SG11202000274RA - Oligonucleotide compositions and methods thereof - Google Patents
Oligonucleotide compositions and methods thereofInfo
- Publication number
- SG11202000274RA SG11202000274RA SG11202000274RA SG11202000274RA SG11202000274RA SG 11202000274R A SG11202000274R A SG 11202000274RA SG 11202000274R A SG11202000274R A SG 11202000274RA SG 11202000274R A SG11202000274R A SG 11202000274RA SG 11202000274R A SG11202000274R A SG 11202000274RA
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- cambridge
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- life sciences
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1072—Differential gene expression library synthesis, e.g. subtracted libraries, differential screening
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Saccharide Compounds (AREA)
Abstract
INTERNATIONAL APPLICATION (19) World Intellectual Property Organization International Bureau PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) IiiiionolivivivillEviiliololviiiilviviiEmolivimovois (10) International Publication Number WO 2019/032612 Al (43) International Publication Date 14 February 2019 (14.02.2019) WIPO I PCT (51) International Patent Classification: (72) Inventors; and A61K 31/711 (2006.01) A61K 31/7125 (2006.01) (71) Applicants (for US only): VARGEESE, Chandra A61K 31/712 (2006.01) C12N 15/113 (2010.01) [US/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord (21) International Application Number: PCT/US2018/045659 Avenue, Cambridge, Massachusetts 02138 (US). ZHONG, Zhong [US/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Massachusetts 02138 (US). IWAMOTO, Naoki [JP/US]; c/o WAVE LIFE (22) International Filing Date: SCIENCES LTD., 733 Concord Avenue, Cambridge, Mass- 07 August 2018 (07.08.2018) achusetts 02138 (US). ZHANG, Jason Jingxin [US/US]; (25) Filing Language: English c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Massachusetts 02138 (US). DODART, Jean- English (26) Publication Language: Cosme [US/US]; c/o WAVE LIFE SCIENCES LTD., 733 (30) Priority Data: Concord Avenue, Cambridge, Massachusetts 02138 (US). 62/542,778 08 August 2017 (08.08.2017) US LIU, Yuanjing [CN/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Massachusetts (71) Applicant: WAVE LIFE SCIENCES LTD. [SG/SG]; 7 02138 (US). KANDASAMY, Pachamuthu [CA/US]; c/ Straits View #12-00, Marina One East Tower, Singapore o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, 018936 (SG). Cambridge, Massachusetts 02138 (US). DIVAKARA- (54) Title: OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF FIG. 4 mRNA Vi V3 V2 Pa Ioaical allele PGGGCC)30+ lb 2 34 5 6 7 8 91011 ViDet—E}Filia arefl—OH-Eli11-14-414 V3 Det -- EN - 00 - 4} - 04÷00 MUM Vi vs V2 Era min 81111111111111IM V2 13-EIHEHHHHO Pathological b V1 /Y3 + intronl ace Stabilized in tron 1 4 AS RNA Almi— acts I~tItII IIt (57) : Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders. [Continued on next page] Healthy allele NAM VIII WO 2019/032612 Al MIDEDIMOMOIDEIROM010101110010HEMOHHIMEMOIMIE MENON, Sethumadhavan [IN/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Mass- achusetts 02138 (US). LU, Genliang [US/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Massachusetts 02138 (US). MARAPPAN, Subramanian [US/US]; c/o WAVE LIFE SCIENCES LTD., 733 Concord Avenue, Cambridge, Massachusetts 02138 (US). (74) Agent: LI, Xiaodong et al.; Choate, Hall & Stewart LLP, Two International Place, Boston, Massachusetts 02110 (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, 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. (84) 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, 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) Published: — with international search report (Art. 21(3))
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
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US201762542778P | 2017-08-08 | 2017-08-08 | |
PCT/US2018/045659 WO2019032612A1 (en) | 2017-08-08 | 2018-08-07 | Oligonucleotide compositions and methods thereof |
Publications (1)
Publication Number | Publication Date |
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SG11202000274RA true SG11202000274RA (en) | 2020-02-27 |
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Family Applications (1)
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SG11202000274RA SG11202000274RA (en) | 2017-08-08 | 2018-08-07 | Oligonucleotide compositions and methods thereof |
Country Status (11)
Country | Link |
---|---|
US (2) | US11739325B2 (en) |
EP (2) | EP3664815A4 (en) |
JP (3) | JP7422068B2 (en) |
KR (1) | KR20200035301A (en) |
CN (2) | CN111032057A (en) |
AR (1) | AR112779A1 (en) |
AU (1) | AU2018316159A1 (en) |
CA (1) | CA3072076A1 (en) |
SG (1) | SG11202000274RA (en) |
TW (1) | TW201920672A (en) |
WO (2) | WO2019032607A1 (en) |
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TW201722439A (en) | 2015-10-09 | 2017-07-01 | 波濤生命科學有限公司 | Oligonucleotide compositions and methods thereof |
CN114685589A (en) | 2016-03-13 | 2022-07-01 | 波涛生命科学有限公司 | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
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JP2020522510A (en) | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | Oligonucleotide composition and method of using the same |
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JP7402696B2 (en) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | Compounds, compositions, and methods for synthesis |
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2018
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- 2018-08-07 US US16/636,902 patent/US11739325B2/en active Active
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- 2018-08-07 JP JP2020506814A patent/JP7422068B2/en active Active
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- 2018-08-07 JP JP2020506776A patent/JP2020534253A/en active Pending
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- 2018-08-07 TW TW107127371A patent/TW201920672A/en unknown
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AU2018316159A1 (en) | 2020-02-06 |
AR112779A1 (en) | 2019-12-11 |
US11739325B2 (en) | 2023-08-29 |
TW201920672A (en) | 2019-06-01 |
JP2020534254A (en) | 2020-11-26 |
US20210032620A1 (en) | 2021-02-04 |
JP2024009824A (en) | 2024-01-23 |
EP3664815A1 (en) | 2020-06-17 |
JP2020534253A (en) | 2020-11-26 |
US20200362337A1 (en) | 2020-11-19 |
EP3664816A1 (en) | 2020-06-17 |
JP7422068B2 (en) | 2024-01-25 |
EP3664815A4 (en) | 2021-08-11 |
KR20200035301A (en) | 2020-04-02 |
EP3664816A4 (en) | 2021-05-19 |
WO2019032612A1 (en) | 2019-02-14 |
WO2019032607A1 (en) | 2019-02-14 |
CN111032057A (en) | 2020-04-17 |
CN110996968A (en) | 2020-04-10 |
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