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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (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 |
|---|---|---|---|
| US201662356923P | 2016-06-30 | 2016-06-30 | |
| US201662357072P | 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 (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102113306B1 (en) | 2009-11-12 | 2020-05-21 | 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 |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| KR20250044471A (en) | 2015-11-30 | 2025-03-31 | 듀크 유니버시티 | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS TO INACTIVATE IN VIVO GENE TARGETS AND METHODS OF USE |
| CN109152792B (en) * | 2016-06-30 | 2021-09-14 | 萨勒普塔医疗公司 | Process for preparing phosphoric acid diamide morpholino oligomer |
| JP7490211B2 (en) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Therapeutic Applications of CPF1-Based Genome Editing |
| JP2021521794A (en) * | 2018-04-26 | 2021-08-30 | 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 oligomers and oligomeric conjugates for muscular dystrophy |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2019241470A2 (en) * | 2018-06-14 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| JP2022526763A (en) * | 2019-03-28 | 2022-05-26 | サレプタ セラピューティクス， インコーポレイテッド | How to treat muscular dystrophy with Casimersen |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| JPWO2022270585A1 (en) * | 2021-06-23 | 2022-12-29 |
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-
2017
- 2017-06-29 SG SG11201810143PA patent/SG11201810143PA/en unknown
- 2017-06-29 WO PCT/US2017/040017 patent/WO2018005805A1/en not_active Ceased
- 2017-06-29 AU AU2017290231A patent/AU2017290231A1/en not_active Abandoned
- 2017-06-29 JP JP2018567259A patent/JP2019525742A/en active Pending
- 2017-06-29 CA CA3025575A patent/CA3025575A1/en not_active Abandoned
- 2017-06-29 US US16/312,803 patent/US20190262375A1/en not_active Abandoned
- 2017-06-29 EP EP17737999.7A patent/EP3478697A1/en not_active Withdrawn
- 2017-06-29 KR KR1020197002453A patent/KR20190024977A/en not_active Ceased
- 2017-06-29 TW TW106121834A patent/TW201811807A/en unknown
- 2017-06-29 CN CN201780038876.7A patent/CN109311919A/en active Pending
- 2017-06-29 BR BR112019000006-2A patent/BR112019000006A2/en not_active Application Discontinuation
- 2017-06-29 MA MA045618A patent/MA45618A/en unknown
- 2017-06-29 MX MX2018016052A patent/MX2018016052A/en unknown
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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
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| AU2017290231A1 (en) | 2019-02-07 |
| MX2018016052A (en) | 2019-05-02 |
| KR20190024977A (en) | 2019-03-08 |
| JP2019525742A (en) | 2019-09-12 |
| US20190262375A1 (en) | 2019-08-29 |
| US20210187003A1 (en) | 2021-06-24 |
| EP3478697A1 (en) | 2019-05-08 |
| CN109311919A (en) | 2019-02-05 |
| TW201811807A (en) | 2018-04-01 |
| IL263892A (en) | 2019-01-31 |
| MA45618A (en) | 2019-05-08 |
| CA3025575A1 (en) | 2018-01-04 |
| WO2018005805A1 (en) | 2018-01-04 |
| CO2018014029A2 (en) | 2019-03-18 |
| BR112019000006A2 (en) | 2019-04-16 |
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