AU2021354969A1 - METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF - Google Patents

METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF Download PDF

Info

Publication number
AU2021354969A1
AU2021354969A1 AU2021354969A AU2021354969A AU2021354969A1 AU 2021354969 A1 AU2021354969 A1 AU 2021354969A1 AU 2021354969 A AU2021354969 A AU 2021354969A AU 2021354969 A AU2021354969 A AU 2021354969A AU 2021354969 A1 AU2021354969 A1 AU 2021354969A1
Authority
AU
Australia
Prior art keywords
promoter
mir
aimp2
seq
target sequence
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.)
Abandoned
Application number
AU2021354969A
Other languages
English (en)
Other versions
AU2021354969A9 (en
Inventor
Kyunghwa BAEK
Jin Woo Choi
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.)
Generoath Co Ltd
Original Assignee
Generoath 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 Generoath Co Ltd filed Critical Generoath Co Ltd
Publication of AU2021354969A1 publication Critical patent/AU2021354969A1/en
Publication of AU2021354969A9 publication Critical patent/AU2021354969A9/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1761—Apoptosis related proteins, e.g. Apoptotic protease-activating factor-1 (APAF-1), Bax, Bax-inhibitory protein(s)(BI; bax-I), Myeloid cell leukemia associated protein (MCL-1), Inhibitor of apoptosis [IAP] or Bcl-2
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C12N15/63—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
    • 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)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2021354969A 2020-09-30 2021-09-30 METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF Abandoned AU2021354969A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063085922P 2020-09-30 2020-09-30
US63/085,922 2020-09-30
PCT/IB2021/059018 WO2022070142A1 (en) 2020-09-30 2021-09-30 METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF

Publications (2)

Publication Number Publication Date
AU2021354969A1 true AU2021354969A1 (en) 2023-07-27
AU2021354969A9 AU2021354969A9 (en) 2024-09-19

Family

ID=80949802

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021354969A Abandoned AU2021354969A1 (en) 2020-09-30 2021-09-30 METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF

Country Status (8)

Country Link
US (1) US20240050527A1 (https=)
EP (1) EP4221760A4 (https=)
JP (1) JP2023544142A (https=)
KR (1) KR20230110503A (https=)
CN (1) CN116406304A (https=)
AU (1) AU2021354969A1 (https=)
CA (1) CA3192711A1 (https=)
WO (1) WO2022070142A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10716866B2 (en) 2018-03-29 2020-07-21 Generoath Co., Ltd Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
KR102248420B1 (ko) * 2019-03-15 2021-05-06 주식회사 제너로스 miR-142-3p의 표적 서열을 포함하는 재조합 벡터
CN119587566A (zh) * 2022-07-22 2025-03-11 郑州大学 一种干预或治疗视网膜色素变性的生物制剂

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20060057992A (ko) * 2004-11-24 2006-05-29 재단법인서울대학교산학협력재단 ｐ38-ＤＸ2 및 이의 용도
US7459529B2 (en) * 2004-11-24 2008-12-02 Seoul National University Industry Foundation AIMP2-DX2 and its uses
CA2609142C (en) * 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
KR101749138B1 (ko) * 2015-10-07 2017-06-20 원광대학교산학협력단 신경질환 예방 또는 치료를 위한 aimp2-dx2를 포함하는 약학 조성물 및 이의 용도
US10716866B2 (en) * 2018-03-29 2020-07-21 Generoath Co., Ltd Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
KR102248420B1 (ko) * 2019-03-15 2021-05-06 주식회사 제너로스 miR-142-3p의 표적 서열을 포함하는 재조합 벡터

Also Published As

Publication number Publication date
CA3192711A1 (en) 2022-04-07
CN116406304A (zh) 2023-07-07
JP2023544142A (ja) 2023-10-20
KR20230110503A (ko) 2023-07-24
WO2022070142A1 (en) 2022-04-07
EP4221760A1 (en) 2023-08-09
US20240050527A1 (en) 2024-02-15
EP4221760A4 (en) 2025-08-13

Similar Documents

Publication Publication Date Title
JP7503590B2 (ja) 遺伝子構築物
US20240050527A1 (en) METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF
JP6266811B2 (ja) 血管新生病の免疫療法
JP2003522160A (ja) 眼関連疾患の治療のための遺伝子治療
AU2022238492A1 (en) Ocular delivery of therapeutic agents
AU2021354996A9 (en) METHODS OF TREATING NEURONAL DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF
US20250281573A1 (en) Methods of Treating Retinal Degenerative Diseases Using AIMP2-DX2 and Optionally a Target Sequence for miR-142 and Compositions Thereof
EA048792B1 (ru) СПОСОБЫ ЛЕЧЕНИЯ ВОЗРАСТНОЙ МАКУЛОДИСТРОФИИ С ПРИМЕНЕНИЕМ AIMP2-DX2 И, НЕОБЯЗАТЕЛЬНО, ПОСЛЕДОВАТЕЛЬНОСТИ-МИШЕНИ ДЛЯ miR-142 И ИХ КОМПОЗИЦИЙ
EP2383286A1 (en) Methods and compositions for treatment of retinal degenerative diseases
US20220331241A1 (en) Treatment of x-linked juvenile retinoschisis
US20210308169A1 (en) Methods and compositions for treatment of retinal degenerative diseases
Chen et al. AAV2 Delivery of Flt23k Intraceptors Inhibits Murine Choroidal Neovascularization 2

Legal Events

Date Code Title Description
NB Applications allowed - extensions of time section 223(2)

Free format text: THE TIME IN WHICH TO ENTER THE NATIONAL PHASE HAS BEEN EXTENDED TO 30 JUN 2023

SREP Specification republished
MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period