JP7049248B2 - 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー - Google Patents
常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー Download PDFInfo
- Publication number
- JP7049248B2 JP7049248B2 JP2018529250A JP2018529250A JP7049248B2 JP 7049248 B2 JP7049248 B2 JP 7049248B2 JP 2018529250 A JP2018529250 A JP 2018529250A JP 2018529250 A JP2018529250 A JP 2018529250A JP 7049248 B2 JP7049248 B2 JP 7049248B2
- Authority
- JP
- Japan
- Prior art keywords
- mrna
- aso
- intron
- syngap1
- ric
- Prior art date
- Legal status (The legal status 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 status listed.)
- Expired - Fee Related
Links
Images
Classifications
-
- 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
- A61K31/712—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
- A61K31/713—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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- 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
-
- 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
-
- 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
- C12N2310/34—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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pathology (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022013860A JP2022062140A (ja) | 2015-12-14 | 2022-02-01 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267251P | 2015-12-14 | 2015-12-14 | |
| US62/267,251 | 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013860A Division JP2022062140A (ja) | 2015-12-14 | 2022-02-01 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019501892A JP2019501892A (ja) | 2019-01-24 |
| JP2019501892A5 JP2019501892A5 (enExample) | 2020-01-30 |
| JP7049248B2 true JP7049248B2 (ja) | 2022-04-06 |
Family
ID=59057535
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529250A Expired - Fee Related JP7049248B2 (ja) | 2015-12-14 | 2016-12-14 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
| JP2022013860A Pending JP2022062140A (ja) | 2015-12-14 | 2022-02-01 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022013860A Pending JP2022062140A (ja) | 2015-12-14 | 2022-02-01 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11083745B2 (enExample) |
| EP (2) | EP3390636B1 (enExample) |
| JP (2) | JP7049248B2 (enExample) |
| KR (1) | KR102604132B1 (enExample) |
| CN (2) | CN116059236A (enExample) |
| AU (2) | AU2016370653A1 (enExample) |
| CA (1) | CA3005256A1 (enExample) |
| ES (1) | ES2882500T3 (enExample) |
| IL (1) | IL259222A (enExample) |
| SG (1) | SG11201804443UA (enExample) |
| WO (1) | WO2017106377A1 (enExample) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| CN117821509A (zh) | 2017-04-03 | 2024-04-05 | 编码治疗公司 | 组织选择性转基因表达 |
| KR20260009385A (ko) * | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| LT3700570T (lt) * | 2017-10-23 | 2025-04-10 | Stoke Therapeutics, Inc. | Priešprasmiai oligomerai, skirti būklių ir ligų, pagrįstų nonsens kodono nulemta mrnr degradacija, gydymui |
| CN118345115A (zh) * | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス,インク. | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| CA3110211A1 (en) * | 2018-08-20 | 2020-02-27 | Rogcon, Inc. | Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies |
| EA202190736A1 (ru) * | 2018-09-26 | 2021-09-16 | Кейс Вестерн Ризерв Юниверсити | Способы и композиции для увеличения экспрессии белка и/или лечения расстройства, связанного с гаплонедостаточностью |
| JP2022510673A (ja) * | 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 | アンチセンスオリゴヌクレオチドは、abca4の異常スプライシングをレスキューする |
| US20220090087A1 (en) * | 2019-01-23 | 2022-03-24 | The Florey Institute Of Neuroscience And Mental Health | Antisense oligonucleotides targeting scn2a retained introns |
| KR102985982B1 (ko) * | 2019-02-05 | 2026-07-03 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴런 세포 흥분성을 정상화하고 드라베 증후군을 치료하기 위한 개재뉴런-특이적 치료제 |
| KR20210134003A (ko) * | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| EP3959313A4 (en) * | 2019-04-26 | 2023-09-27 | Stoke Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATING SPLICING OF ALTERNATIVE INTRONS |
| SG11202113048SA (en) | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
| KR20220104677A (ko) * | 2019-08-19 | 2022-07-26 | 스톡 테라퓨틱스, 인크. | 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법 |
| US11618900B2 (en) * | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| WO2021113773A2 (en) * | 2019-12-04 | 2021-06-10 | Pai Athma A | Identifying non-productive splice sites |
| KR20220113743A (ko) * | 2019-12-06 | 2022-08-16 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| CN115516094A (zh) * | 2020-02-28 | 2022-12-23 | 芝加哥大学 | 包含反式作用翻译活化剂的方法和组合物 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| US20230174984A1 (en) * | 2020-05-11 | 2023-06-08 | The Florey Indtitute of Neuroscience and Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in syngap1 |
| CN114480651B (zh) * | 2022-02-14 | 2023-10-20 | 天津市泌尿外科研究所 | Pcat1的反义寡核苷酸及其在制备抑制前列腺癌核酸药物中的应用 |
| CA3252931A1 (en) * | 2022-02-24 | 2023-08-31 | Quiver Holdings Inc. | THERAPEUTIC AGENTS FOR HAPLOINS SUFFICIENCY OF SYNGAP |
| EP4627084A1 (en) * | 2022-12-01 | 2025-10-08 | Camp4 Therapeutics Corporation | Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas |
| EP4642913A2 (en) * | 2022-12-28 | 2025-11-05 | The University of Chicago | Splice-switching oligonucleotides for treating syngap1-associated disorders |
| CN116334086A (zh) * | 2023-03-29 | 2023-06-27 | 广州医科大学附属第二医院 | 一种用于修复或抑制SCN1A基因异常剪接的U1-snRNA及其用途 |
| WO2024229421A1 (en) * | 2023-05-03 | 2024-11-07 | Stoke Therapeutics, Inc. | Compounds and methods for treating human subjects |
| WO2025090628A1 (en) * | 2023-10-23 | 2025-05-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies |
| WO2025199503A1 (en) * | 2024-03-22 | 2025-09-25 | Stoke Therapeutics, Inc. | Methods for treating conditions and diseases |
| WO2026065401A1 (zh) * | 2024-09-30 | 2026-04-02 | 时夕(广州)生物科技有限公司 | 一种用于调控SCN1A基因mRNA和蛋白表达的方法和化合物 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140128449A1 (en) | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
Family Cites Families (271)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
| US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
| US5914396A (en) | 1990-01-11 | 1999-06-22 | Isis Pharmaceuticals, Inc. | 2'-O-modified nucleosides and phosphoramidites |
| US7101993B1 (en) | 1990-01-11 | 2006-09-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides containing 2′-O-modified purines |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| ES2259177T3 (es) | 1990-08-13 | 2006-09-16 | Isis Pharmaceuticals, Inc. | Oligonucleotidos modificados en el azucar que detectan y modulan la expresion genica. |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| ATE256143T1 (de) | 1992-07-23 | 2003-12-15 | Isis Pharmaceuticals Inc | 2'-0-alkyl-nukleoside und-phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen |
| DE69334238D1 (de) | 1992-09-25 | 2008-09-25 | Aventis Pharma Sa | Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn |
| ATE368107T1 (de) | 1993-05-11 | 2007-08-15 | Univ North Carolina | Antisense oligonukleotide die anomales splicing verhindern und deren verwendung |
| EP0733110A1 (en) | 1993-12-24 | 1996-09-25 | University of Wales College of Medicine | Tuberous sclerosis 2 gene and uses thereof |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
| US6166197A (en) | 1995-03-06 | 2000-12-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions |
| DK0882061T3 (da) | 1996-02-14 | 2004-09-27 | Isis Pharmaceuticals Inc | Sukkermodificerede gapped oligonukleotider |
| GB9605808D0 (en) | 1996-03-20 | 1996-05-22 | Isis Innovation | CFTR gene regulator |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| ATE312935T1 (de) | 1997-06-03 | 2005-12-15 | Regulatorische sequenzen für die in-vivo- expression einer heterologen dns-sequenz in endothelzellen und ihre verwendungen. | |
| US6963589B1 (en) | 1997-07-03 | 2005-11-08 | Canon Kabushiki Kaisha | Information processing apparatus for and method of transmitting and/or receiving broadcast signal |
| GB9716231D0 (en) | 1997-07-31 | 1997-10-08 | Amersham Int Ltd | Base analogues |
| EP1015469B2 (en) | 1997-09-12 | 2015-11-18 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| US7045610B2 (en) | 1998-04-03 | 2006-05-16 | Epoch Biosciences, Inc. | Modified oligonucleotides for mismatch discrimination |
| US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| US7071324B2 (en) | 1998-10-13 | 2006-07-04 | Brown University Research Foundation | Systems and methods for sequencing by hybridization |
| US20030224514A1 (en) | 2002-05-31 | 2003-12-04 | Isis Pharmaceuticals Inc. | Antisense modulation of PPAR-delta expression |
| US6436657B1 (en) | 1998-12-18 | 2002-08-20 | E. I. Du Pont De Nemours And Company | Polynucleotides encoding aminomethyltransferases |
| BRPI0008131B8 (pt) | 1999-02-12 | 2021-05-25 | Daiichi Sankyo Co Ltd | composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| AU777049B2 (en) | 1999-03-18 | 2004-09-30 | Qiagen Gmbh | Xylo-LNA analogues |
| IL145496A0 (en) | 1999-03-24 | 2002-06-30 | Exiqon As | Improved synthesis of [2.2.1] bicyclo nucleosides |
| US6734291B2 (en) | 1999-03-24 | 2004-05-11 | Exiqon A/S | Synthesis of [2.2.1]bicyclo nucleosides |
| US6383752B1 (en) | 1999-03-31 | 2002-05-07 | Hybridon, Inc. | Pseudo-cyclic oligonucleobases |
| ATE356824T1 (de) | 1999-05-04 | 2007-04-15 | Santaris Pharma As | L-ribo-lna analoge |
| US6083482A (en) | 1999-05-11 | 2000-07-04 | Icn Pharmaceuticals, Inc. | Conformationally locked nucleosides and oligonucleotides |
| US6531591B1 (en) | 1999-07-07 | 2003-03-11 | Exiqon A/S | Synthesis of stable quinone and photoreactive ketone phosphoramidite reagents for solid phase synthesis of photoreactive-oligomer conjugates |
| JP4151751B2 (ja) | 1999-07-22 | 2008-09-17 | 第一三共株式会社 | 新規ビシクロヌクレオシド類縁体 |
| US6677445B1 (en) | 1999-08-27 | 2004-01-13 | Chiron Corporation | Chimeric antisense oligonucleotides and cell transfecting formulations thereof |
| US6187586B1 (en) | 1999-12-29 | 2001-02-13 | Isis Pharmaceuticals, Inc. | Antisense modulation of AKT-3 expression |
| AU5752601A (en) | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
| US6809194B1 (en) | 2000-05-10 | 2004-10-26 | Chiron Corporation | Akt3 inhibitors |
| AU2001271998A1 (en) | 2000-07-13 | 2002-01-30 | The Johns Hopkins University School Of Medicine | Detection and treatment of polycystic kidney disease |
