SG11201900238UA - Compounds and methods for modulation of smn2 - Google Patents
Compounds and methods for modulation of smn2Info
- Publication number
- SG11201900238UA SG11201900238UA SG11201900238UA SG11201900238UA SG11201900238UA SG 11201900238U A SG11201900238U A SG 11201900238UA SG 11201900238U A SG11201900238U A SG 11201900238UA SG 11201900238U A SG11201900238U A SG 11201900238UA SG 11201900238U A SG11201900238U A SG 11201900238UA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- gazelle
- carlsbad
- compounds
- smn2
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 abstract 2
- 102100021947 Survival motor neuron protein Human genes 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 239000003814 drug Substances 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
Classifications
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- 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
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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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- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
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- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/322—2'-R Modification
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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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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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
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111111101111 0 DOI HIM vu 1101 0 011111111111111111111111111111111111111111111 Organization 0 International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/014041 A2 18 January 2018 (18.01.2018) W I P0 I P C T (51) International Patent Classification: A61K 48/00 (2006.01) C07H 21/02 (2006.01) (21) International Application Number: PCT/US2017/042463 (22) International Filing Date: 17 July 2017 (17.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/363,195 15 July 2016 (15.07.2016) US (71) Applicant: IONIS PHARMACEUTICALS, INC. [US/US]; 2855 Gazelle Court, Carlsbad, CA 92010 (US). (72) Inventors: RIGO, Frank; 2855 Gazelle Ct., Carlsbad, CA 92010 (US). PRAKASH, Thazha, P.; 2855 Gazelle Ct., Carlsbad, CA 92010 (US). SETH, Punit, P.; 2855 Gazelle Ct., Carlsbad, CA 92010 (US). Agent: EVANS, William M. et al.; Mcneill Baur PLLC, 125 Cambridge Park Dr, Suite 301, Cambridge, MA 02140 (74) — = (US). Designated States (unless otherwise indicated, for every (81) = 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, 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, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = Designated States (unless otherwise indicated, for every (84) 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, = 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: el — without international search report and to be republished upon receipt of that report (Rule 48.2(g)) — with sequence listing part of description (Rule 5.2(a)) 71' 0 71' 1-1 0 GO\" (54) Title: COMPOUNDS AND METHODS FOR MODULATION OF SMN2 1-1 0 (57) : Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2. Also provided are uses \" of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662363195P | 2016-07-15 | 2016-07-15 | |
PCT/US2017/042463 WO2018014041A2 (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201900238UA true SG11201900238UA (en) | 2019-02-27 |
Family
ID=60952211
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201900238UA SG11201900238UA (en) | 2016-07-15 | 2017-07-17 | Compounds and methods for modulation of smn2 |
Country Status (25)
Country | Link |
---|---|
US (2) | US20190323006A1 (en) |
EP (2) | EP4206213A1 (en) |
JP (2) | JP7197463B2 (en) |
KR (1) | KR102556825B1 (en) |
CN (1) | CN109844115B (en) |
AU (1) | AU2017297622A1 (en) |
BR (1) | BR112019000356A2 (en) |
CA (1) | CA3030864A1 (en) |
CL (2) | CL2019000115A1 (en) |
CO (1) | CO2019001236A2 (en) |
DK (1) | DK3484524T3 (en) |
ES (1) | ES2935658T3 (en) |
FI (1) | FI3484524T3 (en) |
HR (1) | HRP20221378T1 (en) |
HU (1) | HUE060831T2 (en) |
IL (2) | IL264216B2 (en) |
LT (1) | LT3484524T (en) |
MX (1) | MX2019000619A (en) |
PE (1) | PE20190513A1 (en) |
PL (1) | PL3484524T3 (en) |
PT (1) | PT3484524T (en) |
RS (1) | RS63928B1 (en) |
SG (1) | SG11201900238UA (en) |
SI (1) | SI3484524T1 (en) |
WO (1) | WO2018014041A2 (en) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2983676B1 (en) | 2013-04-12 | 2019-03-20 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
WO2017035366A1 (en) | 2015-08-26 | 2017-03-02 | Incyte Corporation | Pyrrolopyrimidine derivatives as tam inhibitors |
JP7034084B2 (en) | 2016-03-28 | 2022-03-11 | インサイト・コーポレイション | Pyrrolotriazine compounds as TAM inhibitors |
TWI821200B (en) | 2017-09-27 | 2023-11-11 | 美商英塞特公司 | Salts of tam inhibitors |
MX2021000127A (en) | 2018-06-29 | 2021-03-29 | Incyte Corp | Formulations of an axl/mer inhibitor. |
JP2022544538A (en) * | 2019-08-15 | 2022-10-19 | バイオジェン・エムエイ・インコーポレイテッド | Combination therapy for spinal muscular atrophy |
EP4032551A4 (en) * | 2019-09-18 | 2023-12-20 | National University Corporation Tokyo Medical and Dental University | Nucleic acid complex |
IL295605A (en) * | 2020-02-28 | 2022-10-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating smn2 |
FR3128873A1 (en) | 2021-11-10 | 2023-05-12 | Biophytis | Phytoecdysones and/or 20-hydroxyecdysone derivatives in combination with an active ingredient aimed at restoring SMN expression for their use in the treatment of spinal muscular atrophy |
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