CN117597151A - 强直性肌肉营养不良1型治疗药 - Google Patents

强直性肌肉营养不良1型治疗药 Download PDF

Info

Publication number
CN117597151A
CN117597151A CN202280030329.5A CN202280030329A CN117597151A CN 117597151 A CN117597151 A CN 117597151A CN 202280030329 A CN202280030329 A CN 202280030329A CN 117597151 A CN117597151 A CN 117597151A
Authority
CN
China
Prior art keywords
glu
lys
leu
gly
asp
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.)
Pending
Application number
CN202280030329.5A
Other languages
English (en)
Chinese (zh)
Inventor
中森雅之
八木祐介
今井崇喜
冲井英里香
玉井乔之
二宫理纱
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.)
Japanese Gene Editing Co ltd
Original Assignee
Japanese Gene Editing Co ltd
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 Japanese Gene Editing Co ltd filed Critical Japanese Gene Editing Co ltd
Publication of CN117597151A publication Critical patent/CN117597151A/zh
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/168—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from plants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/415—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from plants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202280030329.5A 2021-04-30 2022-04-27 强直性肌肉营养不良1型治疗药 Pending CN117597151A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2021-077262 2021-04-30
JP2021077262 2021-04-30
PCT/JP2022/019038 WO2022230924A1 (ja) 2021-04-30 2022-04-27 筋強直性ジストロフィー１型治療薬

Publications (1)

Publication Number Publication Date
CN117597151A true CN117597151A (zh) 2024-02-23

Family

ID=83848514

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280030329.5A Pending CN117597151A (zh) 2021-04-30 2022-04-27 强直性肌肉营养不良1型治疗药

Country Status (12)

Country Link
US (1) US20250281571A1 (https=)
EP (1) EP4331619A4 (https=)
JP (2) JP7461687B2 (https=)
KR (1) KR20240004715A (https=)
CN (1) CN117597151A (https=)
AU (1) AU2022265246A1 (https=)
BR (1) BR112023022129A2 (https=)
CA (1) CA3216313A1 (https=)
IL (1) IL308029A (https=)
TW (1) TW202304948A (https=)
WO (1) WO2022230924A1 (https=)
ZA (1) ZA202309721B (https=)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140335521A1 (en) * 2011-10-21 2014-11-13 Kyushu University, National University Corporation Method for Designing RNA Binding Protein Utilizing PPR Motif, and Use Thereof
US20210024597A1 (en) * 2014-04-14 2021-01-28 Association Institut De Myologie Treatment of myotonic dystrophy

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2900821B1 (en) * 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
EP3498726A4 (en) * 2016-08-10 2020-03-25 Fujifilm Wako Pure Chemical Corporation DNA BINDING PROTEIN USING A PPR PATTERN AND USE OF SAID DNA BINDING PROTEIN
WO2021201198A1 (ja) * 2020-03-31 2021-10-07 エディットフォース株式会社 標的ｒｎａを編集する方法
US20230193265A1 (en) * 2020-05-20 2023-06-22 Osaka University Cug repeat sequence binding agent

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140335521A1 (en) * 2011-10-21 2014-11-13 Kyushu University, National University Corporation Method for Designing RNA Binding Protein Utilizing PPR Motif, and Use Thereof
US20210024597A1 (en) * 2014-04-14 2021-01-28 Association Institut De Myologie Treatment of myotonic dystrophy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
IMAI T等: "Development of an AAV-mediated Pentatricopeptide Repeat Protein (PPR) delivery system for treatment of myotonic dystrophy type 1 (DM1)", 《NEUROMUSCULAR DISORDERS》, 7 October 2023 (2023-10-07), pages 154 *
ONDONO R等: "Design of novel small molecule base-pair recognizers of toxic CUG RNA transcripts characteristics of DM1", 《COMPUT STRUCT BIOTECHNOL J》, 6 December 2020 (2020-12-06), pages 51 - 61 *
张文静: "人造特异性RNA内切酶治疗1型强直性肌营养不良症及优化PUF结构域的初步研究", 《中国优秀博士论文全文数据库》, 15 January 2015 (2015-01-15), pages 1 - 116 *

Also Published As

Publication number Publication date
JP2024069430A (ja) 2024-05-21
JPWO2022230924A1 (https=) 2022-11-03
BR112023022129A2 (pt) 2024-01-09
EP4331619A1 (en) 2024-03-06
JP7854207B2 (ja) 2026-05-01
CA3216313A1 (en) 2022-11-03
AU2022265246A1 (en) 2023-11-02
EP4331619A4 (en) 2024-12-25
IL308029A (en) 2023-12-01
WO2022230924A1 (ja) 2022-11-03
TW202304948A (zh) 2023-02-01
US20250281571A1 (en) 2025-09-11
JP7461687B2 (ja) 2024-04-04
KR20240004715A (ko) 2024-01-11
ZA202309721B (en) 2025-03-26

Similar Documents

Publication Publication Date Title
KR102398039B1 (ko) 선택적 유전자 치료 발현 시스템
JP7416451B2 (ja) ＣＲＩＳＰＲ－Ｃａｓによる標的化された核内ＲＮＡ切断及びポリアデニル化
JP7667595B2 (ja) Ａｑｐ１ ＲＮＡを標的とするｓｇＲＮＡ及びそのベクターと使用
KR20230003511A (ko) 안면견갑상완 근이영양증에 대한 crispr-억제
CN113785066B (zh) 通过靶向dmpk基因治疗肌营养不良症的方法
WO2017151717A1 (en) Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells
CN117580941A (zh) 多重CRISPR/Cas9介导的靶基因激活系统
JP2011504375A (ja) 細胞の中に核酸を導入する医薬組成物及びその方法
TW202112797A (zh) 經由靶向lama1基因治療肌肉萎縮症的方法
KR20230123925A (ko) Neurod1 및 dlx2 벡터
CN104721811A (zh) 多肽在制备防治脑胶质瘤药物中的应用
CN115443154A (zh) 用于基因上调的miRNA-485抑制剂
CN117597151A (zh) 强直性肌肉营养不良1型治疗药
TW202539725A (zh) 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子
US20060070133A1 (en) Methods and compositions for importing nucleic acids into cell nuclei
WO2016181011A1 (es) Método para promover la regeneración muscular
KR102236843B1 (ko) 새로운 재생 치료제로서의 camkk1
HK40101152A (zh) 强直性肌肉营养不良1型治疗药
CN116368223A (zh) siRNA及其用途
HK40101760A (en) Therapeutic drug for myotonic dystrophy type 1
CN113227375A (zh) 合成的微小rna模拟物
WO2020139151A1 (en) Gene therapy dna vector based on gene therapy dna vector vtvaf17
CN102355911A (zh) 调节脂肪生成的Vgll3活性调节剂的用途
US20130143954A1 (en) Recombinant vector for suppressing proliferation of human papilloma virus cells including adenylate cyclase activating polypeptide 1 (pituitary) gene and pharmaceutical composition for treating human papilloma virus
CN116761812A (zh) Neurod1和dlx2载体

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40101152

Country of ref document: HK