| WO2002018388A1 (fr) | 2000-08-29 | 2002-03-07 | Takeshi Imanishi | Analogues de nucleosides et derives d'oligonucleotides renfermant ces analogues |
| PL366039A1 (en) | 2000-09-02 | 2005-01-24 | Grunenthal Gmbh | Antisense oligonucleotides against vr1 |
| EP1334109B1 (en) | 2000-10-04 | 2006-05-10 | Santaris Pharma A/S | Improved synthesis of purine locked nucleic acid analogues |
| JP2002345489A (ja) | 2000-11-03 | 2002-12-03 | Astrazeneca Ab | 化学物質 |
| ATE287413T1 (de) | 2001-07-12 | 2005-02-15 | Santaris Pharma As | Verfahren zur herstellung des lna phosphoramidite |
| WO2003016492A2 (en) | 2001-08-16 | 2003-02-27 | The Regents Of The University Of Michigan | Adamts13 genes and proteins and variants, and uses thereof |
| US7060809B2 (en) | 2001-09-04 | 2006-06-13 | Exiqon A/S | LNA compositions and uses thereof |
| US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
| WO2003040395A2 (en) | 2001-11-07 | 2003-05-15 | Applera Corporation | Universal nucleotides for nucleic acid analysis |
| JP4383176B2 (ja) | 2002-02-13 | 2009-12-16 | 武 今西 | ヌクレオシド類縁体およびそのヌクレオチド類縁体を含むオリゴヌクレオチド誘導体 |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
| AU2003222743B2 (en) | 2002-05-08 | 2008-12-11 | Roche Innovation Center Copenhagen A/S | Synthesis of locked nucleic acid derivatives |
| AU2003247610A1 (en) | 2002-06-21 | 2004-01-06 | Ptc Therapeutics, Inc. | METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY |
| EP1546369A4 (en) | 2002-08-12 | 2007-01-17 | Univ Michigan | DIAGNOSIS AND TREATMENT OF DISEASES THROUGH DEFECTS IN THE TUBEROUS SKLEROSEPFAD |
| JP2005535318A (ja) | 2002-08-12 | 2005-11-24 | ユニヴェルシテ ドゥ シェルブルック | プレメッセンジャーrnaのスプライス部位選択の再プログラミング方法 |
| US8604183B2 (en) | 2002-11-05 | 2013-12-10 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
| CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2284269B1 (en) | 2002-11-18 | 2017-08-09 | Roche Innovation Center Copenhagen A/S | Antisense design |
| US7217807B2 (en) | 2002-11-26 | 2007-05-15 | Rosetta Genomics Ltd | Bioinformatically detectable group of novel HIV regulatory genes and uses thereof |
| CA2518475C (en) | 2003-03-07 | 2014-12-23 | Alnylam Pharmaceuticals, Inc. | Irna agents comprising asymmetrical modifications |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| AU2004221760B2 (en) | 2003-03-21 | 2010-03-18 | Roche Innovation Center Copenhagen A/S | Short interfering RNA (siRNA) analogues |
| AU2004230927B9 (en) | 2003-04-13 | 2011-12-01 | Enzon Pharmaceuticals, Inc. | Polymeric oligonucleotide prodrugs |
| US20050053981A1 (en) | 2003-09-09 | 2005-03-10 | Swayze Eric E. | Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini |
| US20070009899A1 (en) | 2003-10-02 | 2007-01-11 | Mounts William M | Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases |
| GB0326578D0 (en) | 2003-11-14 | 2003-12-17 | Univ Belfast | Cancer diagnosis and therapy |
| US20050221354A1 (en) | 2004-02-18 | 2005-10-06 | Wyeth | Nucleic acid arrays for monitoring expression profiles of drug target genes |
| CA2566519C (en) | 2004-05-14 | 2020-04-21 | Rosetta Genomics Ltd. | Micrornas and uses thereof |
| US8394947B2 (en) | 2004-06-03 | 2013-03-12 | Isis Pharmaceuticals, Inc. | Positionally modified siRNA constructs |
| US20080299104A1 (en) * | 2004-07-01 | 2008-12-04 | California Institute Of Technology | Methods for detecting agents involved in neuronal apoptosis and compositions thereof |
| US20070087376A1 (en) | 2004-08-30 | 2007-04-19 | Potashkin Judith A | Splice variants of pre-mRNA transcripts as biomarkers in idiopathic neurodegenerative diseases |
| JP2008520244A (ja) | 2004-11-19 | 2008-06-19 | アカディア ファーマシューティカルズ インコーポレイテッド | ホルモン核内受容体のリガンドの同定方法 |
| US8211864B2 (en) | 2005-01-26 | 2012-07-03 | Medical College Of Georgia Research Institute | Compositions and methods for the intracellular disruption of VEGF and VEGFR-2 by intraceptors |
| US7718625B2 (en) | 2005-01-27 | 2010-05-18 | University Of South Florida | Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof |
| WO2007094755A2 (en) | 2005-02-04 | 2007-08-23 | Massachusetts Institute Of Technology | Compositions and methods for modulating cognitive function |
| ES2732071T3 (es) | 2005-04-01 | 2019-11-20 | Univ Florida | Biomarcadores de lesiones hepáticas |
| US20100088778A1 (en) | 2005-06-16 | 2010-04-08 | John Charles Mulley | Methods of Treatment, and Diagnosis of Epilepsy by Detecting Mutations in the SCN1A Gene |
| DK1902141T3 (da) | 2005-06-17 | 2012-05-07 | Baxter Int | ADAMTS 13-holdige sammensætninger med thrombolytisk virkning |
| PT2548560E (pt) | 2005-06-23 | 2015-09-22 | Isis Pharmaceuticals Inc | Composições e métodos de modulação da excisão-união de smn2 |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| WO2007028065A2 (en) | 2005-08-30 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| US20080280848A1 (en) | 2005-10-28 | 2008-11-13 | Volker Patzel | Structures of Active Guide Rna Molecules and Method of Selection |
| WO2007048629A2 (en) | 2005-10-28 | 2007-05-03 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Modulation of rna silencing efficiency by argonaute proteins |
