FI3717646T3 - Angelmanin oireyhtymän antisense-hoito - Google Patents

Angelmanin oireyhtymän antisense-hoito

Info

Publication number
FI3717646T3
FI3717646T3 FIEP18883102.8T FI18883102T FI3717646T3 FI 3717646 T3 FI3717646 T3 FI 3717646T3 FI 18883102 T FI18883102 T FI 18883102T FI 3717646 T3 FI3717646 T3 FI 3717646T3
Authority
FI
Finland
Prior art keywords
antisense oligonucleotide
seq
pharmaceutical composition
use according
nucleic acid
Prior art date
Application number
FIEP18883102.8T
Other languages
English (en)
Finnish (fi)
Inventor
Scott Victor Dindot
Original Assignee
Texas A & M Univ Sys
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.)
Filing date
Publication date
Application filed by Texas A & M Univ Sys filed Critical Texas A & M Univ Sys
Application granted granted Critical
Publication of FI3717646T3 publication Critical patent/FI3717646T3/fi

Links

Classifications

    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C07H21/04—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 deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • 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
    • 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
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
FIEP18883102.8T 2017-12-01 2018-11-30 Angelmanin oireyhtymän antisense-hoito FI3717646T3 (fi)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (1)

Publication Number Publication Date
FI3717646T3 true FI3717646T3 (fi) 2025-07-17

Family

ID=66664231

Family Applications (1)

Application Number Title Priority Date Filing Date
FIEP18883102.8T FI3717646T3 (fi) 2017-12-01 2018-11-30 Angelmanin oireyhtymän antisense-hoito

Country Status (26)

Country Link
US (5) US11198869B2 (enExample)
EP (2) EP3717646B1 (enExample)
JP (3) JP7297320B2 (enExample)
KR (2) KR102723989B1 (enExample)
CN (2) CN111433361B (enExample)
AU (1) AU2018375807B2 (enExample)
BR (1) BR112020009431A2 (enExample)
CA (1) CA3079755A1 (enExample)
CL (1) CL2020001344A1 (enExample)
CO (1) CO2020006361A2 (enExample)
DK (1) DK3717646T3 (enExample)
ES (1) ES3032315T3 (enExample)
FI (1) FI3717646T3 (enExample)
HR (1) HRP20250804T1 (enExample)
IL (1) IL274146B2 (enExample)
LT (1) LT3717646T (enExample)
MX (2) MX2020005680A (enExample)
PE (1) PE20211238A1 (enExample)
PL (1) PL3717646T3 (enExample)
PT (1) PT3717646T (enExample)
RS (1) RS66986B1 (enExample)
RU (1) RU2020121752A (enExample)
SG (1) SG11202003596WA (enExample)
SI (1) SI3717646T1 (enExample)
SM (1) SMT202500334T1 (enExample)
WO (1) WO2019109001A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3374509T3 (pl) 2015-11-12 2021-05-04 F. Hoffmann-La Roche Ag Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A
WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
TWI874376B (zh) 2019-03-29 2025-03-01 美商Ionis製藥公司 用於調節ube3a-ats 之化合物及方法
TW202216996A (zh) * 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
CA3197664A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
EP4473116A2 (en) * 2022-03-07 2024-12-11 University of Connecticut Shrna targeting snord115 locations to restore paternal ube3a gene expression in angelman syndrome
CN120476156A (zh) 2023-01-31 2025-08-12 富士胶片株式会社 固化性组合物、固化物、光学材料、衍射光学元件及化合物
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025217466A1 (en) * 2024-04-11 2025-10-16 Ultragenyx Pharmaceutical Inc. Dosing regimens for antisense treatment of angelman syndrome

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2122030C (en) 1991-10-24 1997-03-04 Muthiah Manoharan Derivatized oligonucleotides having improved uptake and other properties
CZ302719B6 (cs) * 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
EP1799859B1 (en) 2004-09-17 2014-07-02 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
EP1676834A1 (en) 2004-12-30 2006-07-05 Sanofi-Aventis Deutschland GmbH Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US20080299659A1 (en) * 2007-03-02 2008-12-04 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting apob gene expression and uses thereof
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
MX2013012071A (es) 2011-04-20 2014-01-20 Roche Glycart Ag Metodo y constructos para el pasaje dependiente del ph de la barrera hematoencefalica.
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
KR20160074368A (ko) * 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
WO2013173637A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
EP4458361A3 (en) 2012-06-25 2025-01-22 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
US20180112217A1 (en) 2014-11-19 2018-04-26 Roche Innovation Center Copenhagen A/S Stereospecific Phosphorothioate LNA
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
JP6689279B2 (ja) 2014-12-16 2020-05-20 ロシュ イノベーション センター コペンハーゲン エーエス キラル毒性のスクリーニング方法
PE20171766A1 (es) * 2015-02-04 2017-12-21 Hoffmann La Roche Oligomeros antisentido de tau y usos de estos
WO2016170348A2 (en) * 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
PL3374509T3 (pl) 2015-11-12 2021-05-04 F. Hoffmann-La Roche Ag Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A
US11261209B2 (en) 2016-05-12 2022-03-01 Roche Innovation Center Copenhagen A/S Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide
GEP20217285B (en) 2016-10-04 2021-08-10 Merck Sharp & Dohme BENZO[b]THIOPHENE COMPOUNDS AS STING AGONISTS
US11591362B2 (en) 2017-03-29 2023-02-28 Roche Innovation Center Copenhagen A/S Orthogonal protecting groups for the preparation of stereodefined phosphorothioate oligonucleotides
US20200172483A1 (en) 2017-06-22 2020-06-04 Curadev Pharma Limited Heterocyclic small molecule modulators of human sting

