JP7036723B2 - 眼疾患の処置のための組成物および方法 - Google Patents

眼疾患の処置のための組成物および方法 Download PDF

Info

Publication number
JP7036723B2
JP7036723B2 JP2018529249A JP2018529249A JP7036723B2 JP 7036723 B2 JP7036723 B2 JP 7036723B2 JP 2018529249 A JP2018529249 A JP 2018529249A JP 2018529249 A JP2018529249 A JP 2018529249A JP 7036723 B2 JP7036723 B2 JP 7036723B2
Authority
JP
Japan
Prior art keywords
mrna
seq
intron
aso
protein
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
JP2018529249A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500348A5 (enExample
JP2019500348A (ja
Inventor
アズナレズ，イザベル
エム． ナッシュ，ヒュー
アゼアリアン，サッサン
ダブリュ． ホール，サミュエル
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 JP2019500348A publication Critical patent/JP2019500348A/ja
Publication of JP2019500348A5 publication Critical patent/JP2019500348A5/ja
Priority to JP2022000109A priority Critical patent/JP2022046724A/ja
Application granted granted Critical
Publication of JP7036723B2 publication Critical patent/JP7036723B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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
    • C12N15/111—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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2018529249A 2015-12-14 2016-12-14 眼疾患の処置のための組成物および方法 Expired - Fee Related JP7036723B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000109A JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562267259P 2015-12-14 2015-12-14
US62/267,259 2015-12-14
US201662318958P 2016-04-06 2016-04-06
US62/318,958 2016-04-06
PCT/US2016/066691 WO2017106370A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of eye diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022000109A Division JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Publications (3)

Publication Number Publication Date
JP2019500348A JP2019500348A (ja) 2019-01-10
JP2019500348A5 JP2019500348A5 (enExample) 2020-01-30
JP7036723B2 true JP7036723B2 (ja) 2022-03-15

Family

ID=59057531

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529249A Expired - Fee Related JP7036723B2 (ja) 2015-12-14 2016-12-14 眼疾患の処置のための組成物および方法
JP2022000109A Pending JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022000109A Pending JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Country Status (4)

Country Link
EP (1) EP3390634A4 (enExample)
JP (2) JP7036723B2 (enExample)
CA (1) CA3005128A1 (enExample)
WO (1) WO2017106370A1 (enExample)

Families Citing this family (29)

* 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
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2018136702A1 (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
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CN111183234A (zh) 2017-10-11 2020-05-19 雷杰纳荣制药公司 在表达pnpla3 i148m变异的患者的肝病治疗中对hsd17b13的抑制
CN120290560A (zh) * 2017-10-23 2025-07-11 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
RU177889U1 (ru) * 2017-11-30 2018-03-15 Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") Дренаж для хирургического лечения глаукомы
KR20250154534A (ko) 2018-03-21 2025-10-28 리제너론 파마슈티칼스 인코포레이티드 17β-하이드록시스테로이드 데하이드로게나제 유형 13 (HSD17B13) iRNA 조성물 및 그것의 사용 방법
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
AU2019287635A1 (en) * 2018-06-14 2020-12-17 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
US11279929B2 (en) 2018-07-03 2022-03-22 Hoffmann-La Roche, Inc. Oligonucleotides for modulating Tau expression
JP2022523065A (ja) * 2019-01-25 2022-04-21 ナヤン セラピューティクス， インク． Ｎｒ２ｅ３発現抑制オリゴヌクレオチド、それを含む組成物、及びそれらの使用方法
JP2022543474A (ja) * 2019-08-08 2022-10-12 ユーシーエル ビジネス リミテッド アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする
JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
AU2021265838A1 (en) * 2020-04-29 2022-10-20 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
JP2023553710A (ja) 2020-12-18 2023-12-25 エフ． ホフマン－ラ ロシュ アーゲー プログラニュリンを標的とするためのアンチセンスオリゴヌクレオチド
CN118434858A (zh) * 2021-12-07 2024-08-02 豪夫迈·罗氏有限公司 靶向actl6b的反义寡核苷酸
JP2025506826A (ja) * 2022-02-25 2025-03-13 北京中因科技有限公司 発現カセット組み合わせ及びその使用
CA3252043A1 (en) 2022-05-13 2023-11-16 Ascidian Therapeutics, Inc. ABCA4 TRANS-SPLICING MOLECULES
EP4619047A1 (en) * 2022-11-17 2025-09-24 Skip Therapeutics Ltd. Compositions and methods for treating retinal diseases
WO2024223696A1 (en) 2023-04-24 2024-10-31 Universiteit Gent Compounds to treat inherited retinal disease
WO2025171404A1 (en) * 2024-02-09 2025-08-14 Utr Therapeutics Inc. Destabilized are 3' utrs of tead1 and yap1 as pan cancer therapeutics