| WO2007053747A2 (en) | 2005-11-01 | 2007-05-10 | President And Fellows Of Harvard College | Modulating endoplasmic reticulum stress in the treatment of tuberous sclerosis |
| WO2007056113A2 (en) | 2005-11-02 | 2007-05-18 | Cylene Pharmaceuticals, Inc. | Methods for targeting quadruplex sequences |
| US7785834B2 (en) | 2005-11-10 | 2010-08-31 | Ercole Biotech, Inc. | Soluble TNF receptors and their use in treatment of disease |
| EP1792981A1 (en) | 2005-12-02 | 2007-06-06 | Humboldt-Universität zu Berlin | Microginin producing proteins and nucleic acids encoding a microginin gene cluster as well as methods for creating microginins |
| EP2422819B1 (en) | 2006-01-26 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Compositions and their uses directed to Huntingtin |
| US7569686B1 (en) | 2006-01-27 | 2009-08-04 | Isis Pharmaceuticals, Inc. | Compounds and methods for synthesis of bicyclic nucleic acid analogs |
| EP2388328A1 (en) | 2006-01-27 | 2011-11-23 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and compositions for the use in modulation of micrornas |
| DE602007009487D1 (de) | 2006-01-27 | 2010-11-11 | Isis Pharmaceutical Inc | 6-modifizierte bicyclische nukleinsäureanaloga |
| US8007790B2 (en) | 2006-04-03 | 2011-08-30 | Stowers Institute For Medical Research | Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases |
| EP2505646A1 (en) | 2006-05-05 | 2012-10-03 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of CRP |
| CA2651453C (en) | 2006-05-11 | 2014-10-14 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| NZ574034A (en) | 2006-06-08 | 2011-09-30 | Amino Up Chemical Co Ltd | SENSE OLIGONUCLEOTIDE CAPABLE OF CONTROLLING THE EXPRESSION OF iNOS AND COMPOSITION COMPRISING THE SAME |
| JPWO2008001778A1 (ja) | 2006-06-27 | 2009-11-26 | 武田薬品工業株式会社 | 遺伝子改変動物およびその用途 |
| CA2993381C (en) | 2006-07-24 | 2020-07-14 | Athena Diagnostics, Inc. | Method of detecting the occurrence of adpkd based on presence of nucleotide sequence alteration in pkd1 |
| EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
| US20090264353A1 (en) | 2007-10-19 | 2009-10-22 | Santaris Pharma A/S | Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease |
| ATE551061T1 (de) | 2006-11-13 | 2012-04-15 | Santaris Pharma As | Lna-nukleosid-phosphoramidate |
| US8293684B2 (en) | 2006-11-29 | 2012-10-23 | Exiqon | Locked nucleic acid reagents for labelling nucleic acids |
| WO2008086807A2 (en) | 2007-01-19 | 2008-07-24 | Exiqon A/S | Mediated cellular delivery of lna oligonucleotides |
| WO2008094945A2 (en) | 2007-01-29 | 2008-08-07 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| ES2385268T3 (es) | 2007-03-09 | 2012-07-20 | Riken | Compuesto que tiene una estructura derivada de mononucleósido o mononucleótido, ácido nucleico, sustancia marcadora y método y kit para la detección de ácido nucleico |
| CL2008000696A1 (es) | 2007-03-09 | 2008-09-12 | Pioneer Hi Bred Int | Polinucleotido aislado que codifica un modificador de un transportador de amonio (amt); casete de expresion y celula huesped que comprende la celula huesped; metodo para modular el amt en plantas. |
| WO2008111908A1 (en) | 2007-03-15 | 2008-09-18 | Jyoti Chattopadhyaya | Five- and six-membered conformationally locked 2',4'- carbocyclic ribo-thymidines for the treatment of infections and cancer |
| EP2149605B1 (en) | 2007-03-22 | 2013-07-03 | Santaris Pharma A/S | Short RNA antagonist compounds for the modulation of target mRNA |
| DK2170917T3 (da) | 2007-05-30 | 2012-10-08 | Isis Pharmaceuticals Inc | N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro |
| WO2008154401A2 (en) | 2007-06-08 | 2008-12-18 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| AU2008272918B2 (en) | 2007-07-05 | 2012-09-13 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| EP2188298B1 (en) | 2007-08-15 | 2013-09-18 | Isis Pharmaceuticals, Inc. | Tetrahydropyran nucleic acid analogs |
| NZ584793A (en) | 2007-10-26 | 2012-05-25 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2009064920A2 (en) | 2007-11-13 | 2009-05-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| EP2229186A2 (en) | 2007-12-04 | 2010-09-22 | Alnylam Pharmaceuticals Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| US8846386B2 (en) | 2007-12-18 | 2014-09-30 | University Of Kentucky Research Foundation | sVEGFR-2 and its role in lymphangiogenesis modulation |
| JP5608863B2 (ja) | 2007-12-28 | 2014-10-15 | 公立大学法人横浜市立大学 | 新生児期〜乳児期発症の難治性てんかんの検出方法 |
| EP2265627A2 (en) | 2008-02-07 | 2010-12-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| JP5409658B2 (ja) | 2008-03-13 | 2014-02-05 | セレラ コーポレーション | 静脈血栓症に関連した遺伝子多型、その検出方法および使用 |
| US8846639B2 (en) | 2008-04-04 | 2014-09-30 | Isis Pharmaceutical, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| US9290534B2 (en) | 2008-04-04 | 2016-03-22 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside |
| WO2009149921A2 (en) | 2008-06-11 | 2009-12-17 | Bionucleon S.R.L. | Inhibition of hrp-3 using modified oligonucleotides |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| JP2012507989A (ja) * | 2008-11-07 | 2012-04-05 | センター ホスピタライヤー ユニヴェルシテール サント−ジュスティーヌ | Syngap1機能不全ならびに精神遅滞の診断および治療用途でのその使用 |
| WO2010065671A2 (en) | 2008-12-04 | 2010-06-10 | Curna, Inc. | Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf |
| WO2010080509A1 (en) | 2008-12-19 | 2010-07-15 | Philadelphia Health And Education Corporation | Compositions and methods for diminishing viral infection and inflammation associated with viral infection |
| US20100166784A1 (en) | 2008-12-30 | 2010-07-01 | The Washington University | Method and compositions for modulating th17 cell development |
| DK2381965T3 (da) | 2009-01-14 | 2020-07-27 | Univ Drexel | Modulation af præ-mrna under anvendelse af splejsningsmodulerende oligonukleotider som terapeutiske midler til behandling af sygdom |
| EP2427554B1 (en) | 2009-05-08 | 2016-11-16 | CuRNA, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
| KR20220084437A (ko) | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법 |
| EP2275545A1 (en) | 2009-07-16 | 2011-01-19 | Julius-Maximilians-Universität Würzburg | Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| WO2011020118A1 (en) | 2009-08-14 | 2011-02-17 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| EP3135772B1 (en) | 2009-09-08 | 2019-10-23 | Laboratory Corporation of America Holdings | Method for diagnosing autism spectrum disorders |
| WO2011032034A2 (en) | 2009-09-10 | 2011-03-17 | University Of Idaho | Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids |
| AU2010312751B2 (en) | 2009-10-29 | 2014-07-24 | Osaka University | Bridged artificial nucleoside and nucleotide |
| HUE040445T2 (hu) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antiszensz molekulák és eljárások betegségek kezelésére |
| ES2574625T3 (es) | 2009-11-25 | 2016-06-21 | Elitechgroup B.V. | Antagonistas de miARN de oligonucleótido conjugado con aglutinante del surco menor (MGB) |
| US8779118B2 (en) | 2010-01-11 | 2014-07-15 | Isis Pharmaceuticals, Inc. | Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom |
| ES2733708T3 (es) | 2010-02-08 | 2019-12-02 | Ionis Pharmaceuticals Inc | Reducción selectiva de variantes alélicas |
| WO2011115818A1 (en) | 2010-03-17 | 2011-09-22 | Isis Pharmaceuticals, Inc. | 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom |
| EP2550361B1 (en) | 2010-03-25 | 2017-02-08 | The J. David Gladstone Institutes | Compositions and methods for treating neurological disorders |
| US9006415B2 (en) | 2010-04-06 | 2015-04-14 | Massachusetts Institute Of Technology | Targeted delivery of nucleic acids |
| US20110269735A1 (en) | 2010-04-19 | 2011-11-03 | Celera Corporation | Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof |
| KR20130103662A (ko) | 2010-04-19 | 2013-09-24 | 엔라이프 떼라퓨틱스, 에스.엘. | 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법 |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| WO2011139695A2 (en) | 2010-04-28 | 2011-11-10 | Isis Pharmaceuticals, Inc. | Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom |
| CA2799944A1 (en) | 2010-05-20 | 2011-11-24 | University Of Rochester | Methods and compositions related to modulating autophagy |
| US8957200B2 (en) | 2010-06-07 | 2015-02-17 | Isis Pharmaceuticals, Inc. | Bicyclic nucleosides and oligomeric compounds prepared therefrom |
| US8846637B2 (en) | 2010-06-08 | 2014-09-30 | Isis Pharmaceuticals, Inc. | Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2011159836A2 (en) | 2010-06-15 | 2011-12-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating interaction between proteins and target nucleic acids |
| CN109112126B (zh) * | 2010-06-23 | 2024-06-14 | 库尔纳公司 | 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病 |
| CN103180445B (zh) | 2010-10-22 | 2018-02-16 | 库尔纳公司 | 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病 |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| US20130309246A1 (en) | 2011-02-02 | 2013-11-21 | The Trustees Of Princeton University | Jagged1 as a marker and therapeutic target for breast cancer bone metastasis |
| US9644035B2 (en) | 2011-04-08 | 2017-05-09 | Omeros Corporation | Methods for treating conditions associated with MASP-2 dependent complement activation |
| US9957558B2 (en) | 2011-04-28 | 2018-05-01 | Life Technologies Corporation | Methods and compositions for multiplex PCR |
| WO2013081755A1 (en) | 2011-11-29 | 2013-06-06 | Life Technologies Corporation | Methods and compositions for multiplex pcr |
| US9012425B2 (en) | 2011-06-10 | 2015-04-21 | Inserm (Institute National De La Sante Et De La Recherche Medicale) | Methods for the treatment of Leber congenital amaurosis |
| WO2012178146A1 (en) | 2011-06-24 | 2012-12-27 | Isis Pharmaceuticals, Inc. | Compositions and methods for treatment of spinal muscular atrophy |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| US8592156B2 (en) | 2011-08-08 | 2013-11-26 | Roche Molecular Systems, Inc. | Predicting response to anti-CD20 therapy in DLBCL patients |
| US10023861B2 (en) | 2011-08-29 | 2018-07-17 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| ES2646716T3 (es) | 2011-09-05 | 2017-12-15 | Stichting Katholieke Universiteit | Oligonucleótidos antisentido para el tratamiento de amaurosis congénita de Leber |
| KR101991980B1 (ko) | 2011-09-06 | 2019-06-21 | 큐알엔에이, 인크. | 소형 분자로 전압-개폐된 나트륨 채널 (SCNxA)의 알파 아단위에 관련된 질환의 치료 |
| US9453261B2 (en) | 2011-09-20 | 2016-09-27 | The George Washington University | Alternative splicing variants of genes associated with prostate cancer risk and survival |
