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)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Analytical Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (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)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or 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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3933041B1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7049248B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR102604132B1 (cg-RX-API-DMAC7.html) |
| CN (2) | CN109312343B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2016370653A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3005256A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2882500T3 (cg-RX-API-DMAC7.html) |
| IL (1) | IL259222A (cg-RX-API-DMAC7.html) |
| SG (1) | SG11201804443UA (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017106377A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (28)
| 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 |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) * | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| CN111278991B (zh) * | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| RS66633B1 (sr) * | 2017-10-23 | 2025-08-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk |
| JP7401432B2 (ja) * | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス, インコーポレイテッド | 操作されたdna結合タンパク質 |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| US11939582B2 (en) | 2018-08-20 | 2024-03-26 | Rogcon, Inc. | Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies |
| MX2021003567A (es) * | 2018-09-26 | 2021-08-24 | Univ Case Western Reserve | Métodos y composiciones para aumentar la expresión de proteínas y/o tratar un trastorno por haploinsuficiencia. |
| 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 |
| WO2020154462A1 (en) * | 2019-01-23 | 2020-07-30 | Rogcon U.R., Inc. | Antisense oligonucleotides targeting scn2a retained introns |
| WO2020176776A1 (en) * | 2019-02-27 | 2020-09-03 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| BR112021021384A2 (pt) * | 2019-04-26 | 2021-12-21 | Stoke Therapeutics Inc | Métodos e composições para modular splicing de íntrons alternativos |
| WO2021034985A1 (en) * | 2019-08-19 | 2021-02-25 | Stoke Therapeutics, Inc. | Compositions and methods for modulating splicing and protein expression |
| US11618900B2 (en) | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| WO2021113755A2 (en) * | 2019-12-04 | 2021-06-10 | Pai Athma A | Anti-slc6a1 oligonucleotides and related methods |
| WO2021113541A1 (en) * | 2019-12-06 | 2021-06-10 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| EP4110918A4 (en) * | 2020-02-28 | 2024-04-03 | The University of Chicago | METHODS AND COMPOSITIONS COMPRISING TRANS-ACTING TRANSLATION ACTIVATORS |
| 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 |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| 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 |
| WO2023163801A1 (en) * | 2022-02-24 | 2023-08-31 | Q-State Biosciences, Inc. | Therapeutics for syngap haploinsufficiency |
| EP4627084A1 (en) * | 2022-12-01 | 2025-10-08 | Camp4 Therapeutics Corporation | Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas |
| WO2024145496A2 (en) * | 2022-12-28 | 2024-07-04 | The University Of Chicago | Splice-switching oligonucleotides for treating syngap1-associated disorders |
| WO2024229421A1 (en) * | 2023-05-03 | 2024-11-07 | Stoke Therapeutics, Inc. | Compounds and methods for treating human subjects |
| WO2025090633A1 (en) * | 2023-10-23 | 2025-05-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies |
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 |
| US7101993B1 (en) | 1990-01-11 | 2006-09-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides containing 2′-O-modified purines |
