JP7054675B2 - 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 - Google Patents

網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 Download PDF

Info

Publication number
JP7054675B2
JP7054675B2 JP2018529240A JP2018529240A JP7054675B2 JP 7054675 B2 JP7054675 B2 JP 7054675B2 JP 2018529240 A JP2018529240 A JP 2018529240A JP 2018529240 A JP2018529240 A JP 2018529240A JP 7054675 B2 JP7054675 B2 JP 7054675B2
Authority
JP
Japan
Prior art keywords
mrna
protein
prpf8
nucleobases
aso
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.)
Expired - Fee Related
Application number
JP2018529240A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500347A5 (cg-RX-API-DMAC7.html
JP2019500347A (ja
Inventor
アズナレズ，イザベル
エム． ナッシュ，ヒュー
クライナー，エイドリアン
Original Assignee
コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス，インク．
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 コールド スプリング ハーバー ラボラトリー, ストーク セラピューティクス，インク． filed Critical コールド スプリング ハーバー ラボラトリー
Publication of JP2019500347A publication Critical patent/JP2019500347A/ja
Publication of JP2019500347A5 publication Critical patent/JP2019500347A5/ja
Priority to JP2022062557A priority Critical patent/JP2022088621A/ja
Application granted granted Critical
Publication of JP7054675B2 publication Critical patent/JP7054675B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2018529240A 2015-12-14 2016-12-14 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 Expired - Fee Related JP7054675B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022062557A JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267261P 2015-12-14 2015-12-14
US62/267,261 2015-12-14
PCT/US2016/066684 WO2017106364A2 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022062557A Division JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Publications (3)

Publication Number Publication Date
JP2019500347A JP2019500347A (ja) 2019-01-10
JP2019500347A5 JP2019500347A5 (cg-RX-API-DMAC7.html) 2020-01-30
JP7054675B2 true JP7054675B2 (ja) 2022-04-14

Family

ID=59057547

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529240A Expired - Fee Related JP7054675B2 (ja) 2015-12-14 2016-12-14 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
JP2022062557A Pending JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022062557A Pending JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Country Status (5)

Country Link
EP (1) EP3390642B1 (cg-RX-API-DMAC7.html)
JP (2) JP7054675B2 (cg-RX-API-DMAC7.html)
CA (1) CA3005254A1 (cg-RX-API-DMAC7.html)
ES (1) ES2903394T3 (cg-RX-API-DMAC7.html)
WO (1) WO2017106364A2 (cg-RX-API-DMAC7.html)

Families Citing this family (15)

* 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
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
KR102422625B1 (ko) 2015-10-09 2022-07-20 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
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
AU2017229778A1 (en) 2016-03-09 2018-08-16 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
RU2019135845A (ru) 2017-04-11 2021-05-11 Ридженерон Фармасьютикалз, Инк. Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b)
CN111278991B (zh) 2017-08-25 2022-04-01 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP4234719B1 (en) 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
CN112218949B (zh) * 2018-03-02 2025-02-11 莱顿大学医学中心附属莱顿教学医院 多瘤病毒复制的抑制
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
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US20220112503A1 (en) * 2018-12-21 2022-04-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing pmp22 expression
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

Family Cites Families (13)

* 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
NZ256018A (en) 1992-09-25 1997-07-27 Rhone Poulenc Rorer Sa Recombinant adenoviral vectors and their use in directing expression and transcription of selected nucleotide sequences in cells of the central nervous system
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
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
JP5707396B2 (ja) 2009-06-17 2015-04-30 アイシス ファーマシューティカルズ， インコーポレーテッド 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法
ES2626488T3 (es) 2011-07-19 2017-07-25 Wave Life Sciences Pte. Ltd. Procedimientos para la síntesis de ácidos nucleicos funcionalizados
CN104004826B (zh) * 2013-01-07 2016-03-02 赵晨 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用
PL3041958T3 (pl) 2013-09-04 2020-11-02 Cold Spring Harbor Laboratory Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Human Molecular Genetics，2011年，Vol.20, No.11，p.2116-2130
IOVS，Vol.56，2015年10月，p.6362-6375
実験医学，2008年，Vol.26, No.10，p.1590-1595

Also Published As

Publication number Publication date
EP3390642A4 (en) 2019-06-26
EP3390642B1 (en) 2021-11-24
WO2017106364A3 (en) 2017-07-13
WO2017106364A2 (en) 2017-06-22
EP3390642A2 (en) 2018-10-24
ES2903394T3 (es) 2022-04-01
CA3005254A1 (en) 2017-06-22
JP2022088621A (ja) 2022-06-14
JP2019500347A (ja) 2019-01-10

Similar Documents

Publication Publication Date Title
JP7054675B2 (ja) 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
JP7049247B2 (ja) 腎臓病の処置のための組成物と方法
JP7051683B2 (ja) 結節性硬化症の処置のためのアンチセンスオリゴマー
EP3700570B1 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP2022106803A (ja) アラジール症候群の処置のためのアンチセンスオリゴマー
JP7049248B2 (ja) 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
JP2022046723A (ja) 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
JP2020189874A (ja) 状態および疾患の処置のためのアンチセンスオリゴマー
US20240254488A1 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP2021523227A (ja) 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
WO2023235509A2 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
CN117413061A (zh) 用于治疗与多囊蛋白表达相关的疾患和疾病的组合物
HK1262960B (en) Compositions for treatment of retinitis pigmentosa 13
CN120641565A (zh) 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚物
HK40038008B (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
HK40038008A (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
HK1262955B (en) Antisense oligomers for treatment of dravet syndrome

Legal Events

Date Code Title Description
RD03 Notification of appointment of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7423

Effective date: 20190531

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20190603

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191213

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20191213

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200819

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20200819

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20201105

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210205

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210430

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210830

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211130

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220128

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220228

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220303

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220404

R150 Certificate of patent or registration of utility model

Ref document number: 7054675

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees