799T2 (en) 2006-05-10 2018-05-28 Sarepta Therapeutics Inc Oligonucleotide analogs having cationic intersubunit linkages
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US20090099066A1 (en) 2007-06-29 2009-04-16 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
CN101790385A (en) 2007-07-12 2010-07-28 普罗森那技术公司 Molecules for targeting compounds to various selected organs, tissues or tumor cells
US7935816B2 (en) 2007-10-25 2011-05-03 Gene Tools, Llc Molecular transporter compositions comprising dendrimeric oligoguanidine with a triazine core that facilitate delivery into cells in vivo
BRPI0819828A8 (en) 2007-11-15 2022-12-27 Avi Biopharma Inc MORPHOLIN OLIGOMER SYNTHESIS PROCESS
SI2607484T1 (en) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
WO2010064146A2 (en) 2008-12-02 2010-06-10 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
PL2417257T3 (en) 2009-04-10 2016-11-30 Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
RU2566724C9 (en) 2009-06-17 2019-03-12 Байоджен Ma Инк. Compositions and methods of modulating smn2 splicing in subject
KR101885383B1 (en) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 Novel nucleic acid prodrugs and methods of use thereof
US8536148B2 (en) 2009-09-04 2013-09-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Disabling autophagy as a treatment for lysosomal storage diseases
US8470987B2 (en) 2009-09-16 2013-06-25 Chiralgen, Ltd. Protective group for synthesis of RNA and derivative
JP5993744B2 (en) 2009-12-29 2016-09-14 カッパーアールエヌエー，インコーポレイテッド Treatment of nuclear respiratory factor 1 related diseases by inhibition of natural antisense transcripts against nuclear respiratory factor 1 (NRF1)
FR2957090A1 (en) 2010-03-05 2011-09-09 Immunosearch METHOD FOR EVALUATING THE IRRITANT POTENTIAL OF A TEST COMPOUND
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
CA2799501C (en) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP5906237B2 (en) 2010-06-01 2016-04-20 エータイアー ファーマ， インコーポレイテッド Innovative discovery of therapeutic, diagnostic and antibody compositions related to protein fragments of lysyl tRNA synthetase
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
CN103154009B (en) 2010-09-30 2015-06-10 日本新药株式会社 Morpholino nucleic acid derivative
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA3092114A1 (en) * 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA2848899A1 (en) 2011-08-25 2013-02-28 Db Equipment As Accessory bag having reinforced sidewalls and variable length
PT2581448E (en) 2011-10-13 2015-05-21 Institut National De La Santé Et De La Rech Médicale (Inserm) Tricyclo-phosphorothioate dna
CN111808135A (en) 2011-11-18 2020-10-23 萨勒普塔医疗公司 Functionally modified oligonucleotides and subunits thereof
US9944926B2 (en) 2011-11-30 2018-04-17 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
US9725716B2 (en) 2011-12-06 2017-08-08 Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
CA3120918A1 (en) 2012-01-27 2013-08-01 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
DE102012101676A1 (en) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Method and device for processing forms with a data processing system
US20150133529A1 (en) 2012-05-16 2015-05-14 Rana Therapeutics, Inc. Compositions and methods for modulating bdnf expression
US20150141320A1 (en) 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
EP3444342B1 (en) * 2012-07-11 2020-05-27 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
DK2872485T3 (en) 2012-07-13 2021-03-08 Wave Life Sciences Ltd ASYMMETRIC ASSISTANCE GROUP
JP6453212B2 (en) 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． Chiral control
JP6449231B2 (en) 2013-03-14 2019-01-09 サレプタ セラピューティクス， インコーポレイテッド Exon skipping composition for treating muscular dystrophy
US9108673B2 (en) 2013-03-15 2015-08-18 Steering Solutions Ip Holding Corporation Crash release mechanism for automotive steering column
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2015108046A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
WO2015108047A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
SG10201912897UA (en) 2014-01-16 2020-02-27 Wave Life Sciences Ltd Chiral design
EP3143141B1 (en) 2014-05-16 2019-10-02 Oregon State University Antisense antibacterial compounds and methods
EP3146050B1 (en) * 2014-05-23 2020-08-12 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
EP3155128B1 (en) * 2014-06-10 2019-05-15 Erasmus University Medical Center Rotterdam Methods for characterizing alternatively or aberrantly spliced mrna isoforms
US10308940B2 (en) 2014-06-10 2019-06-04 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of Pompe disease
CA2917705C (en) 2015-02-25 2019-06-18 Jervis B. Webb Company Method and system for automated luggage security inspection
EP3262056A4 (en) 2015-02-27 2018-09-19 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3445405A4 (en) 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

Also Published As

Publication number Publication date
AU2017254106A1 (en) 2018-11-01
US11060089B2 (en) 2021-07-13
KR102522059B1 (en) 2023-04-14
JP7342169B2 (en) 2023-09-11
BR112018071477A2 (en) 2019-02-19
US20220364085A1 (en) 2022-11-17
MX2018012695A (en) 2019-06-20
EP3445405A4 (en) 2019-12-18
CN109414511A (en) 2019-03-01
WO2017184529A1 (en) 2017-10-26
JP2023155418A (en) 2023-10-20
TW201741459A (en) 2017-12-01
US20190284555A1 (en) 2019-09-19
JP2022082544A (en) 2022-06-02
EA201892366A1 (en) 2019-03-29
CA3021267A1 (en) 2017-10-26
EP3445405A1 (en) 2019-02-27
JP2019513405A (en) 2019-05-30
SA518400251B1 (en) 2023-01-12
CN109414511B (en) 2023-05-23
TWI766861B (en) 2022-06-11
KR20180134389A (en) 2018-12-18
JP7033547B2 (en) 2022-03-10

Similar Documents

Publication Publication Date Title
SG11201808964PA (en) Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
SG11201809912UA (en) Hybrid carriers for nucleic acid cargo
SG11201906297QA (en) Nucleic acids encoding crispr-associated proteins and uses thereof
SG11201806863WA (en) Tetracyclic pyridone compounds as antivirals
SG11202000274RA (en) Oligonucleotide compositions and methods thereof
SG11201809344QA (en) Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde
SG11201908330PA (en) Farnesoid x receptor agonists and uses thereof
SG11201909777YA (en) Modulatory polynucleotides
SG11201805320XA (en) Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
SG11201901445TA (en) A crystalline 19-nor c3, 3-disubstituted c21-n-pyrazolyl steroid
SG11201907846VA (en) Therapeutic rna
SG11201809700YA (en) Gdf15 fusion proteins and uses thereof
SG11201811232XA (en) Treatment of amd using aav2 variant with aflibercept
SG11201900349VA (en) Somatostatin modulators and uses thereof
SG11201908512YA (en) Somatostatin modulators and uses thereof
SG11201903167QA (en) Compounds and methods for reducing atxn3 expression
SG11201901996UA (en) Formulations of ( r)-2-amino-3-phenylpropyl carbamate
SG11201808237UA (en) Substituted indole compound derivatives as dengue viral replication inhibitors
SG11201809534UA (en) Methods of treating autoimmune disease using allogeneic t cells
SG11201908465QA (en) Substituted indoline derivatives as dengue viral replication inhibitors
SG11201811230RA (en) Compositions and methods for reducing ocular neovascularization
SG11201909856XA (en) Mesenchymal lineage precursor or stem cells with enhanced immunosuppression
SG11201808592PA (en) Method for selection of high m6p recombinant proteins
SG11201900633XA (en) Piperidine cxcr7 receptor modulators
SG11201903061YA (en) Combination treatments comprising administration of imidazopyrazinones