SG11201810143PA - Exon skipping oligomers for muscular dystrophy - Google Patents
Exon skipping oligomers for muscular dystrophyInfo
- Publication number
- SG11201810143PA SG11201810143PA SG11201810143PA SG11201810143PA SG11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- june
- pct
- cambridge
- suite
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 208000035342 Isolated congenital onychodysplasia Diseases 0.000 abstract 1
- 241001635911 Sarepta Species 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000003387 muscular Effects 0.000 abstract 1
- 201000010158 nonsyndromic congenital nail disorder 7 Diseases 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 102220240796 rs553605556 Human genes 0.000 abstract 1
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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
- C07F9/65583—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
- C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Management, Administration, Business Operations System, And Electronic Commerce (AREA)
- Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization 0 MD 1101111 0 DOI HIM 011101 0 011111111111101E 111111111011111111 International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/005805 Al 04 January 2018 (04.01.2018) W I PO I PCT (51) International Patent Classification: COIF 9/6533 (2006.01) A61K 31/7088 (2006.01) C12N 15/113 (2010.01) A61P 21/00 (2006.01) (21) International Application Number: PCT/US2017/040017 (22) International Filing Date: 29 June 2017 (29.06.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/357,072 30 June 2016 (30.06.2016) US 62/356,923 30 June 2016 (30.06.2016) US (71) Applicant: SAREPTA THERAPEUTICS, INC. [US/US]; 215 First Street, Suite 415, Cambridge, MA 02142 (US). (72) Inventors: FRANK, Diane, Elizabeth; 215 First Street, _ Suite 415, Cambridge, MA 02142 (US). BESTWICK, Richard, K.; 15052 Fall River Dr., Bend, OR 97707 (US). — = (74) Agent: MANDRAGOURAS, Amy, E. et al.; Nelson = Mullins Riley & Scarborough LLP, One Post Office Square, Boston, MA 02109-2127 (US). = (81) 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, DE, DJ, DK, DM, DO, CA, CH, CL, CN, CO, CR, CU, CZ, = DZ, EC, EE, EG, ES, FI, HN, GB, GD, GE, GH, GM, GT, = HR, HU, ID, IL, IN, IR, IS, JO, 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, ZA, ZM, ZW. UA, UG, US, UZ, VC, VN, = = Designated States indicated, (84) (unless otherwise for every = kind of regional protection available): ARIPO (BW, GH, = GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, = ZM, ZW), Eurasian AZ, BY, KG, KZ, RU, TJ, UG, (AM, 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, = 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). 1-1 -•!! Published: — with international search report (Art. 21(3)) C — with sequence listing part of description (Rule 5.2(a)) GC kr) 0 0 --.... 00 1-1 (54) Title: EXON SKIPPING OLIGOMERS FOR MUSCULAR 0 DYSTROPHY ei (57) : Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping 0 are described.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662357072P | 2016-06-30 | 2016-06-30 | |
US201662356923P | 2016-06-30 | 2016-06-30 | |
PCT/US2017/040017 WO2018005805A1 (en) | 2016-06-30 | 2017-06-29 | Exon skipping oligomers for muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201810143PA true SG11201810143PA (en) | 2019-01-30 |
Family
ID=59315756
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201810143PA SG11201810143PA (en) | 2016-06-30 | 2017-06-29 | Exon skipping oligomers for muscular dystrophy |
Country Status (15)
Country | Link |
---|---|
US (2) | US20190262375A1 (en) |
EP (1) | EP3478697A1 (en) |
JP (1) | JP2019525742A (en) |
KR (1) | KR20190024977A (en) |
CN (1) | CN109311919A (en) |
AU (1) | AU2017290231A1 (en) |
BR (1) | BR112019000006A2 (en) |
CA (1) | CA3025575A1 (en) |
CO (1) | CO2018014029A2 (en) |
IL (1) | IL263892A (en) |
MA (1) | MA45618A (en) |