Also Published As

Publication number Publication date
US20250066786A1 (en) 2025-02-27
WO2019109001A1 (en) 2019-06-06
EP4328306A2 (en) 2024-02-28
CN118291458A (zh) 2024-07-05
EP3717646A4 (en) 2021-12-01
US11685920B2 (en) 2023-06-27
JP2021505129A (ja) 2021-02-18
US12180480B2 (en) 2024-12-31
PL3717646T3 (pl) 2025-10-27
CO2020006361A2 (es) 2020-08-10
PT3717646T (pt) 2025-07-28
IL274146A (en) 2020-06-30
US20230332152A1 (en) 2023-10-19
JP2023123517A (ja) 2023-09-05
CN111433361B (zh) 2024-03-29
EP4328306A3 (en) 2025-05-21
AU2018375807B2 (en) 2025-04-10
DK3717646T3 (en) 2025-05-19
IL274146B2 (en) 2024-11-01
CL2020001344A1 (es) 2020-09-21
JP2025041632A (ja) 2025-03-26
US20230112629A1 (en) 2023-04-13
KR102723989B1 (ko) 2024-11-01
KR20200094152A (ko) 2020-08-06
US11739328B2 (en) 2023-08-29
US11198869B2 (en) 2021-12-14
KR20240161203A (ko) 2024-11-12
CA3079755A1 (en) 2019-06-06
HRP20250804T1 (hr) 2025-09-12
IL274146B1 (en) 2024-07-01
PE20211238A1 (es) 2021-07-09
SG11202003596WA (en) 2020-05-28
CN111433361A (zh) 2020-07-17
EP3717646A1 (en) 2020-10-07
US20220090075A1 (en) 2022-03-24
JP7601441B2 (ja) 2024-12-17
RS66986B1 (sr) 2025-07-31
SI3717646T1 (sl) 2025-08-29
MX2025009778A (es) 2025-09-02
SMT202500334T1 (it) 2025-11-10
LT3717646T (lt) 2025-06-10
BR112020009431A2 (pt) 2020-10-13
RU2020121752A (ru) 2021-12-30
JP7297320B2 (ja) 2023-06-26
AU2018375807A1 (en) 2020-05-07
US20200370046A1 (en) 2020-11-26
EP3717646B1 (en) 2025-04-23
MX2020005680A (es) 2020-08-20
ES3032315T3 (en) 2025-07-17

Similar Documents

Publication Publication Date Title
FI3717646T3 (fi) Angelmanin oireyhtymän antisense-hoito
FI4220360T3 (fi) Oligonukleotideja paternaalisen ube3a:n ilmentämiseksi
JP2021505129A5 (enExample)
CA2640058C (en) Oligomeric compounds and compositions for the use in modulation of micrornas
RU2017127609A (ru) Антисмысловые олигомеры тау-белка и их применение
JP2008283975A5 (enExample)
JP7348922B2 (ja) Ｂ型肝炎感染の治療用のＰＡＰＤ５及びＰＡＰＤ７ ｍＲＮＡを低減させるための核酸分子
RU2017117510A (ru) Редактирование целевой рнк
JP2019503394A5 (enExample)
EP4455285A3 (en) Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
JP2016116520A5 (enExample)
JP2015519057A5 (enExample)
JP2018516091A5 (enExample)
JP2014527401A5 (enExample)
JP2015523855A5 (enExample)
JP2007500018A5 (enExample)
JP2019534025A (ja) 治療用オリゴヌクレオチドの捕捉および検出
CA3159501A1 (en) Methods of synthesizing rna molecules
RU2020125769A (ru) Олигонуклеотиды для модуляции экспрессии tmem106b
JP6882735B2 (ja) 構造強化されたｍｉＲＮＡ阻害剤Ｓ−ＴｕＤ
RU2019143004A (ru) Антисмысловые олигонуклеотиды для модулирования экспрессии htra1
CN114829599A (zh) Scamp3抑制剂用于治疗乙型肝炎病毒感染的用途
JPWO2019140236A5 (enExample)
Jorge 7 Advances in therapeutic oligonucleotide
EP4453206A1 (en) Threose nucleic acid antisense oligonucleotides and methods thereof