SG11201804443UA - Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome - Google Patents

Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome

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Publication number
SG11201804443UA
SG11201804443UA SG11201804443UA SG11201804443UA SG11201804443UA SG 11201804443U A SG11201804443U A SG 11201804443UA SG 11201804443U A SG11201804443U A SG 11201804443UA SG 11201804443U A SG11201804443U A SG 11201804443UA SG 11201804443U A SG11201804443U A SG 11201804443UA
Authority
SG
Singapore
Prior art keywords
international
mental retardation
massachusetts
pct
dravet syndrome
Prior art date
Application number
SG11201804443UA
Inventor
Isabel Aznarez
Huw M Nash
Original Assignee
Cold Spring Harbor Laboratory
Stoke 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.)
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Publication date
Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of SG11201804443UA publication Critical patent/SG11201804443UA/en

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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
    • C12N2310/30—Chemical structure
    • 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
    • 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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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau (43) International Publication Date ..... .....r .,„0 22 June 2017(22.06.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111111111111111111111111 2017/106377 Al (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, C12N 15/113 (2010.01) C12Q 1/68 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, C12N 15/11 (2006.01) 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, (21) International Application Number: KP, KR, KW, KZ, LA, LC, LK, LR, LS LU, LY MA, PCT/US2016/066708 MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (22) International Filing Date: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 14 December 2016 (14.12.2016) 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, (25) Filing Language: English ZA, ZM, ZW. (26) Publication Language: English (84) Designated States (unless otherwise indicated, for every (30) Priority Data: kind of regional protection available): ARIPO (BW, GH, 62/267,251 14 December 2015 (14.12.2015) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (71) Applicants: COLD SPRING HARBOR LABORAT- TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, ORY [US/US]; One Bungtown Road, Nicholas Buliding, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, P.O. Box 100, Cold Spring Harbor, New York 11724 (US). LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, STOKE THERAPEUTICS, INC. [US/US]; 3 Preston SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Court, Bedford, Massachusetts 01730 (US). GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: AZNAREZ, Isabel; 55 Magazine Street, Apt. Published: 12A, Cambridge, Massachusetts 02139 (US). NASH, Huw with international search report (Art 21(3)) M.; 4 Washington St., Lexington, Massachusetts 02421 — before the expiration of the time limit for amending the (US). claims and to be republished in the event of receipt of (74) Agent: SCHULTZ, Jason B.; Wilson Sonsini Goodrich & amendments (Rule 48.2(h)) Rosati, 650 Page Mill Road, Palo Alto, California 94304 — with sequence listing part of description (Rule 5.2(a)) (US). = (81) Designated States (unless otherwise indicated, for every kind of national available): AE, AG, AL, AM, protection (54) Title: ANTISENSE OLIGOMERS FOR TREATMENT OF AUTOSOMAL DOMINANT MENTAL RETARDATION-5 AND DRAVET SYNDROME IN TARGET REGIONS RIC pre-mRNA f 74 7 ,7 er/ war/ P inton retar ed 1-1 .4t IN ef) r------i 10 1 / 46 1 1 i CA 6 grtaagninmInnnrmnrmnrmrmnnnrnfinrmminminmmrm spliuz n y g 0 1-1 FIG. 1 IN Il O (57) : Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
SG11201804443UA 2015-12-14 2016-12-14 Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome SG11201804443UA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267251P 2015-12-14 2015-12-14
PCT/US2016/066708 WO2017106377A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome

Publications (1)

Publication Number Publication Date
SG11201804443UA true SG11201804443UA (en) 2018-06-28

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US (2) US11083745B2 (en)
EP (2) EP3390636B1 (en)
JP (2) JP7049248B2 (en)
KR (1) KR102604132B1 (en)
CN (2) CN116059236A (en)
AU (2) AU2016370653A1 (en)
CA (1) CA3005256A1 (en)
ES (1) ES2882500T3 (en)
IL (1) IL259222A (en)
SG (1) SG11201804443UA (en)
WO (1) WO2017106377A1 (en)

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WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
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CN111278991B (en) * 2017-08-25 2022-04-01 斯托克制药公司 Antisense oligomers for the treatment of conditions and diseases
GB2610100B (en) * 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
CN111727259B (en) * 2017-12-01 2024-04-19 编码治疗公司 Engineered DNA binding proteins
CN113329739A (en) 2018-08-20 2021-08-31 罗贡股份有限公司 Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathy
JP2022510673A (en) * 2018-12-04 2022-01-27 スティッチング カソリーケ ウニベルシテイト Antisense oligonucleotide rescues abnormal splicing of ABCA4
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
JP2022522205A (en) * 2019-02-27 2022-04-14 ストーク セラピューティクス，インク． Antisense oligomers for the treatment of pathological conditions and diseases
JP2022544702A (en) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． Compositions and methods for modulating splicing and protein expression
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BR112022010882A2 (en) * 2019-12-06 2022-10-04 Stoke Therapeutics Inc ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
AU2021272832A1 (en) * 2020-05-11 2022-12-15 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating disorders associated with loss-of-function mutations in SYNGAP1
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