| US5914396A (en) | 1990-01-11 | 1999-06-22 | Isis Pharmaceuticals, Inc. | 2'-O-modified nucleosides and phosphoramidites |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| EP1443051A3 (en) | 1990-08-13 | 2005-08-17 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| AU4684993A (en) | 1992-07-23 | 1994-02-14 | Isis Pharmaceuticals, Inc. | Novel 2'-o-alkyl nucleosides and phosphoramidites processes for the preparation and uses thereof |
| NZ256018A (en) | 1992-09-25 | 1997-07-27 | Rhone Poulenc Rorer Sa | Recombinant adenoviral vectors and their use in directing expression and transcription of selected nucleotide sequences in cells of the central nervous system |
| AU6786594A (en) | 1993-05-11 | 1994-12-12 | University Of North Carolina At Chapel Hill, The | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
| JP3715314B2 (ja) | 1993-12-24 | 2005-11-09 | ユニバーシティ オブ ウェールズ カレッジ オブ メディスン | 結節硬化症2遺伝子及びその利用 |
| 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 |
| CN1214688A (zh) | 1996-02-14 | 1999-04-21 | 伊希斯药物有限公司 | 糖修饰的缺口寡核苷酸 |
| 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 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| 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 |
| JP2002511750A (ja) | 1997-06-03 | 2002-04-16 | マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ | in vivoにおいて内皮細胞に異種DNA配列を発現させる能力のある制御配列とその使用法 |
| 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 |
| EP2253639A1 (en) | 1997-09-12 | 2010-11-24 | 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 |
| EP1152009B2 (en) | 1999-02-12 | 2017-09-06 | Daiichi Sankyo Company, Limited | Novel nucleosides and oligonucleotide analogues |
| CA2368135C (en) | 1999-03-18 | 2010-06-08 | Exiqon A/S | Xylo-lna analogues |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| US6639059B1 (en) | 1999-03-24 | 2003-10-28 | Exiqon A/S | 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 |
| CN102180924A (zh) | 1999-05-04 | 2011-09-14 | 桑塔里斯制药公司 | L-核糖-lna类似物 |
| 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 |
| WO2001083740A2 (en) | 2000-05-04 | 2001-11-08 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| US6809194B1 (en) | 2000-05-10 | 2004-10-26 | Chiron Corporation | Akt3 inhibitors |
| CA2697031C (en) | 2000-07-13 | 2017-10-31 | The Johns Hopkins University School Of Medicine | Detection and treatment of polycystic kidney disease |
| EP1314734A1 (en) | 2000-08-29 | 2003-05-28 | Takeshi Imanishi | Novel nucleoside analogs and oligonucleotide derivatives containing these analogs |
| AU2001295531A1 (en) | 2000-09-02 | 2002-03-13 | Grünenthal GmbH | Antisense oligonucleotides against vanilloid receptor 1 |
| ATE325806T1 (de) | 2000-10-04 | 2006-06-15 | Santaris Pharma As | Verbesserte synthese von purin-blockierten nukleinsäure-analoga |
| JP2002345489A (ja) | 2000-11-03 | 2002-12-03 | Astrazeneca Ab | 化学物質 |
| DE60202681T2 (de) | 2001-07-12 | 2006-01-12 | Santaris Pharma A/S | Verfahren zur herstellung des lna phosphoramidite |
| AU2002336367A1 (en) | 2001-08-16 | 2003-03-03 | The Regents Of The University Of Michigan | Adamts13 genes and proteins and variants, and uses thereof |
| WO2003020739A2 (en) | 2001-09-04 | 2003-03-13 | Exiqon A/S | Novel 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 |
| DK2354148T3 (da) | 2002-02-13 | 2013-10-14 | Takeshi Imanishi | Nukleosidanaloger og oligonukleotiderivate omfattende nukleotidanalog deraf |
| ES2290448T3 (es) | 2002-05-08 | 2008-02-16 | Santaris Pharma A/S | Sistesis de derivados de acidos nucleicos bloqueados. |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma 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 |
| CA2495085A1 (en) | 2002-08-12 | 2004-02-19 | The Regents Of The University Of Michigan | Diagnosis and treatment of tuberous sclerosis |
| EP1529105B1 (en) | 2002-08-12 | 2012-10-03 | Société de commercialisation des produits de la recherche appliquée SOCPRA Sciences Santé et Humaines s.e.c. | Methods to reprogram splice site selection in pre-messenger rnas |
| AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| WO2004044139A2 (en) | 2002-11-05 | 2004-05-27 | Isis Parmaceuticals, Inc. | Modified oligonucleotides for use in rna interference |
| DK1569661T3 (da) | 2002-11-18 | 2010-01-11 | Santaris Pharma As | Antisense design |
| US7217807B2 (en) | 2002-11-26 | 2007-05-15 | Rosetta Genomics Ltd | Bioinformatically detectable group of novel HIV regulatory genes and uses thereof |
| ATE479752T1 (de) | 2003-03-07 | 2010-09-15 | Alnylam Pharmaceuticals Inc | Therapeutische zusammensetzungen |
| 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 |
| DK1606406T4 (da) | 2003-03-21 | 2013-12-16 | Santaris Pharma As | Short Interfering RNA (siRNA) Analogues |
| FI20051017A7 (fi) | 2003-04-13 | 2005-11-28 | Enzon Pharmaceutivals Inc | Polymeeriset oligonukleotidiaihiolääkkeet |
| 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 |
| IL179285A (en) | 2004-05-14 | 2011-04-28 | 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 |
| CA2588646A1 (en) | 2004-11-19 | 2006-05-26 | Acadia Pharmaceuticals Inc. | Methods to identify ligands of hormone nuclear receptors |
| WO2006081311A2 (en) | 2005-01-26 | 2006-08-03 | 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 |
| US20100150839A1 (en) | 2005-02-04 | 2010-06-17 | Massachusetts Institute Of Technology | Compositions and Methods for Modulating Cognitive Function |
| AU2006232370B2 (en) | 2005-04-01 | 2011-10-06 | Banyan Biomarkers | Biomakers of liver injury |
| NZ564892A (en) * | 2005-06-16 | 2011-10-28 | Bionomics Ltd | Methods of treatment, and diagnosis of epilepsy by detecting a D674G mutation in the SCN1A gene |
| CA2606351C (en) | 2005-06-17 | 2016-12-13 | Baxter International Inc. | Adamts13-comprising compositions having thrombolytic activity |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| EP1937312B1 (en) | 2005-08-30 | 2016-06-29 | Ionis 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 |
| EP2161038B1 (en) | 2006-01-26 | 2013-12-25 | Isis 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 |
| CN102908630B (zh) | 2006-01-27 | 2014-11-19 | Isis制药公司 | 6-修饰的双环核酸类似物 |
| JP5213723B2 (ja) | 2006-01-27 | 2013-06-19 | アイシス ファーマシューティカルズ, インコーポレーテッド | マイクロrnaの調節に使用するためのオリゴマー化合物及び組成物 |
| 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 |
| US20090292006A1 (en) | 2006-05-05 | 2009-11-26 | Sanjay Bhanot | Compounds and methods for modulating expression of dgat2 |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| JP5441688B2 (ja) | 2006-05-11 | 2014-03-12 | アイシス ファーマシューティカルズ, インコーポレーテッド | 5’修飾二環式核酸類似体 |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| AU2007255732B2 (en) | 2006-06-08 | 2014-04-03 | Amino Up Chemical Co., Ltd. | Sense oligonucleotide capable of controlling the expression of iNOS and composition comprising the same |
| EP2034018A4 (en) | 2006-06-27 | 2010-02-10 | Takeda Pharmaceutical | GENETICALLY MODIFIED ANIMAL AND ITS USE |
| CA3081362C (en) | 2006-07-24 | 2024-04-09 | Athena Diagnostics, Inc. | Method of detecting the occurrence of adpkd based on presence of nucleotide sequence alteration in pkd1 |
| WO2008049085A1 (en) | 2006-10-18 | 2008-04-24 | Isis 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 |
| WO2007054100A2 (en) | 2006-11-13 | 2007-05-18 | Santaris Pharma A/S | Lna nucleoside phosphoramidates |
| 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 |
| EP2641971A1 (en) | 2007-01-29 | 2013-09-25 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| KR101551985B1 (ko) | 2007-03-09 | 2015-09-09 | 리가가쿠 겐큐쇼 | 모노뉴클레오시드 또는 모노뉴클레오티드로부터 유도되는 구조를 갖는 화합물, 핵산, 표지 물질, 핵산 검출 방법 및 키트 |
| AR065648A1 (es) | 2007-03-09 | 2009-06-24 | Pioneer Hi Bred Int | Identificacion de transportadores de amonio (amts) para la regulacion del metabolismo de nitrogeno en plantas |
| EP2134736A4 (en) | 2007-03-15 | 2013-05-29 | Jyoti Chattopadhyaya | FIVE- AND SIX-PIECE CONFORMATIVELY FIXED 2 ', 4'-CARBOCYCLIC RIBOTHYMIDINE 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 |
| ES2386492T3 (es) | 2007-06-08 | 2012-08-21 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos carbocíclicos |
| ES2376507T5 (es) | 2007-07-05 | 2015-08-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos 6-disustituidos |
| EP2188298B1 (en) | 2007-08-15 | 2013-09-18 | Isis Pharmaceuticals, Inc. | Tetrahydropyran nucleic acid analogs |
| ES2564563T3 (es) | 2007-10-26 | 2016-03-23 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar trastornos del músculo |
| EP2219680A2 (en) | 2007-11-13 | 2010-08-25 | 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 |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | 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 |
| US8530640B2 (en) | 2008-02-07 | 2013-09-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| US8071291B2 (en) | 2008-03-13 | 2011-12-06 | Celera Corporation | Genetic polymorphisms associated with venous thrombosis, methods of detection and uses thereof |
| EP2274423A2 (en) | 2008-04-04 | 2011-01-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| DK2285819T3 (da) | 2008-04-04 | 2013-12-02 | Isis Pharmaceuticals Inc | Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider |
| AU2009256906A1 (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 |
| EP2356129B1 (en) | 2008-09-24 | 2013-04-03 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| WO2010051632A1 (en) * | 2008-11-07 | 2010-05-14 | Centre Hospitalier Universitaire Sainte-Justine | Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation |
| US8927511B2 (en) | 2008-12-04 | 2015-01-06 | 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 |
| WO2010083338A2 (en) | 2009-01-14 | 2010-07-22 | Philadelphia Health And Education Corporation | Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease |
| JP5931720B2 (ja) | 2009-05-08 | 2016-06-08 | クルナ・インコーポレーテッド | Dmdファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 |
| JP5707396B2 (ja) | 2009-06-17 | 2015-04-30 | アイシス ファーマシューティカルズ, インコーポレーテッド | 被験体におけるsmn2スプライシングのモジュレーションのための組成物および方法 |
| 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 |
| US9012421B2 (en) | 2009-08-06 | 2015-04-21 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| US20120252877A1 (en) | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| JP2013503646A (ja) | 2009-09-08 | 2013-02-04 | ラボラトリー コーポレイション オブ アメリカ ホールディングス | 自閉症スペクトラム障害を診断するための組成物および方法 |
| WO2011032034A2 (en) | 2009-09-10 | 2011-03-17 | University Of Idaho | Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids |
| CA2778171C (en) | 2009-10-29 | 2017-07-25 | Osaka University | Bridged artificial nucleoside and nucleotide |
| LT2499249T (lt) | 2009-11-12 | 2018-12-27 | The University Of Western Australia | Priešprasmės molekulės ir patologijų gydymo būdai |
| WO2011066312A1 (en) | 2009-11-25 | 2011-06-03 | Elitech Holding B.V. | Minor groove binder (mgb)-oligonucleotide mirna antagonists |
| US8779118B2 (en) | 2010-01-11 | 2014-07-15 | Isis Pharmaceuticals, Inc. | Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom |
| EP3561060A1 (en) | 2010-02-08 | 2019-10-30 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| WO2011115818A1 (en) | 2010-03-17 | 2011-09-22 | Isis Pharmaceuticals, Inc. | 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2011119842A1 (en) | 2010-03-25 | 2011-09-29 | The J. David Gladstone Institutes | Compositions and methods for treating neurological disorders |
| WO2011127210A1 (en) | 2010-04-06 | 2011-10-13 | Massachusetts Institute Of Technology | Targeted delivery of nucleic acids |
| EP2560687B1 (en) | 2010-04-19 | 2017-05-24 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| 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 |
| 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 |
| WO2011156278A1 (en) | 2010-06-07 | 2011-12-15 | 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 |
