EP3389672A4 - COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES - Google Patents

COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES Download PDF

Info

Publication number
EP3389672A4
EP3389672A4 EP16876543.6A EP16876543A EP3389672A4 EP 3389672 A4 EP3389672 A4 EP 3389672A4 EP 16876543 A EP16876543 A EP 16876543A EP 3389672 A4 EP3389672 A4 EP 3389672A4
Authority
EP
European Patent Office
Prior art keywords
compositions
methods
hepatic diseases
treating hepatic
treating
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.)
Withdrawn
Application number
EP16876543.6A
Other languages
German (de)
English (en)
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.)
Filing date
Publication date
Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of EP3389672A1 publication Critical patent/EP3389672A1/en
Publication of EP3389672A4 publication Critical patent/EP3389672A4/en
Withdrawn legal-status Critical Current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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
EP16876543.6A 2015-12-14 2016-12-14 COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER 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 METHOD FOR THE TREATMENT OF LIVER DISEASES

Country Status (4)

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

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for 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
JP7094963B2 (ja) 2017-01-23 2022-07-04 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 Ｈｓｄ１７ｂ１３バリアントおよびその使用
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
CA3073515A1 (en) 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. 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
SMT202500127T1 (it) 2017-10-23 2025-05-12 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di malattie e condizioni basate sul decadimento dell’rna mediato da un nonsenso
WO2019161105A1 (en) * 2018-02-14 2019-08-22 Deep Genomics Incorporated Oligonucleotide therapy for wilson disease
MX2020008581A (es) * 2018-02-21 2020-09-21 Bristol Myers Squibb Co Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
MX2020009812A (es) 2018-03-21 2021-01-08 Regeneron Pharma COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS.
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
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
CN111849968A (zh) * 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
KR20220070206A (ko) * 2019-09-20 2022-05-30 락티젠 세러퓨틱스 혈소판 감소증 치료용 핵산 분자 및 그의 이용
WO2021074772A1 (en) * 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
EP4069255A4 (en) * 2019-12-04 2024-02-21 Pai, Athma A. IDENTIFICATION OF NON-PRODUCTIVE SPLICE JOINTS
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
WO2021231698A1 (en) * 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
CN116209761A (zh) 2020-07-23 2023-06-02 豪夫迈·罗氏有限公司 靶向rna结合蛋白位点的寡核苷酸
WO2022109030A1 (en) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases

Family Cites Families (16)

* Cited by examiner, † Cited by third party
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
CA2145535C (en) 1992-09-25 2007-07-17 Axel Kahn Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain
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
US7291461B2 (en) * 2002-06-21 2007-11-06 Ptc Therapeutics, Inc. Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay
EP1874958B1 (en) * 2005-04-01 2019-03-27 University of Florida Research Foundation, Incorporated Biomarkers of liver injury
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
MX361732B (es) 2009-06-17 2018-12-14 Cold Spring Harbor Laboratory Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP3690048A1 (en) 2013-09-04 2020-08-05 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HOMEDAN CHADI ET AL: "Acute intermittent porphyria causes hepatic mitochondrial energetic failure in a mouse model", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, PERGAMON, GB, vol. 51, 13 April 2014 (2014-04-13), pages 93 - 101, XP029026620, ISSN: 1357-2725, DOI: 10.1016/J.BIOCEL.2014.03.032 *
JANA KRALOVICOVA ET AL: "Optimal antisense target reducing INS intron 1 retention is adjacent to a parallel G quadruplex", NUCLEIC ACIDS RESEARCH, vol. 42, no. 12, 8 July 2014 (2014-07-08), pages 8161 - 8173, XP055211568, ISSN: 0305-1048, DOI: 10.1093/nar/gku507 *
See also references of WO2017106283A1 *

Also Published As

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

Similar Documents

Publication Publication Date Title
EP3389672A4 (en) COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES
EP3341392A4 (en) COMPOSITIONS AND METHODS OF PAIN TREATMENT
EP3390666A4 (en) COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
MA43380A (fr) Récepteurs chimériques modifiés et compositions et procédés associés
MA47613A (fr) Compositions et procédés de traitement du cancer
EP3294065A4 (en) METHOD FOR THE TREATMENT OF CANCER
EP3319976A4 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF LUNG DISEASES AND LUNG LESIONS
MA43283A (fr) Procédés et compositions pour le traitement du cancer
EP2953950A4 (en) COMPOSITIONS AND METHOD FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
EP3426250A4 (en) TREATMENT PROCEDURE
EP2983791A4 (en) METHOD AND COMPOSITIONS FOR TREATING AUTOIMMUNE DISEASES
EP3331550A4 (en) METHOD FOR THE TREATMENT OF MYELOPROLIFERATIVE DISEASES
EP2909317A4 (en) METHOD AND COMPOSITIONS FOR TREATING AND COMBATING PLANT HAZARD
EP3408344A4 (en) METHOD AND COMPOSITIONS FOR BORING TREATMENT
EP3390642A4 (en) COMPOSITIONS AND METHODS OF TREATING RETINITIS PIGMENTOSA 18 AND RETINITIS PIGMENTOSA 13
EP3331612A4 (en) METHOD AND COMPOSITIONS FOR TUMOR THERAPY
EP3389657A4 (en) METHOD FOR THE TREATMENT OF HYPERALGESIA
EP3302207A4 (en) SURFACE TREATMENT DEVICES AND METHOD
IL249098A0 (en) Methods and preparations for the treatment of allergy and inflammatory diseases
EP3310174A4 (en) METHOD AND COMPOSITIONS FOR TREATING NON-SMALL CELLULAR CANCER
EP3442946A4 (en) METHOD FOR THE TREATMENT OF CANCER
EP3402575A4 (en) METHOD AND COMPOSITIONS FOR TREATING DAMAGED HAIR
EP2968478A4 (en) COMPOSITIONS AND METHOD FOR THE TREATMENT OF CARBOXYHEMOGLOBINEMIA
EP3309165A4 (en) SURFACE TREATMENT AGENT
EP3359553A4 (en) COMPOSITIONS AND METHODS OF TREATING DIABETIC RETINOPATHY

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

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

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20180706

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

AX Request for extension of the european patent

Extension state: BA ME

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
A4 Supplementary search report drawn up and despatched

Effective date: 20190712

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

REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1262957

Country of ref document: HK

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.

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: EXAMINATION IS IN PROGRESS

17Q First examination report despatched

Effective date: 20211215

GRAJ Information related to disapproval of communication of intention to grant by the applicant or resumption of examination proceedings by the epo deleted

Free format text: ORIGINAL CODE: EPIDOSDIGR1

GRAP Despatch of communication of intention to grant a patent

Free format text: ORIGINAL CODE: EPIDOSNIGR1

GRAP Despatch of communication of intention to grant a patent

Free format text: ORIGINAL CODE: EPIDOSNIGR1

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: GRANT OF PATENT IS INTENDED

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

INTG Intention to grant announced

Effective date: 20221006

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN

18D Application deemed to be withdrawn

Effective date: 20230217