PL2417257T3 - Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby - Google Patents
Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia chorobyInfo
- Publication number
- PL2417257T3 PL2417257T3 PL10713338.1T PL10713338T PL2417257T3 PL 2417257 T3 PL2417257 T3 PL 2417257T3 PL 10713338 T PL10713338 T PL 10713338T PL 2417257 T3 PL2417257 T3 PL 2417257T3
- Authority
- PL
- Poland
- Prior art keywords
- tricyclo
- disease
- compositions
- treatment
- methods
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Rheumatology (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21238409P | 2009-04-10 | 2009-04-10 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2417257T3 true PL2417257T3 (pl) | 2016-11-30 |
Family
ID=42293782
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL10713338.1T PL2417257T3 (pl) | 2009-04-10 | 2010-04-09 | Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20120149756A1 (pl) |
| EP (1) | EP2417257B1 (pl) |
| JP (3) | JP2012523225A (pl) |
| CN (2) | CN102625840A (pl) |
| CA (1) | CA2758189C (pl) |
| DK (1) | DK2417257T3 (pl) |
| ES (1) | ES2573981T3 (pl) |
| HU (1) | HUE028036T2 (pl) |
| IL (1) | IL215682A (pl) |
| PL (1) | PL2417257T3 (pl) |
| PT (1) | PT2417257E (pl) |
| WO (1) | WO2010115993A1 (pl) |
Families Citing this family (88)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2361325T3 (es) | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| NZ584793A (en) | 2007-10-26 | 2012-05-25 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| EP4174178A1 (en) | 2008-10-24 | 2023-05-03 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| WO2010119368A2 (en) * | 2009-04-13 | 2010-10-21 | Sulur Subramaniam Vanangamudi | A medicinal cream made using miconazole nitrate and chitosan, and a process to make the same |
| CA3185821A1 (en) * | 2009-05-08 | 2010-11-11 | Curna, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
| EP3431603A1 (en) | 2009-11-12 | 2019-01-23 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
| CN106434648A (zh) * | 2010-07-19 | 2017-02-22 | F·C·贝内特 | 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| AU2011325956B2 (en) | 2010-11-12 | 2016-07-14 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP2699269A1 (en) * | 2011-04-22 | 2014-02-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| US9340783B2 (en) * | 2011-10-11 | 2016-05-17 | Inserm (Institut National De La Sante Et De La Recherche Medicale | Exon skipping therapy for dystrophic epidermolysis bullosa |
| PT2581448E (pt) | 2011-10-13 | 2015-05-21 | Institut National De La Santé Et De La Rech Médicale (Inserm) | Dna triciclo-fosforotioato |
| WO2013068441A1 (en) * | 2011-11-11 | 2013-05-16 | Santaris Pharma A/S | Compounds for the modulation of smn2 splicing |
| EP2806900B1 (en) | 2012-01-27 | 2021-12-15 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 |
| IL280443B (en) | 2013-03-14 | 2022-07-01 | Sarepta Therapeutics Inc | Preparations that skip axon for the treatment of muscular dystrophy |
| EA201591792A1 (ru) | 2013-03-15 | 2016-02-29 | Сарепта Терапьютикс, Инк. | Улучшенные композиции для лечения мышечной дистрофии |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| WO2015035231A1 (en) | 2013-09-05 | 2015-03-12 | Sarepta Therapeutics, Inc. | 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 |
| US10174328B2 (en) | 2013-10-04 | 2019-01-08 | Translate Bio Ma, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| AU2015272128B2 (en) | 2014-06-10 | 2021-10-28 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of Pompe disease |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| AU2015327836B2 (en) * | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| EP3256591A4 (en) | 2015-02-13 | 2018-08-08 | Translate Bio Ma, Inc. | Hybrid oligonucleotides and uses thereof |
| EP3271460A4 (en) | 2015-03-17 | 2019-03-13 | The General Hospital Corporation | INTERACTOME RNA OF COMPLEX REPRESSIVE POLYCOMB 1 (PRC1) |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| LT3283500T (lt) | 2015-04-08 | 2020-12-10 | The University Of Chicago | Kompozicijos ir būdai, skirti 2c tipo dubens-žasto raumenų distrofijos korekcijai, panaudojant egzono peršokimą |
| WO2016196670A1 (en) | 2015-06-01 | 2016-12-08 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| BR112018007066A2 (pt) | 2015-10-09 | 2018-10-23 | Sarepta Therapeutics Inc | composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados |
| CA3005434A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| AU2015416656B2 (en) | 2015-12-07 | 2023-02-23 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for Pompe disease |
| CA3021267A1 (en) | 2016-04-18 | 2017-10-26 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| NZ747685A (en) | 2016-04-29 | 2023-05-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| LT3464306T (lt) | 2016-05-24 | 2024-05-10 | Sarepta Therapeutics, Inc. | Fosforodiamidato morfolino oligomerų gamybos būdas |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| TWI801843B (zh) | 2016-05-24 | 2023-05-11 | 美商薩羅塔治療公司 | 製備寡聚物之方法 |
| CA3025575A1 (en) | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| NL2017294B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| NL2017295B1 (en) | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Antisense oligomeric compound for Pompe disease |
| WO2018055577A1 (en) | 2016-09-23 | 2018-03-29 | Synthena Ag | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| CN110636866A (zh) | 2016-12-19 | 2019-12-31 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| BR112019012647A2 (pt) | 2016-12-19 | 2019-11-19 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| JP7125940B2 (ja) | 2016-12-19 | 2022-08-25 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| CN110536694A (zh) | 2017-04-20 | 2019-12-03 | Atyr 医药公司 | 用于治疗肺部炎症的组合物和方法 |
| CA3059321A1 (en) | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| WO2018193428A1 (en) | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EP3689863A1 (en) | 2017-08-04 | 2020-08-05 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| WO2019057649A1 (en) | 2017-09-19 | 2019-03-28 | INSERM (Institut National de la Santé et de la Recherche Médicale) | METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF ACUTE MYELOID LEUKEMIA |
| JP7330174B2 (ja) | 2017-09-20 | 2023-08-21 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | オートファジーをモジュレーションするための方法及び医薬組成物 |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| US20210145852A1 (en) | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
| EP3687519A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019079637A2 (en) | 2017-10-18 | 2019-04-25 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERIC COMPOUNDS |
| WO2019101995A1 (en) | 2017-11-27 | 2019-05-31 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for cardiac regeneration |
| EA202091828A1 (ru) * | 2018-01-31 | 2021-05-24 | Дзе Борд Оф Риджентс Оф Дзе Юниверсити Оф Техас Систем | Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека |
| WO2019193375A1 (en) | 2018-04-04 | 2019-10-10 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Use of fzd7 inhibitors for the treatment of retinal neovascularization |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY |
| TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| EP3864161A4 (en) * | 2018-10-09 | 2022-11-23 | The University of North Carolina at Chapel Hill | REGULATED GENE EDIT SYSTEM |
| MX2021006737A (es) | 2018-12-13 | 2021-09-23 | Sarepta Therapeutics Inc | Conjugados de oligómeros de salto de exones para distrofia muscular. |
| WO2020163405A1 (en) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| KR20210135507A (ko) | 2019-02-06 | 2021-11-15 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| EP3955966A1 (en) | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| KR20220148230A (ko) | 2020-02-28 | 2022-11-04 | 아이오니스 파마수티컬즈, 인코포레이티드 | Smn2를 조절하기 위한 화합물 및 방법 |
| CN113444722A (zh) * | 2020-03-24 | 2021-09-28 | 中国科学院脑科学与智能技术卓越创新中心 | 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用 |
| EP3978608A1 (en) | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51 |
| CN112779252B (zh) * | 2020-12-31 | 2023-05-26 | 首都儿科研究所 | 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸 |
| KR20240012425A (ko) | 2021-05-10 | 2024-01-29 | 엔트라다 테라퓨틱스, 인크. | 세포내 치료제를 위한 조성물 및 방법 |
| CN113430198B (zh) * | 2021-06-08 | 2023-03-31 | 上海苹谱医疗科技有限公司 | 基于crispr增加smn蛋白表达的方法及其应用 |
| WO2022257010A1 (zh) * | 2021-06-08 | 2022-12-15 | 上海苹谱医疗科技有限公司 | 基于crispr增加smn蛋白表达的方法及其应用 |
| KR20240038967A (ko) | 2021-06-23 | 2024-03-26 | 엔트라다 테라퓨틱스, 인크. | Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법 |
| WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| KR20240070615A (ko) | 2021-09-30 | 2024-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 하나 이상의 비염기성 유닛을 갖는 안티센스 올리고뉴클레오티드 |
| WO2023070086A1 (en) | 2021-10-22 | 2023-04-27 | Sarepta Therapeutics, Inc. | Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU637800B2 (en) | 1989-08-31 | 1993-06-10 | City Of Hope | Chimeric dna-rna catalytic sequences |
| US5962219A (en) | 1990-06-11 | 1999-10-05 | Nexstar Pharmaceuticals, Inc. | Systematic evolution of ligands by exponential enrichment: chemi-selex |
| ES2061416T3 (es) | 1990-10-12 | 1997-03-01 | Max Planck Gesellschaft | Ribozimas modificadas. |
| DE4216134A1 (de) | 1991-06-20 | 1992-12-24 | Europ Lab Molekularbiolog | Synthetische katalytische oligonukleotidstrukturen |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| US6001311A (en) | 1997-02-05 | 1999-12-14 | Protogene Laboratories, Inc. | Apparatus for diverse chemical synthesis using two-dimensional array |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| JP2003528024A (ja) | 1997-12-16 | 2003-09-24 | バレンティス・インコーポレーテッド | 製剤化された核酸分子の無針注入 |
| WO1999054459A2 (en) | 1998-04-20 | 1999-10-28 | Ribozyme Pharmaceuticals, Inc. | Nucleic acid molecules with novel chemical compositions capable of modulating gene expression |
| JP2002537828A (ja) | 1999-03-10 | 2002-11-12 | フォゲン リミティド | 細胞への物質のデリバリー |
| 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 |
| ES2361325T3 (es) * | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
| FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| JP2008539698A (ja) * | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| ES2545223T3 (es) * | 2005-06-23 | 2015-09-09 | Isis Pharmaceuticals, Inc. | Composiciones y procedimientos para modular el corte y empalme de SMN2 |
| WO2007123391A1 (en) * | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
| EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| WO2008018795A1 (en) * | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| EP2282744B1 (en) * | 2008-03-21 | 2018-01-17 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising tricyclic nucleosides and methods for their use |
-
2010
- 2010-04-09 CA CA2758189A patent/CA2758189C/en active Active
- 2010-04-09 PL PL10713338.1T patent/PL2417257T3/pl unknown
- 2010-04-09 EP EP10713338.1A patent/EP2417257B1/en active Active
- 2010-04-09 CN CN2010800223762A patent/CN102625840A/zh active Pending
- 2010-04-09 HU HUE10713338A patent/HUE028036T2/en unknown
- 2010-04-09 DK DK10713338.1T patent/DK2417257T3/da active
- 2010-04-09 US US13/263,949 patent/US20120149756A1/en not_active Abandoned