| US9518259B2 (en) | 2010-06-15 | 2016-12-13 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating interaction between proteins and target nucleic acids |
| PT2585596T (pt) | 2010-06-23 | 2021-03-23 | Curna Inc | Tratamento de doenças relacionadas com a subunidade alfa de canais de sódio dependentes de voltagem (scna) por inibição da transcrição antissentido natural para scna |
| KR101865433B1 (ko) | 2010-10-22 | 2018-07-13 | 큐알엔에이, 인크. | 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료 |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| WO2012106529A1 (en) | 2011-02-02 | 2012-08-09 | 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 |
| WO2013081755A1 (en) | 2011-11-29 | 2013-06-06 | Life Technologies Corporation | Methods and compositions for multiplex pcr |
| EP2702170B1 (en) | 2011-04-28 | 2015-12-16 | Life Technologies Corporation | Methods and compositions for multiplex pcr |
| ES2683161T3 (es) | 2011-06-10 | 2018-09-25 | Institut National De La Santé Et De La Recherche Médicale (Inserm) | Procedimientos de tratamiento de amaurosis congénita de Leber |
| US20140357558A1 (en) | 2011-06-24 | 2014-12-04 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of spinal muscular atrophy |
| ES2626488T3 (es) | 2011-07-19 | 2017-07-25 | Wave Life Sciences Pte. Ltd. | Procedimientos para la síntesis de ácidos nucleicos funcionalizados |
| US8592156B2 (en) | 2011-08-08 | 2013-11-26 | Roche Molecular Systems, Inc. | Predicting response to anti-CD20 therapy in DLBCL patients |
| EP3640332A1 (en) | 2011-08-29 | 2020-04-22 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| US9771580B2 (en) | 2011-09-05 | 2017-09-26 | Stichting Katholieke Universiteit | Antisense oligonucleotides for the treatment of Leber congenital amaurosis |
| JP6125505B2 (ja) | 2011-09-06 | 2017-05-10 | カッパーアールエヌエー,インコーポレイテッド | 小分子による電位開口型ナトリウムチャネル・アルファサブユニット関連疾患の治療 |
| WO2013043878A2 (en) | 2011-09-20 | 2013-03-28 | The George Washington University | Alternative splicing variants of genes associated with prostate cancer risk and survival |
| KR20140091587A (ko) | 2011-11-11 | 2014-07-21 | 산타리스 팔마 에이/에스 | Smn2 스플라이싱의 조절을 위한 화합물 |
| US9534222B2 (en) | 2011-11-15 | 2017-01-03 | University Of Utah Research Foundation | Morpholinos, morpholino upregulating, and associated methods |
| WO2013106770A1 (en) | 2012-01-11 | 2013-07-18 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of ikbkap splicing |
| WO2013120003A1 (en) | 2012-02-08 | 2013-08-15 | Isis Pharmaceuticals, Inc. | Modulation of rna by repeat targeting |
| ES2697174T3 (es) | 2012-02-10 | 2019-01-22 | Ptc Therapeutics Inc | Compuestos para tratar atrofia muscular espinal |
| 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 |
| EP2852605B1 (en) | 2012-05-22 | 2018-01-31 | Idenix Pharmaceuticals LLC | 3',5'-cyclic phosphate prodrugs for hcv infection |
| US9109001B2 (en) | 2012-05-22 | 2015-08-18 | Idenix Pharmaceuticals, Inc. | 3′,5′-cyclic phosphoramidate prodrugs for HCV infection |
| US20150267192A1 (en) | 2012-06-08 | 2015-09-24 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| ES2940887T3 (es) | 2012-07-13 | 2023-05-12 | Wave Life Sciences Ltd | Método de preparación de oligonucleótidos quirales |
| 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 |
| CN104781271B (zh) | 2012-08-20 | 2018-07-06 | 加利福尼亚大学董事会 | 具有生物可逆的基团的多核苷酸 |
| WO2014045283A1 (en) | 2012-09-24 | 2014-03-27 | Yissum Research 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 |
| EP2900682A1 (en) | 2012-09-27 | 2015-08-05 | 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 |
| US9909128B2 (en) | 2012-11-15 | 2018-03-06 | 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 |
| EP2946019A1 (en) | 2013-01-18 | 2015-11-25 | Anna Mary Rose | Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1) |
| US10260069B2 (en) | 2013-02-04 | 2019-04-16 | 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 |
| US9187515B2 (en) | 2013-04-01 | 2015-11-17 | Idenix Pharmaceuticals Llc | 2′,4′-fluoro nucleosides for the treatment of HCV |
| US10590412B2 (en) | 2013-04-19 | 2020-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation nucleic acids through nonsense mediated decay |
| HUE050394T2 (hu) | 2013-05-01 | 2020-11-30 | Ionis Pharmaceuticals Inc | Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények |
| US9549909B2 (en) | 2013-05-03 | 2017-01-24 | The Katholieke Universiteit Leuven | Method for the treatment of dravet syndrome |
| EP3008184A1 (en) | 2013-06-13 | 2016-04-20 | 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 |
| RU2673542C2 (ru) | 2013-06-25 | 2018-11-28 | Ф. Хоффманн-Ля Рош Аг | Соединения для лечения спинальной мышечной атрофии |
| 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 |
| WO2015023938A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Epigenetic regulators of frataxin |
| MX2016002044A (es) | 2013-08-16 | 2016-08-17 | Rana Therapeutics Inc | Composiciones y metodos para modular el acido ribonucleico. |
| US20160201064A1 (en) | 2013-08-16 | 2016-07-14 | Rana Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin |
| CA2915764A1 (en) | 2013-08-19 | 2015-02-26 | F. Hoffmann-La Roche Ag | Screening method |
| PL3041958T3 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
| 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 |
| KR20230008892A (ko) | 2013-11-21 | 2023-01-16 | 메모리얼 슬로안 케터링 캔서 센터 | 인간 다능성 줄기 세포로부터 기능적 두개 기원판 유도체의 전문화 |
| 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 |
| JP6851201B2 (ja) | 2014-06-10 | 2021-03-31 | エラスムス ユニバーシティ メディカルセンター ロッテルダムErasmus University Medical Center Rotterdam | ポンペ病の治療に有用なアンチセンスオリゴヌクレオチド |
| 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 |
| WO2016027168A2 (en) | 2014-08-20 | 2016-02-25 | Lifesplice Pharma Llc | Splice modulating oligonucleotides and methods of use thereof |
| CA2958524A1 (en) | 2014-08-20 | 2016-02-25 | Lifesplice Pharma Llc | Splice modulating oligonucleotides and methods of use thereof |
| 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 |
| US10822369B2 (en) | 2014-11-14 | 2020-11-03 | 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 |
| JP2018509387A (ja) | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
| 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 |
| JP2018509143A (ja) | 2015-02-27 | 2018-04-05 | サレプタ セラピューティクス,インコーポレイテッド | 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン2の組入れ |
| EP3277817A4 (en) | 2015-04-03 | 2018-12-05 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating tmprss6 expression |
| EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| EP3352872A4 (en) | 2015-09-24 | 2019-07-10 | The Trustees of the University of Pennsylvania | TRIPTYENE DERIVATIVES FOR STABILIZING A NUCLEIC ACID COMPOUND |
| GB2546719B (en) | 2015-10-09 | 2021-07-14 | Univ Southampton | Modulation of gene expression and screening for deregulated protein expression |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| TW201729807A (zh) | 2015-10-30 | 2017-09-01 | Ptc治療公司 | 用於治療癲癇的方法 |
| WO2017106211A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of polycystic kidney disease |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP4104867A3 (en) | 2015-12-14 | 2023-03-01 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of central nervous system diseases |
| CA3005245A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of alagille syndrome |
| EP3390642B1 (en) | 2015-12-14 | 2021-11-24 | Cold Spring Harbor Laboratory | Compositions for treatment of retinitis pigmentosa 13 |
| CA3005128A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of eye diseases |
| WO2017106292A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of kidney diseases |
| EP3389672A4 (en) | 2015-12-14 | 2019-08-14 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES |
| EP3390635A4 (en) | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| EP3443001B1 (en) | 2016-04-11 | 2025-04-30 | Obsidian Therapeutics, Inc. | REGULATED BIOCIRCUIT SYSTEMS |
| EP3481857A1 (en) | 2016-07-06 | 2019-05-15 | Crispr Therapeutics AG | Materials and methods for treatment of pain related disorders |
| KR102443631B1 (ko) | 2016-09-16 | 2022-09-15 | 올리패스 주식회사 | Scn9a 안티센스 올리고뉴클레오티드 |
| EP4403642A3 (en) | 2017-04-03 | 2024-10-09 | Encoded Therapeutics, Inc. | Tissue selective transgene expression |
| MX2019012285A (es) | 2017-04-13 | 2020-11-06 | Ovid Therapeutics Inc | Metodos de tratamiento de encefalopatias del desarrollo. |
| WO2018206924A1 (en) | 2017-05-09 | 2018-11-15 | Zogenix International Limited | Methods of treating doose syndrome using fenfluramine |
| US20200085838A1 (en) | 2017-05-16 | 2020-03-19 | Praxis Precision Medicines, Inc. | Methods of treating epilepsy and neurodevelopmental disorders |
| CN111278991B (zh) | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| RS66633B1 (sr) | 2017-10-23 | 2025-08-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk |
| JP7401432B2 (ja) | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス, インコーポレイテッド | 操作されたdna結合タンパク質 |
| AU2019243155A1 (en) | 2018-03-27 | 2020-10-15 | Factor Bioscience Inc. | Nucleic acid-based therapeutics |
| US12121563B2 (en) | 2018-04-09 | 2024-10-22 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| EP3806834A4 (en) | 2018-05-25 | 2022-07-27 | The Florey Institute of Neuroscience and Mental Health | DYNAMIC CLAMPS AND METHODS OF USE THEREOF |
| US20210228531A1 (en) | 2018-06-05 | 2021-07-29 | Tufts Medical Center, Inc. | Targeted treatment of autism spectrum disorder and other neurological or psychiatric disorders |
| KR20210027384A (ko) | 2018-06-22 | 2021-03-10 | 에프. 호프만-라 로슈 아게 | Scn9a 발현을 조절하기 위한 올리고뉴클레오티드 |
| US11939582B2 (en) | 2018-08-20 | 2024-03-26 | Rogcon, Inc. | Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies |
| US10905778B2 (en) | 2018-09-26 | 2021-02-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| WO2020176776A1 (en) | 2019-02-27 | 2020-09-03 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
-
2016
- 2016-12-14 KR KR1020187019006A patent/KR102604132B1/ko active Active
- 2016-12-14 SG SG11201804443UA patent/SG11201804443UA/en unknown
- 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 WO PCT/US2016/066708 patent/WO2017106377A1/en not_active Ceased
- 2016-12-14 EP EP21167500.4A patent/EP3933041B1/en active Active
- 2016-12-14 AU AU2016370653A patent/AU2016370653A1/en not_active Abandoned
- 2016-12-14 EP EP16876616.0A patent/EP3390636B1/en active Active
- 2016-12-14 CN CN202211146489.4A patent/CN116059236A/zh active Pending
- 2016-12-14 JP JP2018529250A patent/JP7049248B2/ja not_active Expired - Fee Related
- 2016-12-14 ES ES16876616T patent/ES2882500T3/es active Active
- 2016-12-14 CA CA3005256A patent/CA3005256A1/en active Pending
-
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 |
|---|---|
| EP3933041A1 (en) | 2022-01-05 |
| WO2017106377A1 (en) | 2017-06-22 |
| CA3005256A1 (en) | 2017-06-22 |
| AU2016370653A1 (en) | 2018-06-21 |
| EP3390636A4 (en) | 2019-08-14 |
| IL259222A (en) | 2018-07-31 |
| JP2022062140A (ja) | 2022-04-19 |
| US11083745B2 (en) | 2021-08-10 |
| ES2882500T3 (es) | 2021-12-02 |
| KR102604132B1 (ko) | 2023-11-17 |
| JP2019501892A (ja) | 2019-01-24 |
| EP3390636B1 (en) | 2021-05-19 |
| AU2023202216A1 (en) | 2023-07-06 |
| US20220088058A1 (en) | 2022-03-24 |
| SG11201804443UA (en) | 2018-06-28 |
| EP3390636A1 (en) | 2018-10-24 |
| CN109312343A (zh) | 2019-02-05 |
| CN109312343B (zh) | 2022-09-27 |
| KR20180093977A (ko) | 2018-08-22 |
| US20180369275A1 (en) | 2018-12-27 |
| EP3933041B1 (en) | 2024-01-31 |
| CN116059236A (zh) | 2023-05-05 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7049248B2 (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 | |
| JP2025098252A (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 |