| US20140343127A1 (en) | 2011-11-11 | 2014-11-20 | Santaris Pharma A/S a corporation | Compounds for the modulation of smn2 splicing |
| US9534222B2 (en) | 2011-11-15 | 2017-01-03 | University Of Utah Research Foundation | Morpholinos, morpholino upregulating, and associated methods |
| IL266882B2 (en) | 2012-01-11 | 2025-11-01 | Ionis Pharmaceuticals Inc | Compositions and methods for modulation of ikbkap splicing |
| EP3330278A1 (en) | 2012-02-08 | 2018-06-06 | Ionis Pharmaceuticals, Inc. | Modulation of rna by repeat targeting |
| EP3406252B1 (en) | 2012-02-10 | 2020-05-13 | PTC Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| US8846885B2 (en) | 2012-02-17 | 2014-09-30 | Ajinomoto Co., Inc. | Oligonucleotide with protected base |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| EP3336189A1 (en) | 2012-04-20 | 2018-06-20 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| US20140378526A1 (en) | 2012-05-11 | 2014-12-25 | City Of Hope | Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations |
| EP2852604B1 (en) | 2012-05-22 | 2017-04-12 | Idenix Pharmaceuticals LLC | 3',5'-cyclic phosphoramidate prodrugs for hcv infection |
| WO2013177195A1 (en) | 2012-05-22 | 2013-11-28 | Idenix Pharmaceuticals, Inc. | 3',5'-cyclic phosphate prodrugs for hcv infection |
| EP3536787A1 (en) | 2012-06-08 | 2019-09-11 | Translate Bio, Inc. | Nuclease resistant polynucleotides and uses thereof |
| DK2872147T3 (da) | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| US8729263B2 (en) | 2012-08-13 | 2014-05-20 | Novartis Ag | 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions |
| JP2015529073A (ja) | 2012-08-20 | 2015-10-05 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物可逆性基を有するポリヌクレオチド |
| AU2013319788B2 (en) | 2012-09-24 | 2019-04-11 | Yissum Reasearch Development Company of the Hebrew University Of Jerusalem Ltd. | Restoration of the CFTR function by splicing modulation |
| WO2014049536A2 (en) | 2012-09-25 | 2014-04-03 | Universidade De Lisboa | Drug targets for cystic fibrosis and other conditions |
| WO2014052638A1 (en) | 2012-09-27 | 2014-04-03 | Idenix Pharmaceuticals, Inc. | Esters and malonates of sate prodrugs |
| UA116639C2 (uk) | 2012-10-09 | 2018-04-25 | Рег'Юлес Терап'Ютікс Інк. | Способи лікування синдрому альпорта |
| US9029335B2 (en) | 2012-10-16 | 2015-05-12 | Isis Pharmaceuticals, Inc. | Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2014066915A2 (en) | 2012-10-26 | 2014-05-01 | Smith Larry J | Methods and compositions to produce ss-rnai activity with enhanced potency |
| WO2014078749A1 (en) | 2012-11-15 | 2014-05-22 | The Regents Of The University Of California | Splice modulating oligonucleotides that inhibit cancer |
| US9211300B2 (en) | 2012-12-19 | 2015-12-15 | Idenix Pharmaceuticals Llc | 4′-fluoro nucleosides for the treatment of HCV |
| CN104004826B (zh) | 2013-01-07 | 2016-03-02 | 赵晨 | 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用 |
| AU2014206668A1 (en) | 2013-01-18 | 2015-08-06 | Anna Mary Rose | Therapeutics and diagnostics based on minisatellite repeat element 1 (MSR1) |
| EP2951304B1 (en) | 2013-02-04 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2014126229A1 (ja) | 2013-02-18 | 2014-08-21 | 塩野義製薬株式会社 | 含窒素複素環構造を有するヌクレオシド及びヌクレオチド |
| WO2014137930A1 (en) | 2013-03-04 | 2014-09-12 | Idenix Pharmaceuticals, Inc. | Thiophosphate nucleosides for the treatment of hcv |
| WO2014137926A1 (en) | 2013-03-04 | 2014-09-12 | Idenix Pharmaceuticals, Inc. | 3'-deoxy nucleosides for the treatment of hcv |
| EP2981542B1 (en) | 2013-04-01 | 2021-09-15 | Idenix Pharmaceuticals LLC | 2',4'-fluoro nucleosides for the treatment of hcv |
| WO2014172698A1 (en) | 2013-04-19 | 2014-10-23 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation nucleic acids through nonsense mediated decay |
| PL2992098T3 (pl) | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| US9549909B2 (en) | 2013-05-03 | 2017-01-24 | The Katholieke Universiteit Leuven | Method for the treatment of dravet syndrome |
| WO2014198890A1 (en) | 2013-06-13 | 2014-12-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for the treatment of erythropoietic protoporphyria |
| WO2014201413A1 (en) | 2013-06-14 | 2014-12-18 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating non-coding rna |
| CA2913785C (en) | 2013-06-25 | 2021-08-31 | F. Hoffmann-La Roche Ag | Compounds for treating spinal muscular atrophy |
| WO2015023939A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin |
| EP3033423A4 (en) | 2013-08-16 | 2017-04-26 | Rana Therapeutics Inc. | Epigenetic regulators of frataxin |
| CA2921556A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating rna |
| JP2016531570A (ja) | 2013-08-16 | 2016-10-13 | ラナ セラピューティクス インコーポレイテッド | ユークロマチン領域を標的とするオリゴヌクレオチド |
| CA2915764A1 (en) | 2013-08-19 | 2015-02-26 | F. Hoffmann-La Roche Ag | Screening method |
| WO2015035091A1 (en) * | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| AU2014317961B2 (en) | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| DK3044315T3 (da) | 2013-09-11 | 2019-05-20 | Synthena Ag | Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom |
| EP3071685B1 (en) | 2013-11-21 | 2026-07-29 | Memorial Sloan-Kettering Cancer Center | Specification of functional cranial placode derivatives from human pluripotent stem cells |
| US10119168B2 (en) | 2014-03-12 | 2018-11-06 | The Brigham And Women's Hospital, Inc. | Methods for the treatment of kidney fibrosis |
| US20170044540A1 (en) | 2014-04-22 | 2017-02-16 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| EP4039807A1 (en) | 2014-06-10 | 2022-08-10 | Erasmus University Rotterdam Medical Center | Antisense oligonucleotides useful in treatment of pompe disease |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| GB201411468D0 (en) | 2014-06-27 | 2014-08-13 | Univ Edinburgh | Novel treatment for endometriosis |
| GB2547586A (en) | 2014-08-20 | 2017-08-23 | Lifesplice Pharma Llc | Splice modulating oligonucleotides and methods of use thereof(In the PCT request) |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| US20170224758A1 (en) | 2014-10-17 | 2017-08-10 | The Broad Institute, Inc. | Compositions and methods of treating muscular dystrophy |
| AU2015346042A1 (en) | 2014-11-14 | 2017-04-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of proteins |
| GB201421379D0 (en) | 2014-12-02 | 2015-01-14 | Isis Innovation Ltd And Medical Res Council | Molecule |
| EP3247328B1 (en) | 2015-01-21 | 2026-01-07 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| EP3256126B1 (en) | 2015-02-09 | 2024-03-27 | F. Hoffmann-La Roche AG | Compounds for the treatment of cancer |
| US9840709B2 (en) | 2015-02-20 | 2017-12-12 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting genes associated with cystic fibrosis |
| MX2017011004A (es) | 2015-02-27 | 2018-02-09 | Sarepta Therapeutics Inc | Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. |
| CN107429250B (zh) | 2015-04-03 | 2022-03-01 | Ionis制药公司 | 用于调节tmprss6表达的化合物和方法 |
| EP3310169B1 (en) | 2015-05-30 | 2023-05-17 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| US20180265911A1 (en) | 2015-09-24 | 2018-09-20 | The Trustees Of The University Of Pennsylvania | Triptycene derivatives for nucleic acid junction stabilization |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| GB2546719B (en) | 2015-10-09 | 2021-07-14 | Univ Southampton | Modulation of gene expression and screening for deregulated protein expression |
| EP3183347A4 (en) | 2015-10-17 | 2018-04-18 | Lifesplice Pharma LLC | Splice modulating oligonucleotides and methods of use thereof |
| TW201729807A (zh) | 2015-10-30 | 2017-09-01 | Ptc治療公司 | 用於治療癲癇的方法 |
| EP3389671A4 (en) | 2015-12-14 | 2019-07-17 | Cold Spring Harbor Laboratory | ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| JP7049247B2 (ja) | 2015-12-14 | 2022-04-06 | コールド スプリング ハーバー ラボラトリー | 腎臓病の処置のための組成物と方法 |
| JP7036723B2 (ja) | 2015-12-14 | 2022-03-15 | コールド スプリング ハーバー ラボラトリー | 眼疾患の処置のための組成物および方法 |
| WO2017106283A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
| CA3005131A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of tuberous sclerosis complex |
| CA3005247A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of polycystic kidney disease |
| ES2903394T3 (es) | 2015-12-14 | 2022-04-01 | Cold Spring Harbor Laboratory | Composiciones para el tratamiento de la retinitis pigmentosa 13 |
| WO2017106382A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of central nervous system diseases |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| WO2017180587A2 (en) | 2016-04-11 | 2017-10-19 | Obsidian Therapeutics, Inc. | Regulated biocircuit systems |
| EP3481857B1 (en) | 2016-07-06 | 2026-02-11 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of pain related disorders |
| US20190218255A1 (en) | 2016-09-16 | 2019-07-18 | Olipass Corporation | Scn9a antisense oligonucleotides |
| CN117821509A (zh) | 2017-04-03 | 2024-04-05 | 编码治疗公司 | 组织选择性转基因表达 |
| MX2019012285A (es) | 2017-04-13 | 2020-11-06 | Ovid Therapeutics Inc | Metodos de tratamiento de encefalopatias del desarrollo. |
| CA3062247A1 (en) | 2017-05-09 | 2018-11-15 | Zogenix International Limited | Methods of treating doose syndrome using fenfluramine |
| WO2018213491A1 (en) | 2017-05-16 | 2018-11-22 | Praxis Precision Medicines, Inc. | Methods of treating epilepsy and neurodevelopmental disorders |
| KR20260009385A (ko) | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| LT3700570T (lt) | 2017-10-23 | 2025-04-10 | Stoke Therapeutics, Inc. | Priešprasmiai oligomerai, skirti būklių ir ligų, pagrįstų nonsens kodono nulemta mrnr degradacija, gydymui |
| CN118345115A (zh) | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| CA3095125A1 (en) | 2018-03-27 | 2019-10-03 | Factor Bioscience Inc. | Nucleic acid-based therapeutics |
| EP3781174A4 (en) | 2018-04-09 | 2022-01-26 | Allen Institute | RESCUE OF VOLTAGE CONTROLLED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS |
| WO2019227096A1 (en) | 2018-05-25 | 2019-11-28 | Rogcon U.R., Inc. | Dynamic clamps and methods of use thereof |
| WO2019236750A2 (en) | 2018-06-05 | 2019-12-12 | Tufts Medical Center, Inc. | Targeted treatment of autism spectrum disorder and other neurological or psychiatric disorders |
| CN112513273A (zh) | 2018-06-22 | 2021-03-16 | 豪夫迈·罗氏有限公司 | 用于调节scn9a表达的寡核苷酸 |
| CA3110211A1 (en) | 2018-08-20 | 2020-02-27 | Rogcon, Inc. | Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies |
| WO2020069194A1 (en) | 2018-09-26 | 2020-04-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
-
2016
- 2016-12-14 CN CN202211146489.4A patent/CN116059236A/zh active Pending
- 2016-12-14 CN CN201680081827.7A patent/CN109312343B/zh not_active Expired - Fee Related
- 2016-12-14 US US16/062,286 patent/US11083745B2/en active Active
- 2016-12-14 JP JP2018529250A patent/JP7049248B2/ja not_active Expired - Fee Related
- 2016-12-14 KR KR1020187019006A patent/KR102604132B1/ko active Active
- 2016-12-14 EP EP16876616.0A patent/EP3390636B1/en active Active
- 2016-12-14 WO PCT/US2016/066708 patent/WO2017106377A1/en not_active Ceased
- 2016-12-14 AU AU2016370653A patent/AU2016370653A1/en not_active Abandoned
- 2016-12-14 EP EP21167500.4A patent/EP3933041B1/en active Active
- 2016-12-14 CA CA3005256A patent/CA3005256A1/en active Pending
- 2016-12-14 SG SG11201804443UA patent/SG11201804443UA/en unknown
- 2016-12-14 ES ES16876616T patent/ES2882500T3/es active Active
-
2018
- 2018-05-08 IL IL259222A patent/IL259222A/en unknown
-
2021
- 2021-06-23 US US17/356,147 patent/US20220088058A1/en not_active Abandoned
-
2022
- 2022-02-01 JP JP2022013860A patent/JP2022062140A/ja active Pending
-
2023
- 2023-04-11 AU AU2023202216A patent/AU2023202216A1/en not_active Abandoned
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140128449A1 (en) | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
Non-Patent Citations (3)
| Title |
|---|
| HUMAN MUTATION,2005年,Vol.25,pp.535-542(& S1-S7) |
| PLOS ONE,2012年,Vol.7, No.7,e41802, pp.1-11 |
| PNAS,1996年,Vol.93,pp.12840-12844 |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3390636A4 (en) | 2019-08-14 |
| KR102604132B1 (ko) | 2023-11-17 |
| KR20180093977A (ko) | 2018-08-22 |
| US20180369275A1 (en) | 2018-12-27 |
| WO2017106377A1 (en) | 2017-06-22 |
| AU2023202216A1 (en) | 2023-07-06 |
| JP2019501892A (ja) | 2019-01-24 |
| JP2022062140A (ja) | 2022-04-19 |
| IL259222A (en) | 2018-07-31 |
| CN109312343A (zh) | 2019-02-05 |
| EP3933041B1 (en) | 2024-01-31 |
| EP3933041A1 (en) | 2022-01-05 |
| CN116059236A (zh) | 2023-05-05 |
| US11083745B2 (en) | 2021-08-10 |
| US20220088058A1 (en) | 2022-03-24 |
| AU2016370653A1 (en) | 2018-06-21 |
| CN109312343B (zh) | 2022-09-27 |
| CA3005256A1 (en) | 2017-06-22 |
| EP3390636A1 (en) | 2018-10-24 |
| SG11201804443UA (en) | 2018-06-28 |
| ES2882500T3 (es) | 2021-12-02 |
| EP3390636B1 (en) | 2021-05-19 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022062140A (ja) | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー | |
| US11873490B2 (en) | Antisense oligomers for treatment of conditions and diseases | |
| JP7049247B2 (ja) | 腎臓病の処置のための組成物と方法 | |
| JP2022106803A (ja) | アラジール症候群の処置のためのアンチセンスオリゴマー | |
| JP7054675B2 (ja) | 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法 | |
| JP2022046723A (ja) | 多発性嚢胞腎の処置のためのアンチセンスオリゴマー | |
| JP7660515B2 (ja) | 病態及び疾患の治療用アンチセンスオリゴマー | |
| US20240254488A1 (en) | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases | |
| EP4532723A2 (en) | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases | |
| HK40062703A (en) | Antisense oligomers for treatment of autosomal dominant retardation | |
| HK1262955B (en) | Antisense oligomers for treatment of dravet syndrome | |
| CN120641565A (zh) | 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚物 | |
| HK40064034A (en) | Antisense oligomers for treatment of conditions and diseases | |
| HK40105710A (en) | Antisense oligomers for treatment of conditions and diseases |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20180801 |
|
| RD03 | Notification of appointment of power of attorney |
Free format text: JAPANESE INTERMEDIATE CODE: A7423 Effective date: 20190531 |
|
| RD04 | Notification of resignation of power of attorney |
Free format text: JAPANESE INTERMEDIATE CODE: A7424 Effective date: 20190603 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20191213 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20191213 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200819 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20200819 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20201119 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210219 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210519 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20211001 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20220201 |
|
| C60 | Trial request (containing other claim documents, opposition documents) |
Free format text: JAPANESE INTERMEDIATE CODE: C60 Effective date: 20220201 |
|
| A911 | Transfer to examiner for re-examination before appeal (zenchi) |
Free format text: JAPANESE INTERMEDIATE CODE: A911 Effective date: 20220214 |
|
| C21 | Notice of transfer of a case for reconsideration by examiners before appeal proceedings |
Free format text: JAPANESE INTERMEDIATE CODE: C21 Effective date: 20220215 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20220301 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20220325 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 7049248 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| LAPS | Cancellation because of no payment of annual fees |