MX (1) | MX2018016052A (en) |
SG (1) | SG11201810143PA (en) |
TW (1) | TW201811807A (en) |
WO (1) | WO2018005805A1 (en) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR102581868B1 (en) | 2009-11-12 | 2023-10-04 | 더 유니버시티 오브 웨스턴 오스트레일리아 | Antisense Molecules and Methods for Treating Pathologies |
TWI541024B (en) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | Antisense nucleic acid |
MX2018014160A (en) * | 2016-06-30 | 2019-04-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers. |
US20210102205A1 (en) * | 2018-04-26 | 2021-04-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP3810150A4 (en) * | 2018-06-14 | 2023-01-25 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
KR20210145192A (en) * | 2019-03-28 | 2021-12-01 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | How to Treat Muscular Dystrophy with Casimersen |
WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
AU2022298028A1 (en) * | 2021-06-23 | 2023-12-21 | National Center Of Neurology And Psychiatry | Combination of antisense oligomers |
Family Cites Families (88)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CH445129A (en) | 1964-04-29 | 1967-10-15 | Nestle Sa | Process for the preparation of high molecular weight inclusion compounds |
US3459731A (en) | 1966-12-16 | 1969-08-05 | Corn Products Co | Cyclodextrin polyethers and their production |
US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic 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 |
US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
JP3022967B2 (en) | 1985-03-15 | 2000-03-21 | アンチバイラルズ インコーポレイテッド | Stereoregular polynucleotide binding polymer |
US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
KR0166088B1 (en) | 1990-01-23 | 1999-01-15 | . | Derivatives of cyclodextrins exhibiting enhanced aqueous solubility and the use thereof |
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 |
WO1994002595A1 (en) | 1992-07-17 | 1994-02-03 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of animal diseases |
ES2290951T3 (en) | 1993-05-11 | 2008-02-16 | The University Of North Carolina At Chapel Hill | OLIGONUCLEOTID ANTISENTIDO THAT COMBAT THE CUTTING AND OPENING PUMP AND METHODS OF THE SAME USE. |
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 |
US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
JP2000125448A (en) | 1998-10-14 | 2000-04-28 | Yazaki Corp | Electrical junction box |
JP2000256547A (en) | 1999-03-10 | 2000-09-19 | Sumitomo Dow Ltd | Resin composition for heat-resistant card |
US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
DK1178999T3 (en) | 1999-05-04 | 2007-08-06 | Santaris Pharma As | L-ribo-LNA analogues |
JP2000325085A (en) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | Pharmaceutical composition for treatment of duchenne muscular dystrophy |
EP1242052A4 (en) | 1999-12-29 | 2003-07-02 | A James Mixson | Histidine copolymer and methods for using same |
US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
EP1446412B1 (en) | 2001-09-04 | 2012-03-07 | Exiqon A/S | Novel lna compositions and uses thereof |
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 |
WO2004043977A2 (en) | 2002-11-05 | 2004-05-27 | Isis Pharmaceuticals, Inc. | 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations |
CA3001404C (en) | 2002-11-25 | 2020-07-28 | Masafumi Matsuo | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
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 |
US7211668B2 (en) | 2003-07-28 | 2007-05-01 | Panagene, Inc. | PNA monomer and precursor |
EP3808845A1 (en) | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
NZ563206A (en) | 2005-04-22 | 2009-09-25 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mRNA by interfering with the binding or SR proteins and by interfering with secondary RNA structure |
WO2007058894A2 (en) | 2005-11-10 | 2007-05-24 | The University Of North Carolina At Chapel Hill | Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease |
JP3151554U (en) | 2006-05-17 | 2009-07-02 | スヴェトラナ アナトレフナ ソコロヴァ | Means of transport |
EP2167135A2 (en) | 2007-07-12 | 2010-03-31 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
ES2914775T3 (en) | 2007-10-26 | 2022-06-16 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
WO2009127230A1 (en) | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
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 |
BRPI0920276B1 (en) | 2008-10-24 | 2021-06-08 | Sarepta Therapeutics, Inc | antisense oligonucleotide, composition comprising the same and use of said oligonucleotide to treat muscular dystrophy |
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 |
EP2370451B1 (en) | 2008-12-02 | 2016-11-16 | Wave Life Sciences Japan, Inc. | Method for the synthesis of phosphorus atom modified nucleic acids |
CN102625840A (en) | 2009-04-10 | 2012-08-01 | 肌肉学研究协会 | Tricyclo-DNA antisense oligonucleotides, compositions, and methods for the treatment of disease |
AU2010239779A1 (en) | 2009-04-24 | 2011-11-17 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating DMD |
RU2612521C2 (en) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Novel prodrugs of nucleic acids and their application methods |
ES2586683T3 (en) | 2009-09-16 | 2016-10-18 | Wave Life Sciences Japan, Inc. | Novel protective group to synthesize RNA and derivatives thereof |
KR102581868B1 (en) * | 2009-11-12 | 2023-10-04 | 더 유니버시티 오브 웨스턴 오스트레일리아 | Antisense Molecules and Methods for Treating Pathologies |
NZ603606A (en) * | 2010-05-28 | 2015-06-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
TWI541024B (en) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | Antisense nucleic acid |
WO2012039448A1 (en) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | Asymmetric auxiliary group |
WO2012109296A1 (en) | 2011-02-08 | 2012-08-16 | The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center | Antisense oligonucleotides |
CN107693797B (en) * | 2011-05-05 | 2021-05-11 | 萨勒普塔医疗公司 | Peptide oligonucleotide conjugates |
US9607308B2 (en) | 2011-06-29 | 2017-03-28 | American Express Travel Related Services Company, Inc. | Spend based digital ad targeting and measurement |
DK2581448T3 (en) | 2011-10-13 | 2015-04-27 | Ass Inst De Myologie | Tricyclo-DNA phosphorothioate |
CN110055244A (en) * | 2011-12-28 | 2019-07-26 | 日本新药株式会社 | Antisense nucleic acid |
CN111893117B (en) | 2012-01-27 | 2024-06-04 | 比奥马林技术公司 | RNA regulatory oligonucleotides with improved properties for the treatment of duchenne muscular dystrophy 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 |
CN104603271B (en) | 2012-07-03 | 2019-07-19 | 马林生物科技有限公司 | For treating the oligonucleotides of muscular dystrophy patient |
AU2013289880B2 (en) | 2012-07-13 | 2018-08-02 | Wave Life Sciences Ltd. | Chiral control |
ES2862073T3 (en) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Asymmetric auxiliary group |
EP3885439A1 (en) | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
KR102258326B1 (en) * | 2013-03-14 | 2021-06-02 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Exon skipping compositions for treating muscular dystrophy |
NZ631132A (en) * | 2013-03-14 | 2017-06-30 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
US9506058B2 (en) * | 2013-03-15 | 2016-11-29 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
JPWO2015108048A1 (en) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | Chiral nucleic acid adjuvant and antitumor agent having antitumor activity |
EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
MY193116A (en) | 2014-01-16 | 2022-09-26 | Wave Life Sciences Ltd | Chiral design |
RS58573B1 (en) | 2014-03-12 | 2019-05-31 | Nippon Shinyaku Co Ltd | Antisense nucleic acid |
HUE047502T2 (en) | 2014-06-17 | 2020-04-28 | Nippon Shinyaku Co Ltd | Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy |
EP3359668A4 (en) * | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
-
2017
- 2017-06-29 JP JP2018567259A patent/JP2019525742A/en active Pending
- 2017-06-29 US US16/312,803 patent/US20190262375A1/en not_active Abandoned
- 2017-06-29 CN CN201780038876.7A patent/CN109311919A/en active Pending
- 2017-06-29 WO PCT/US2017/040017 patent/WO2018005805A1/en unknown
- 2017-06-29 MA MA045618A patent/MA45618A/en unknown
- 2017-06-29 KR KR1020197002453A patent/KR20190024977A/en not_active Application Discontinuation
- 2017-06-29 MX MX2018016052A patent/MX2018016052A/en unknown
- 2017-06-29 AU AU2017290231A patent/AU2017290231A1/en not_active Abandoned
- 2017-06-29 TW TW106121834A patent/TW201811807A/en unknown
- 2017-06-29 BR BR112019000006-2A patent/BR112019000006A2/en not_active Application Discontinuation
- 2017-06-29 EP EP17737999.7A patent/EP3478697A1/en not_active Withdrawn
- 2017-06-29 SG SG11201810143PA patent/SG11201810143PA/en unknown
- 2017-06-29 CA CA3025575A patent/CA3025575A1/en not_active Abandoned
-
2018
- 2018-12-21 CO CONC2018/0014029A patent/CO2018014029A2/en unknown
- 2018-12-23 IL IL263892A patent/IL263892A/en unknown
-
2020
- 2020-07-28 US US16/941,318 patent/US20210187003A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
US20190262375A1 (en) | 2019-08-29 |
AU2017290231A1 (en) | 2019-02-07 |
BR112019000006A2 (en) | 2019-04-16 |
IL263892A (en) | 2019-01-31 |
CN109311919A (en) | 2019-02-05 |
KR20190024977A (en) | 2019-03-08 |
TW201811807A (en) | 2018-04-01 |
US20210187003A1 (en) | 2021-06-24 |
CO2018014029A2 (en) | 2019-03-18 |
MA45618A (en) | 2019-05-08 |
MX2018016052A (en) | 2019-05-02 |
JP2019525742A (en) | 2019-09-12 |
WO2018005805A1 (en) | 2018-01-04 |
CA3025575A1 (en) | 2018-01-04 |
EP3478697A1 (en) | 2019-05-08 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
SG11201810143PA (en) | Exon skipping oligomers for muscular dystrophy | |
SG11201811491YA (en) | Quinazoline and indole compounds to treat medical disorders | |
SG11201810579YA (en) | Pyrimidin-2-ylamino-1h-pyrazols as lrrk2 inhibitors for use in the treatment of neurodegenerative disorders | |
SG11201809064QA (en) | Chimeric neurotoxins | |
SG11201909955XA (en) | Formulations of anti-lag3 antibodies and co-formulations of anti-lag3 antibodies and anti-pd-1 antibodies | |
SG11201804758QA (en) | Aqueous pharmaceutical formulation comprising anti-pd-1 antibody avelumab | |
SG11201907034PA (en) | Methods of treating influenza | |
SG11201909949XA (en) | Targeted immunotolerance | |
SG11201804934PA (en) | Novel Compounds | |
SG11201804647TA (en) | Dosing regimens for 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde | |
SG11201809344QA (en) | Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde | |
SG11201808990QA (en) | Compositions for topical application of compounds | |
SG11201809499UA (en) | Processes for preparing phosphorodiamidate morpholino oligomers | |
SG11201901834WA (en) | Micrornas as biomarkers for endometriosis | |
SG11201500005SA (en) | Pyrimidine pyrazolyl derivatives | |
SG11201407851RA (en) | Synthesis of pyrrolo [2, 3 - b] pyridines | |
SG11201906436VA (en) | Novel heterocyclic compound, its preparation method, and pharmaceutical composition comprising the same | |
SG11201407898WA (en) | Electrode testing apparatus | |
SG11201408501UA (en) | Preparation, uses and solid forms of obeticholic acid | |
SG11201909807TA (en) | Methods of manufacturing of niraparib | |
SG11201809497RA (en) | Processes for preparing phosphorodiamidate morpholino oligomers | |
SG11201808106YA (en) | Use of masitinib for treatment of an amyotrophic lateral sclerosis patient subpopulation | |
SG11201804589TA (en) | Piperidinone formyl peptide 2 receptor and formyl peptide 1 receptor agonists | |
SG11201404836QA (en) | Long-acting coagulation factors and methods of producing same | |
SG11201900132QA (en) | Selecting neoepitopes as disease-specific targets for therapy with enhanced efficacy |