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2597972C2 (ru) * 2010-10-22 2016-09-20 Курна Инк. Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена idua
WO2014121287A2 (en) * 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Journal of the Neurological Sciences，Vol.309，2011年，p.16-17
NATURE，2010年，Vol 465，p.223-227

Also Published As

Publication number Publication date
CA3005128A1 (en) 2017-06-22
JP2022046724A (ja) 2022-03-23
EP3390634A4 (en) 2019-08-14
JP2019500348A (ja) 2019-01-10
WO2017106370A1 (en) 2017-06-22
EP3390634A1 (en) 2018-10-24

Similar Documents

Publication Publication Date Title
JP7036723B2 (ja) 眼疾患の処置のための組成物および方法
US11096956B2 (en) Antisense oligomers and uses thereof
JP7049249B2 (ja) 中枢神経系疾患の処置のための組成物および方法
JP6395058B2 (ja) ハンチンチン対する、組成物及びその使用
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
CA3137740A1 (en) Oligonucleotide compositions and methods of use thereof
CN105209619B (zh) miR-204和miR-211及其用途
EP2900821B1 (en) Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
JP2019500345A (ja) 肝臓病の処置のための組成物および方法
TWI901675B (zh) 用於病症及疾病之治療的opa1反義寡聚物
Lentz et al. Deafness and retinal degeneration in a novel USH1C knock‐in mouse model
CN119731328A (zh) Abca4反式剪接分子
Palmer et al. Expression of Gtf2ird1, the Williams syndrome-associated gene, during mouse development
CN113134076B (zh) 一种利用转录因子再生具备功能的视网膜神经节细胞的方法
Millo et al. Identification of autosomal recessive novel genes and retinal phenotypes in members of the solute carrier (SLC) superfamily
Guziewicz et al. Molecular consequences of BEST1 gene mutations in canine multifocal retinopathy predict functional implications for human bestrophinopathies
JP2018526031A (ja) ＮＲＡＲＰ遺伝子の発現を阻害するためのｓｉＲＮＡ、並びにそのための方法及び組成物におけるそれらの使用
CN110066870A (zh) hsa-miR-382-5p在制备诊断视网膜变性疾病的试剂盒中的应用
JP2018524982A (ja) マイクロｒｎａ‐３２８アンチセンス組成物及び治療用途
CN107532181A (zh) Mirna模拟物和它们在治疗感觉病患中的用途
Marianelli et al. Retinal degeneration in spinocerebellar ataxia type 7: an overview of the current knowledge
KR102678740B1 (ko) Prox1 기능 억제제를 유효성분으로 포함하는, 망막신경 퇴행성 질환의 예방 또는 치료용 약학적 조성물
JP7682808B2 (ja) エクソンスキッピング戦略による網膜ジストロフィーの処置方法
CN120758573A (zh) 常染色体显性视神经萎缩动物模型的构建方法、试剂盒和应用
CN117866988A (zh) 核酸片段及其作为标志物在制备回旋状视网膜脉络膜萎缩的诊断试剂中的应用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180806

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

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210830

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220104

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20220104

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20220113

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20220114

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: 20220215

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220303

R150 Certificate of patent or registration of utility model

Ref document number: 7036723

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees