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

ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME Download PDF

Info

Publication number
EP3389671A4
EP3389671A4 EP16876499.1A EP16876499A EP3389671A4 EP 3389671 A4 EP3389671 A4 EP 3389671A4 EP 16876499 A EP16876499 A EP 16876499A EP 3389671 A4 EP3389671 A4 EP 3389671A4
Authority
EP
European Patent Office
Prior art keywords
alagille
syndrome
treatment
antisense oligomers
antisense
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Withdrawn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
EP16876499.1A
Other languages
German (de)
English (en)
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.)
Filing date
Publication date
Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of EP3389671A1 publication Critical patent/EP3389671A1/en
Publication of EP3389671A4 publication Critical patent/EP3389671A4/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Withdrawn legal-status Critical Current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
    • 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

Landscapes

  • 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)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Pathology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
EP16876499.1A 2015-12-14 2016-12-13 ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME Withdrawn EP3389671A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267210P 2015-12-14 2015-12-14
PCT/US2016/066414 WO2017106210A1 (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
EP3389671A4 true EP3389671A4 (en) 2019-07-17

Family

ID=59057483

Family Applications (1)

Application Number Title Priority Date Filing Date
EP16876499.1A Withdrawn EP3389671A4 (en) 2015-12-14 2016-12-13 ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME

Country Status (4)

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

Families Citing this family (17)

* 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
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3359685A1 (en) 2015-10-09 2018-08-15 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
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
KR102712656B1 (ko) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
CN110536964A (zh) 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物
US11479802B2 (en) 2017-04-11 2022-10-25 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxy steroid (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
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
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
CN115976028B (zh) * 2018-03-09 2025-03-25 第一三共株式会社 糖原病Ia型治疗药
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
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 (pt) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. métodos e composições para tratamento de doença de armazenamento de éster de colesteril
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040102401A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of jagged 1 expression
WO2005049651A2 (en) * 2003-11-14 2005-06-02 The Queen's University Of Belfast Cancer diagnosis and therapy
WO2007048629A2 (en) * 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
WO2007048628A2 (en) * 2005-10-28 2007-05-03 Max Planck Gesellschaft Structures of active guide rna molecules and method of selection

Family Cites Families (12)

* 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
FI951404A7 (fi) 1992-09-25 1995-03-24 Rhone Poulenc Rorer Sa Adenovirusvektoreita vieraiden geenien siirtämiseksi keskushermostojärjestelmän , erityisesti aivojen, soluihin
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
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
EP3705125B1 (en) 2007-12-04 2023-07-05 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
SI3449926T1 (sl) 2009-06-17 2020-04-30 Biogen Ma Inc. Sestave in metode za modulacijo združevanja SMN2 pri subjektu
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
CA2930859C (en) 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040102401A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of jagged 1 expression
WO2005049651A2 (en) * 2003-11-14 2005-06-02 The Queen's University Of Belfast Cancer diagnosis and therapy
WO2007048629A2 (en) * 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
WO2007048628A2 (en) * 2005-10-28 2007-05-03 Max Planck Gesellschaft Structures of active guide rna molecules and method of selection

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
GIUSEPPE PILIA ET AL: "Jagged-1 mutation analysis in Italian Alagille syndrome patients", HUMAN MUTATION, vol. 14, no. 5, 22 October 1999 (1999-10-22), US, pages 394 - 400, XP055587922, ISSN: 1059-7794, DOI: 10.1002/(SICI)1098-1004(199911)14:5<394::AID-HUMU5>3.0.CO;2-1 *
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, 17 June 2014 (2014-06-17), pages 8161 - 8173, XP055211568, ISSN: 0305-1048, DOI: 10.1093/nar/gku507 *
LITING LI ET AL: "JAG1 Mutation Spectrum and Origin in Chinese Children with Clinical Features of Alagille Syndrome", PLOS ONE, vol. 10, no. 6, 15 June 2015 (2015-06-15), pages e0130355, XP055587896, DOI: 10.1371/journal.pone.0130355 *
M. YAMAMOTO ET AL: "Mib-Jag1-Notch signalling regulates patterning and structural roles of the notochord by controlling cell-fate decisions", DEVELOPMENT, vol. 137, no. 15, 23 June 2010 (2010-06-23), GB, pages 2527 - 2537, XP055587656, ISSN: 0950-1991, DOI: 10.1242/dev.051011 *
NANCY B. SPINNER ET AL: "Jagged1 mutations in Alagille syndrome", HUMAN MUTATION, vol. 17, no. 1, 22 December 2000 (2000-12-22), US, pages 18 - 33, XP055587899, ISSN: 1059-7794, DOI: 10.1002/1098-1004(2001)17:1<18::AID-HUMU3>3.0.CO;2-T *
PETER D TURNPENNY ET AL: "Alagille syndrome: pathogenesis, diagnosis and management", EUROPEAN JOURNAL OF HUMAN GENETICS., vol. 20, no. 3, 21 September 2011 (2011-09-21), CH, pages 251 - 257, XP055590188, ISSN: 1018-4813, DOI: 10.1038/ejhg.2011.181 *
RYSZARD KOLE ET AL: "RNA therapeutics: beyond RNA interference and antisense oligonucleotides", NATURE REVIEWS DRUG DISCOVERY, vol. 11, no. 2, 20 January 2012 (2012-01-20), pages 125 - 40, XP055138361, ISSN: 1474-1776, DOI: 10.1038/nrd3625 *
See also references of WO2017106210A1 *

Also Published As

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

Similar Documents

Publication Publication Date Title
EP3389671A4 (en) ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME
EP3390635A4 (en) Antisense oligomers for the treatment of a tuberous sclerosis complex
IL250016B (en) Antisense oligonucleotides for the treatment of usher syndrome type 2
EP3458107A4 (en) FOR JAGGED1-CODING POLYNUCLEOTIDES FOR TREATING ALAGILLE SYNDROME
PL3673080T3 (pl) Oligomery antysensowne do leczenia stanów i chorób
MA45539A (fr) Dihydropyranopyrimidines pour le traitement d&#39;infections virales
MA47719A (fr) Esketamine pour le traitement de la dépression
EP3374502A4 (en) METHOD FOR THE TREATMENT OF CORNEAL DYSTROPHIES
EP3389782A4 (en) ANTISENSE OLIGOMERS FOR THE TREATMENT OF A POLYCYSTIC KIDNEY DISEASE
MA40867A (fr) Méthodes pour le traitement d&#39;infections virales à filoviridae
EP3390636A4 (en) ANTISENSE OLIGOMERS FOR THE TREATMENT OF AUTOSOMAL-DOMINANT MENTAL RETARDATION 5 AND DRAVET SYNDROME
LT3261640T (lt) 5ht agonistai epilepsijos sutrikimų gydymui
MA39748A (fr) Cenicriviroc pour le traitement de la fibrose
GB201616202D0 (en) Antisense oligonucleotides for the treatment of eye deisease
EP3504187A4 (en) USE OF PRIDOPIDIN FOR THE TREATMENT OF FUNCTIONAL DECAY
EP3233878A4 (en) PHOSPHORAMIDATE FOR THE TREATMENT OF HEPATITIS C VIRUS
MA41449A (fr) Polythérapies pour le traitement de cancers
EP3380200A4 (en) COMPOSITIONS FOR TREATING THE HAIR
EP3717646A4 (en) ANTISENSE TREATMENT OF ANGELMAN&#39;S SYNDROME
IL269735A (en) Antisense oligonucleotides for the treatment of stargardt disease
MA42999A (fr) Polythérapie pour le traitement de malignités
EP3380063A4 (en) COMPOSITIONS FOR TREATING THE HAIR
EP3335041A4 (en) BIOMARKER FOR THE TREATMENT OF ALOPECIA AREATA
EP3334432A4 (en) CERDULATINIB FOR THE TREATMENT OF MYELOMA
EP3512506A4 (en) USE OF PRIDOPIDIN TO TREAT THE RETT SYNDROME

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

RIN1 Information on inventor provided before grant (corrected)

Inventor name: NASH, HUW M.

Inventor name: AZNAREZ, ISABEL

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
REG Reference to a national code

Ref country code: DE

Ref legal event code: R079

Free format text: PREVIOUS MAIN CLASS: A61K0031708800

Ipc: C12N0015113000

A4 Supplementary search report drawn up and despatched

Effective date: 20190614

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 15/11 20060101ALI20190607BHEP

Ipc: A61K 31/7088 20060101ALI20190607BHEP

Ipc: C12N 15/113 20100101AFI20190607BHEP

Ipc: A61P 1/16 20060101ALI20190607BHEP

Ipc: C12Q 1/68 20180101ALI20190607BHEP

REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 1262747

Country of ref document: HK

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: 20200227

RIN1 Information on inventor provided before grant (corrected)

Inventor name: NASH, HUW M.

Inventor name: KRAINER, ADRIAN

Inventor name: AZNAREZ, ISABEL

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

INTG Intention to grant announced

Effective date: 20230803

RIN1 Information on inventor provided before grant (corrected)

Inventor name: KRAINER, ADRIAN

Inventor name: NASH, HUW M.

Inventor name: AZNAREZ, ISABEL

P01 Opt-out of the competence of the unified patent court (upc) registered

Effective date: 20230808

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: 20231214