JP7660515B2 - 病態及び疾患の治療用アンチセンスオリゴマー - Google Patents

病態及び疾患の治療用アンチセンスオリゴマー Download PDF

Info

Publication number
JP7660515B2
JP7660515B2 JP2021550217A JP2021550217A JP7660515B2 JP 7660515 B2 JP7660515 B2 JP 7660515B2 JP 2021550217 A JP2021550217 A JP 2021550217A JP 2021550217 A JP2021550217 A JP 2021550217A JP 7660515 B2 JP7660515 B2 JP 7660515B2
Authority
JP
Japan
Prior art keywords
nucleotides
exon
fold
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.)
Active
Application number
JP2021550217A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2020176776A5 (enExample
JP2022522205A (ja
Inventor
アズナレズ，イザベル
Original Assignee
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 ストーク セラピューティクス，インク． filed Critical ストーク セラピューティクス，インク．
Publication of JP2022522205A publication Critical patent/JP2022522205A/ja
Publication of JPWO2020176776A5 publication Critical patent/JPWO2020176776A5/ja
Priority to JP2025060382A priority Critical patent/JP2025098252A/ja
Application granted granted Critical
Publication of JP7660515B2 publication Critical patent/JP7660515B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
    • 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)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pain & Pain Management (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2021550217A 2019-02-27 2020-02-27 病態及び疾患の治療用アンチセンスオリゴマー Active JP7660515B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025060382A JP2025098252A (ja) 2019-02-27 2025-04-01 病態及び疾患の治療用アンチセンスオリゴマー

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962811511P 2019-02-27 2019-02-27
US62/811,511 2019-02-27
PCT/US2020/020175 WO2020176776A1 (en) 2019-02-27 2020-02-27 Antisense oligomers for treatment of conditions and diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025060382A Division JP2025098252A (ja) 2019-02-27 2025-04-01 病態及び疾患の治療用アンチセンスオリゴマー

Publications (3)

Publication Number Publication Date
JP2022522205A JP2022522205A (ja) 2022-04-14
JPWO2020176776A5 JPWO2020176776A5 (enExample) 2023-03-07
JP7660515B2 true JP7660515B2 (ja) 2025-04-11

Family

ID=72239261

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021550217A Active JP7660515B2 (ja) 2019-02-27 2020-02-27 病態及び疾患の治療用アンチセンスオリゴマー
JP2025060382A Pending JP2025098252A (ja) 2019-02-27 2025-04-01 病態及び疾患の治療用アンチセンスオリゴマー

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025060382A Pending JP2025098252A (ja) 2019-02-27 2025-04-01 病態及び疾患の治療用アンチセンスオリゴマー

Country Status (10)

Country Link
US (1) US20220162605A1 (enExample)
EP (1) EP3931329A4 (enExample)
JP (2) JP7660515B2 (enExample)
KR (1) KR20210134003A (enExample)
CN (1) CN113748209A (enExample)
AU (1) AU2020227825A1 (enExample)
CA (1) CA3131591A1 (enExample)
IL (1) IL285867A (enExample)
SG (1) SG11202109371VA (enExample)
WO (1) WO2020176776A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
CA3159162A1 (en) * 2019-12-06 2021-06-10 Isabel AZNAREZ Antisense oligomers for treatment of conditions and diseases
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP6827148B2 (ja) 2017-08-25 2021-02-10 ストーク セラピューティクス，インク． 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

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2612180A1 (en) * 2005-06-16 2006-12-21 Bionomics Limited Methods of treatment, and diagnosis of epilepsy by detecting mutations in the scn1a gene
ES2863526T3 (es) * 2010-06-23 2021-10-11 Curna Inc Tratamiento de enfermedades relacionadas con la subunidad alfa del canal de sodio (SCNA), controlado por voltaje, mediante inhibición de la transcripción antisentido natural a SCNA

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP6827148B2 (ja) 2017-08-25 2021-02-10 ストーク セラピューティクス，インク． 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
JP7420679B2 (ja) 2017-08-25 2024-01-23 ストーク セラピューティクス，インク． 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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Am. J. Hum. Genet.，2018年，103, [6]，p.1022-1029

Also Published As

Publication number Publication date
CN113748209A (zh) 2021-12-03
JP2025098252A (ja) 2025-07-01
WO2020176776A1 (en) 2020-09-03
KR20210134003A (ko) 2021-11-08
SG11202109371VA (en) 2021-09-29
IL285867A (en) 2021-10-31
EP3931329A1 (en) 2022-01-05
US20220162605A1 (en) 2022-05-26
CA3131591A1 (en) 2020-09-03
AU2020227825A1 (en) 2021-10-07
JP2022522205A (ja) 2022-04-14
EP3931329A4 (en) 2023-12-13

Similar Documents

Publication Publication Date Title
US11873490B2 (en) Antisense oligomers for treatment of conditions and diseases
EP3700570B1 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP2019501892A (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
JP7660515B2 (ja) 病態及び疾患の治療用アンチセンスオリゴマー
JP2019500347A (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
US20240254488A1 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
EP4532723A2 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP2024507717A (ja) ポリシスチン発現に関連する状態及び疾患の治療のための組成物
HK40105710A (en) Antisense oligomers for treatment of conditions and diseases
HK40064034A (en) Antisense oligomers for treatment of conditions and diseases
HK40071463A (en) Antisense oligomers for treatment of conditions and diseases
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
EA045521B1 (ru) Антисмысловые олигомеры для лечения состояний и заболеваний

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230112

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230227

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230227

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240219

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240517

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240719

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240930

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241219

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241220

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: 20250303

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250401

R150 Certificate of patent or registration of utility model

Ref document number: 7660515

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150