EP3389671A4 - ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE'S SYNDROME - Google Patents

ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE'S SYNDROME Download PDF

Info

Publication number: EP3389671A4
Authority: EP; European Patent Office
Prior art keywords: alagille; syndrome; treatment; antisense oligomers; antisense
Prior art date: 2015-12-14
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 Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Withdrawn

Application number

EP16876499.1A

Other languages

German (de)

French (fr)

Other versions

EP3389671A1 (en

Inventor

Isabel AZNAREZ

Huw M. Nash

Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)

Cold Spring Harbor Laboratory

Stoke Therapeutics Inc

Original Assignee

Cold Spring Harbor Laboratory

Stoke Therapeutics Inc

Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)

2015-12-14

Filing date

2016-12-13

Publication date

2019-07-17

2016-12-13Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics IncfiledCriticalCold Spring Harbor Laboratory

2018-10-24Publication of EP3389671A1publicationCriticalpatent/EP3389671A1/en

2019-07-17Publication of EP3389671A4publicationCriticalpatent/EP3389671A4/en

StatusWithdrawnlegal-statusCriticalCurrent

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
- 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
- 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
- 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
- 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
- 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
- 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
- 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

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Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Chemical & Material Sciences (AREA)
Engineering & Computer Science (AREA)
Organic Chemistry (AREA)
Genetics & Genomics (AREA)
Bioinformatics & Cheminformatics (AREA)
Zoology (AREA)
Wood Science & Technology (AREA)
General Health & Medical Sciences (AREA)
Biomedical Technology (AREA)
Molecular Biology (AREA)
General Engineering & Computer Science (AREA)
Biotechnology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Pharmacology & Pharmacy (AREA)
Biophysics (AREA)
Veterinary Medicine (AREA)
Physics & Mathematics (AREA)
Microbiology (AREA)
Public Health (AREA)
Animal Behavior & Ethology (AREA)
Biochemistry (AREA)
Medicinal Chemistry (AREA)
General Chemical & Material Sciences (AREA)
Analytical Chemistry (AREA)
Chemical Kinetics & Catalysis (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Immunology (AREA)
Plant Pathology (AREA)
Gastroenterology & Hepatology (AREA)
Neurology (AREA)
Orthopedic Medicine & Surgery (AREA)
Physical Education & Sports Medicine (AREA)
Epidemiology (AREA)
Pathology (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Saccharide Compounds (AREA)

EP16876499.1A2015-12-142016-12-13 ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE'S SYNDROMEWithdrawnEP3389671A4 (en)

Applications Claiming Priority (2)

Application Number	Priority Date	Filing Date	Title
US201562267210P	2015-12-14	2015-12-14
PCT/US2016/066414WO2017106210A1 (en)	2015-12-14	2016-12-13	Antisense oligomers for treatment of alagille syndrome

Publications (2)

Publication Number	Publication Date
EP3389671A1 EP3389671A1 (en)	2018-10-24
EP3389671A4true EP3389671A4 (en)	2019-07-17

Family

ID=59057483

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
EP16876499.1AWithdrawnEP3389671A4 (en)	2015-12-14	2016-12-13	ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE'S SYNDROME

Country Status (4)

Country	Link
EP (1)	EP3389671A4 (en)
JP (2)	JP2018538288A (en)
CA (1)	CA3005245A1 (en)
WO (1)	WO2017106210A1 (en)

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KR102422625B1 (en)	2015-10-09	2022-07-20	유니버시티 오브 사우스앰톤	Regulation of gene expression and screening of deregulated protein expression
US11096956B2 (en)	2015-12-14	2021-08-24	Stoke Therapeutics, Inc.	Antisense oligomers and uses thereof
EP3933041B1 (en)	2015-12-14	2024-01-31	Cold Spring Harbor Laboratory	Antisense oligomers for treatment of autosomal dominant retardation
CN118325899A (en)	2017-01-23	2024-07-12	瑞泽恩制药公司	HSD17B13 variants and their applications
CN110536964A (en)	2017-03-10	2019-12-03	国立研究开发法人国立成育医疗研究中心	Antisense oligonucleotide and composition for preventing or treating glycogen storage disease type Ia
MX2019012169A (en)	2017-04-11	2019-12-11	Regeneron Pharma	Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family.
US11197884B2 (en)	2017-08-18	2021-12-14	Ionis Pharmaceuticals, Inc.	Modulation of the notch signaling pathway for treatment of respiratory disorders
EP4303321A3 (en)	2017-08-25	2024-07-24	Stoke Therapeutics, Inc.	Antisense oligomers for treatment of conditions and diseases
EP4234719A3 (en)	2017-10-11	2023-10-18	Regeneron Pharmaceuticals, Inc.	Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
KR102398295B1 (en)*	2018-03-09	2022-05-17	다이이찌 산쿄 가부시키가이샤	Glycogen disease type Ia treatment
CA3091146A1 (en)	2018-03-21	2019-09-26	Regeneron Pharmaceuticals, Inc.	17.beta.-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2019210279A1 (en)*	2018-04-27	2019-10-31	The Regents Of The University Of California	De novo formation of the biliary system by hepatocyte transdifferentiation
BR112020022512A2 (en)	2018-05-04	2021-05-04	Stoke Therapeutics, Inc.	methods and compositions for treating cholesteryl ester storage disease
EP3847255A4 (en)*	2018-09-06	2024-01-10	The Regents of The University of California	ADAR RECRUITMENT DEVELOPED RNA AND DNA BASE EDITING
AU2021270720A1 (en)	2020-05-11	2022-12-08	Stoke Therapeutics, Inc.	OPA1 antisense oligomers for treatment of conditions and diseases

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2016
- 2016-12-13EPEP16876499.1Apatent/EP3389671A4/ennot_activeWithdrawn
- 2016-12-13WOPCT/US2016/066414patent/WO2017106210A1/ennot_activeCeased
- 2016-12-13JPJP2018529221Apatent/JP2018538288A/ennot_activeCeased
- 2016-12-13CACA3005245Apatent/CA3005245A1/ennot_activeAbandoned
2022
- 2022-04-20JPJP2022069164Apatent/JP2022106803A/enactivePending

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See also references ofWO2017106210A1*

Also Published As

Publication number	Publication date
EP3389671A1 (en)	2018-10-24
CA3005245A1 (en)	2017-06-22
WO2017106210A1 (en)	2017-06-22
JP2022106803A (en)	2022-07-20
JP2018538288A (en)	2018-12-27

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