CA3005245A1 - Oligomeres antisens pour le traitement du syndrome d'alagille - Google Patents
Oligomeres antisens pour le traitement du syndrome d'alagille Download PDFInfo
- Publication number
- CA3005245A1 CA3005245A1 CA3005245A CA3005245A CA3005245A1 CA 3005245 A1 CA3005245 A1 CA 3005245A1 CA 3005245 A CA3005245 A CA 3005245A CA 3005245 A CA3005245 A CA 3005245A CA 3005245 A1 CA3005245 A1 CA 3005245A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleobases
- mrna
- fold
- protein
- ric pre
- 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.)
- Pending
Links
Classifications
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
- 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
- C12Q2600/156—Polymorphic or mutational markers
Abstract
L'invention concerne des procédés et des compositions pour augmenter l'expression de JAGl, et pour traiter un sujet en ayant besoin, par exemple un sujet souffrant d'une déficience d'expression de la protéine JAGl ou un sujet atteint du syndrome d'Alagille (SAG).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562267210P | 2015-12-14 | 2015-12-14 | |
US62/267,210 | 2015-12-14 | ||
PCT/US2016/066414 WO2017106210A1 (fr) | 2015-12-14 | 2016-12-13 | Oligomères antisens pour le traitement du syndrome d'alagille |
Publications (1)
Publication Number | Publication Date |
---|---|
CA3005245A1 true CA3005245A1 (fr) | 2017-06-22 |
Family
ID=59057483
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA3005245A Pending CA3005245A1 (fr) | 2015-12-14 | 2016-12-13 | Oligomeres antisens pour le traitement du syndrome d'alagille |
Country Status (4)
Country | Link |
---|---|
EP (1) | EP3389671A4 (fr) |
JP (2) | JP2018538288A (fr) |
CA (1) | CA3005245A1 (fr) |
WO (1) | WO2017106210A1 (fr) |
Families Citing this family (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
WO2016054615A2 (fr) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Augmentation ciblée de la production de gènes nucléaires |
KR20220105174A (ko) | 2015-10-09 | 2022-07-26 | 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 |
JP7049248B2 (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 | 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 |
JP7458785B2 (ja) | 2017-01-23 | 2024-04-01 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ヒドロキシステロイド17-βデヒドロゲナーゼ13(HSD17B13)バリアント及びその使用 |
CN110325649A (zh) | 2017-04-11 | 2019-10-11 | 雷杰纳荣制药公司 | 用于筛选羟基类固醇(17-β)脱氢酶(HSD17B)家族成员的调节剂的活性的测定法 |
EP3668984A4 (fr) | 2017-08-18 | 2021-09-08 | Ionis Pharmaceuticals, Inc. | Modulation de la voie de signalisation notch pour le traitement de troubles respiratoires |
WO2019040923A1 (fr) | 2017-08-25 | 2019-02-28 | Stoke Therapeutics, Inc. | Oligomères antisens pour le traitement d'états pathologiques et autres maladies |
JP7434151B2 (ja) | 2017-10-11 | 2024-02-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Pnpla3 i148mの変異を発現している患者の肝疾患の治療におけるhsd17b13の阻害 |
CN115976028A (zh) * | 2018-03-09 | 2023-04-18 | 第一三共株式会社 | 糖原病Ia型治疗药 |
EP3784224A4 (fr) * | 2018-04-27 | 2022-08-03 | The Regents of the University of California | Formation de novo du système biliaire par transdifférenciation des hépatocytes |
TW202208627A (zh) | 2020-05-11 | 2022-03-01 | 美商斯托克治療公司 | 用於病症及疾病之治療的opa1反義寡聚物 |
Family Cites Families (16)
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 |
US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
JPH08501686A (ja) | 1992-09-25 | 1996-02-27 | ローン−プーラン・ロレ・ソシエテ・アノニム | 中枢神経系、特に脳における細胞への外来遺伝子の転移のためのアデノウィルスベクター |
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 |
US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
US20040102401A1 (en) * | 2002-11-22 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of jagged 1 expression |
GB0326578D0 (en) * | 2003-11-14 | 2003-12-17 | Univ Belfast | Cancer diagnosis and therapy |
WO2007047913A2 (fr) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions et procédés pour la modulation de l'expression du gène lmna |
WO2007048629A2 (fr) * | 2005-10-28 | 2007-05-03 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Modulation de l'efficacite du silençage d'un arn a l'aide de proteines argonautes |
EP1945765A2 (fr) | 2005-10-28 | 2008-07-23 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Structures de molecules d'arn-guide actives et leur procede de selection |
CA2708173C (fr) | 2007-12-04 | 2016-02-02 | Alnylam Pharmaceuticals, Inc. | Lipides de ciblage |
CN102665731A (zh) | 2009-06-17 | 2012-09-12 | Isis制药公司 | 用于在对象中调节smn2剪接的组合物和方法 |
US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
CA2930859C (fr) | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reduction de degradation d'arnm a mediation non-sens |
-
2016
- 2016-12-13 EP EP16876499.1A patent/EP3389671A4/fr active Pending
- 2016-12-13 JP JP2018529221A patent/JP2018538288A/ja not_active Ceased
- 2016-12-13 CA CA3005245A patent/CA3005245A1/fr active Pending
- 2016-12-13 WO PCT/US2016/066414 patent/WO2017106210A1/fr active Application Filing
-
2022
- 2022-04-20 JP JP2022069164A patent/JP2022106803A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
JP2022106803A (ja) | 2022-07-20 |
WO2017106210A1 (fr) | 2017-06-22 |
EP3389671A1 (fr) | 2018-10-24 |
EP3389671A4 (fr) | 2019-07-17 |
JP2018538288A (ja) | 2018-12-27 |
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Legal Events
Date | Code | Title | Description |
---|---|---|---|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |