EP3389672A4 - COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES - Google Patents

COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES Download PDF

Info

Publication number: EP3389672A4
Authority: EP; European Patent Office
Prior art keywords: compositions; methods; hepatic diseases; treating hepatic; treating
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 Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Withdrawn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

EP16876543.6A

Other languages

German (de)

French (fr)

Other versions

EP3389672A1 (en

Inventor

Isabel AZNAREZ

Huw M. Nash

Samuel W. HALL

Enxuan Jing

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

2016-12-14

Publication date

2019-08-14

2016-12-14 Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory

2018-10-24 Publication of EP3389672A1 publication Critical patent/EP3389672A1/en

2019-08-14 Publication of EP3389672A4 publication Critical patent/EP3389672A4/en

Status Withdrawn legal-status Critical Current

Links

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
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- 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
- 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
- C12N2310/34—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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Chemical & Material Sciences (AREA)
Engineering & Computer Science (AREA)
Molecular Biology (AREA)
General Health & Medical Sciences (AREA)
Veterinary Medicine (AREA)
Animal Behavior & Ethology (AREA)
Medicinal Chemistry (AREA)
Genetics & Genomics (AREA)
Public Health (AREA)
Pharmacology & Pharmacy (AREA)
Epidemiology (AREA)
Biochemistry (AREA)
Biomedical Technology (AREA)
Organic Chemistry (AREA)
Bioinformatics & Cheminformatics (AREA)
Wood Science & Technology (AREA)
Biotechnology (AREA)
Zoology (AREA)
General Engineering & Computer Science (AREA)
Plant Pathology (AREA)
Physics & Mathematics (AREA)
Biophysics (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Hematology (AREA)
Diabetes (AREA)
Microbiology (AREA)
Chemical Kinetics & Catalysis (AREA)
General Chemical & Material Sciences (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Investigating Or Analysing Biological Materials (AREA)

EP16876543.6A 2015-12-14 2016-12-14 COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES Withdrawn EP3389672A4 (en)

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
US201562267238P	2015-12-14	2015-12-14
US201662319015P	2016-04-06	2016-04-06
PCT/US2016/066564 WO2017106283A1 (en)	2015-12-14	2016-12-14	Compositions and methods for treatment of liver diseases

Publications (2)

Publication Number	Publication Date
EP3389672A1 EP3389672A1 (en)	2018-10-24
EP3389672A4 true EP3389672A4 (en)	2019-08-14

Family

ID=59057460

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
EP16876543.6A Withdrawn EP3389672A4 (en)	2015-12-14	2016-12-14	COMPOSITIONS AND METHODS FOR TREATING HEPATIC DISEASES

Country Status (4)

Country	Link
EP (1)	EP3389672A4 (en)
JP (1)	JP2019500345A (en)
CA (1)	CA3005090A1 (en)
WO (1)	WO2017106283A1 (en)

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CA3049980A1 (en)	2017-01-23	2018-07-26	Regeneron Pharmaceuticals, Inc.	Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
CA3059348A1 (en)	2017-04-11	2018-10-18	Regeneron Pharmaceuticals, Inc.	Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
KR20260009385A (en)	2017-08-25	2026-01-19	스톡 테라퓨틱스, 인크.	Antisense oligomers for treatment of conditions and diseases
EP3461837A1 (en) *	2017-09-28	2019-04-03	Secarna Pharmaceuticals GmbH & Co. KG	Inhibitor inhibiting the expression of pprx1
AU2018348195B2 (en)	2017-10-11	2025-05-15	Regeneron Pharmaceuticals, Inc.	Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
LT3700570T (en) *	2017-10-23	2025-04-10	Stoke Therapeutics, Inc.	ADVANTAGEOUS OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON NONSENSE CODO-INDUCED mRNA DEGRADATION
AU2019222767A1 (en) *	2018-02-14	2020-08-27	Deep Genomics Incorporated	Oligonucleotide therapy for Wilson disease
WO2019165067A1 (en) *	2018-02-21	2019-08-29	Bristol-Myers Squibb Company	Camk2d antisense oligonucleotides and uses thereof
MY207352A (en)	2018-03-21	2025-02-21	Regeneron Pharma	17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
JP2021523227A (en)	2018-05-04	2021-09-02	ストークセラピューティクス，インク．	Methods and Compositions for the Treatment of Cholesteryl Ester Accumulation
TWI869213B (en) *	2018-09-19	2025-01-01	美商Ionis製藥公司	Modulators of pnpla3 expression
US10913951B2 (en)	2018-10-31	2021-02-09	University of Pittsburgh—of the Commonwealth System of Higher Education	Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
KR20210134003A (en)	2019-02-27	2021-11-08	스톡 테라퓨틱스, 인크.	Antisense oligomers for the treatment of conditions and diseases
CN111849968A (en) *	2019-04-30	2020-10-30	中美瑞康核酸技术(南通)研究院有限公司	Oligonucleotide molecule and application thereof in acute intermittent porphyria treatment
KR20220104677A (en) *	2019-08-19	2022-07-26	스톡 테라퓨틱스, 인크.	Compositions and methods for modulating splicing and protein expression
CA3148158A1 (en) *	2019-09-20	2021-03-25	Longcheng Li	Nucleic acid molecule for treating thrombocytopenia and use thereof
CN114728017B (en) *	2019-10-14	2025-10-28	阿斯利康(瑞典)有限公司	Regulators of PNPLA3 expression
WO2021113773A2 (en)	2019-12-04	2021-06-10	Pai Athma A	Identifying non-productive splice sites
TWI817107B (en) *	2020-04-15	2023-10-01	中央研究院	Oligonucleotide conjugates and preparation and applications thereof
US12338437B2 (en)	2020-05-11	2025-06-24	Stoke Therapeutics, Inc.	OPA1 antisense oligomers for treatment of conditions and diseases
WO2021231698A1 (en) *	2020-05-15	2021-11-18	Korro Bio, Inc.	Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4185696A1 (en)	2020-07-23	2023-05-31	F. Hoffmann-La Roche AG	Oligonucleotides targeting rna binding protein sites
WO2022109030A1 (en) *	2020-11-17	2022-05-27	Skyhawk Therapeutics, Inc.	Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases
US20250333742A1 (en) *	2024-04-25	2025-10-30	Insitro, Inc.	Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

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2016
- 2016-12-14 WO PCT/US2016/066564 patent/WO2017106283A1/en not_active Ceased
- 2016-12-14 CA CA3005090A patent/CA3005090A1/en active Pending
- 2016-12-14 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/en active Pending

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See also references of WO2017106283A1 *

Also Published As

Publication number	Publication date
CA3005090A1 (en)	2017-06-22
EP3389672A1 (en)	2018-10-24
WO2017106283A1 (en)	2017-06-22
JP2019500345A (en)	2019-01-10

Legal Events

Date	Code	Title	Description
2017-06-23	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE
2018-09-21	PUAI	Public reference made under article 153(3) epc to a published international application that has entered the european phase	Free format text: ORIGINAL CODE: 0009012
2018-09-21	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE
2018-10-24	17P	Request for examination filed	Effective date: 20180706
2018-10-24	AK	Designated contracting states	Kind code of ref document: A1 Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
2018-10-24	AX	Request for extension of the european patent	Extension state: BA ME
2019-03-20	DAV	Request for validation of the european patent (deleted)
2019-03-20	DAX	Request for extension of the european patent (deleted)
2019-08-14	A4	Supplementary search report drawn up and despatched	Effective date: 20190712
2019-08-14	RIC1	Information provided on ipc code assigned before grant	Ipc: A61K 31/7125 20060101ALI20190708BHEP Ipc: A61K 31/7088 20060101AFI20190708BHEP Ipc: C12N 15/113 20100101ALI20190708BHEP Ipc: C12Q 1/68 20180101ALI20190708BHEP Ipc: A61P 7/00 20060101ALI20190708BHEP Ipc: A61K 31/712 20060101ALI20190708BHEP
2020-01-24	REG	Reference to a national code	Ref country code: HK Ref legal event code: DE Ref document number: 1262957 Country of ref document: HK
2020-05-06	RIN1	Information on inventor provided before grant (corrected)	Inventor name: AZNAREZ, ISABEL Inventor name: JING, ENXUAN Inventor name: KRAINER, ADRIAN Inventor name: NASH, HUW M. Inventor name: HALL, SAMUEL W.
2021-12-16	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: EXAMINATION IS IN PROGRESS
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2022-10-19	RIN1	Information on inventor provided before grant (corrected)	Inventor name: KRAINER, ADRIAN Inventor name: JING, ENXUAN Inventor name: HALL, SAMUEL W. Inventor name: NASH, HUW M. Inventor name: AZNAREZ, ISABEL
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