- 2010-04-09 WO PCT/EP2010/054735 patent/WO2010115993A1/en active Application Filing
- 2010-04-09 CN CN201811113867.2A patent/CN109576268A/zh active Pending
- 2010-04-09 PT PT107133381T patent/PT2417257E/pt unknown
- 2010-04-09 ES ES10713338.1T patent/ES2573981T3/es active Active
- 2010-04-09 JP JP2012504034A patent/JP2012523225A/ja active Pending
-
2011
- 2011-10-10 IL IL215682A patent/IL215682A/en active IP Right Grant
-
2015
- 2015-10-02 JP JP2015197054A patent/JP6675851B2/ja active Active
-
2017
- 2017-02-09 US US15/428,385 patent/US10465191B2/en active Active
-
2018
- 2018-12-07 JP JP2018229679A patent/JP2019055990A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP2417257A1 (en) | 2012-02-15 |
| CN109576268A (zh) | 2019-04-05 |
| EP2417257B1 (en) | 2016-03-09 |
| ES2573981T3 (es) | 2016-06-13 |
| JP6675851B2 (ja) | 2020-04-08 |
| CN102625840A (zh) | 2012-08-01 |
| PT2417257E (pt) | 2016-06-03 |
| IL215682A0 (en) | 2012-01-31 |
| IL215682A (en) | 2017-06-29 |
| WO2010115993A1 (en) | 2010-10-14 |
| HUE028036T2 (en) | 2016-11-28 |
| DK2417257T3 (da) | 2016-06-06 |
| US20170260524A1 (en) | 2017-09-14 |
| CA2758189A1 (en) | 2010-10-14 |
| JP2016027823A (ja) | 2016-02-25 |
| JP2012523225A (ja) | 2012-10-04 |
| US20120149756A1 (en) | 2012-06-14 |
| JP2019055990A (ja) | 2019-04-11 |
| CA2758189C (en) | 2020-12-29 |
| US10465191B2 (en) | 2019-11-05 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL215682A0 (en) | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease | |
| IL262668A (en) | Preparations for the treatment of Gaucher disease | |
| IL214396A (en) | Anti-viral anti-viral nucleosides, pharmaceutical preparations containing them and methods for their preparation, as well as their preparation of antiviral intermediates | |
| EP2260109A4 (en) | METHODS AND COMPOSITIONS BASED ON MICRO-RNA FOR DIAGNOSING, PROGNOSING AND TREATING PROSTATE-RELATED DISORDERS | |
| EP2432476A4 (en) | METHOD FOR THE TREATMENT OR PREVENTION OF EYE DISEASES | |
| EP2459565A4 (en) | METHOD AND COMPOSITIONS FOR STUDYING, PRESENTING AND TREATING PAIN | |
| HK1169941A1 (zh) | 治療外周血管疾病的組合物 | |
| EP2497482A4 (en) | COMPOSITION FOR PROPHYLAXIS AND TREATMENT OF INFLUENZA AVIRUSED INDUCED DISEASES | |
| IL215061A0 (en) | Compounds, compositions and methods of treating cancer and fibrotic diseases | |
| GB0908193D0 (en) | Treatment of disease state | |
| EP2429584A4 (en) | TREATMENT PROCEDURE AND COMPOSITIONS | |
| EP2328417A4 (en) | COMPOUNDS, COMPOSITIONS AND METHODS FOR THE TREATMENT OF Beta-AMYLOID AND SYNUCLEINOPATHY DISEASES | |
| ZA201203281B (en) | Pharmaceutical combinations, pharmaceutical compositions, medicament and method for the treatment of animals | |
| PL2209458T3 (pl) | Kompozycje do traktowania skóry | |
| EP2215035A4 (en) | BISULFIT TREATMENT OF RNA | |
| EP2274411A4 (en) | XANTHOHUMOL COMPOSITIONS AND METHODS FOR TREATING SKIN DISEASES OR CONDITIONS | |
| ZA201007005B (en) | Methods of diagnosing, preventing and treating bone mass diseases | |
| EP2502989A4 (en) | IMPLANT COMPOSITION FOR THE REGENERATION OF NEURAL TISSUE, PREPARATION METHOD AND USES THEREOF | |
| EP2453019A4 (en) | GENE THERAPY COMPOSITIONS FOR PREVENTING AND / OR TREATING AUTOIMMUNE DISEASES | |
| IL225755B (en) | amido derivatives - 6 of epoxymorphinans - a5, 4 for the treatment of pain | |
| EP2410846A4 (en) | GLYCOLIPIDES AS TREATMENT OF DISEASES | |
| GB2488494B (en) | Solution, use and method of preparation thereof | |
| EP2462131A4 (en) | COMPOSITIONS AND METHODS FOR TREATING DISEASES ASSOCIATED WITH BETA-AMYLOID | |
| EP2432322A4 (en) | OPTIMAL COMPOSITIONS AND METHODS OF TREATING ORAL DISEASES AND PAIN | |
| GB0808326D0 (en) | Compositions and methods for the treatment of fybromyalgia |