KR20220160052A - 분할 인테인 및 그 용도 - Google Patents

분할 인테인 및 그 용도 Download PDF

Info

Publication number
KR20220160052A
KR20220160052A KR1020227037261A KR20227037261A KR20220160052A KR 20220160052 A KR20220160052 A KR 20220160052A KR 1020227037261 A KR1020227037261 A KR 1020227037261A KR 20227037261 A KR20227037261 A KR 20227037261A KR 20220160052 A KR20220160052 A KR 20220160052A
Authority
KR
South Korea
Prior art keywords
fragment
intein
protein
degron
polynucleotide
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
KR1020227037261A
Other languages
English (en)
Korean (ko)
Inventor
도밍구에즈 실비아 프루토스
비달 제러드 카엘레즈
빌바오 아나벨 오테로
페렐로 미켈 빌라
Original Assignee
스플라이스바이오 에스.엘.
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 스플라이스바이오 에스.엘. filed Critical 스플라이스바이오 에스.엘.
Publication of KR20220160052A publication Critical patent/KR20220160052A/ko
Pending legal-status Critical Current

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
    • C12N15/63—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
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020227037261A 2020-03-26 2021-03-26 분할 인테인 및 그 용도 Pending KR20220160052A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20382236.6 2020-03-26
EP20382236.6A EP3885440A1 (en) 2020-03-26 2020-03-26 Split inteins and their uses
PCT/EP2021/058016 WO2021191447A1 (en) 2020-03-26 2021-03-26 Split inteins and their uses

Publications (1)

Publication Number Publication Date
KR20220160052A true KR20220160052A (ko) 2022-12-05

Family

ID=70292931

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227037261A Pending KR20220160052A (ko) 2020-03-26 2021-03-26 분할 인테인 및 그 용도

Country Status (10)

Country Link
US (1) US20230116688A1 (enExample)
EP (2) EP3885440A1 (enExample)
JP (1) JP2023518596A (enExample)
KR (1) KR20220160052A (enExample)
CN (1) CN115698307A (enExample)
AU (1) AU2021241899A1 (enExample)
BR (1) BR112022019167A2 (enExample)
CA (1) CA3175696A1 (enExample)
IL (1) IL296712A (enExample)
WO (1) WO2021191447A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
CA3152679A1 (en) * 2019-08-28 2021-03-04 The Trustees Of Princeton University Atypical split inteins and uses thereof
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
EP4424714A4 (en) * 2021-10-29 2025-10-15 Shanghai Sinobay Biotechnology Co Ltd Condition-Controlled Splicable Chimeric Antigen Receptor Molecule and Its Use
EP4527933A4 (en) * 2022-05-17 2025-12-24 Eye & Ent Hospital Of Fudan Univ DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE
KR20250141200A (ko) * 2023-02-01 2025-09-26 알렌 인스티튜트 전압-게이팅 나트륨 채널 기능의 인테인 매개 재구성
WO2024251077A1 (zh) 2023-06-07 2024-12-12 北京中因科技有限公司 表达盒组合及其应用
WO2025038955A1 (en) * 2023-08-16 2025-02-20 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
AU2024338787A1 (en) 2023-09-04 2026-03-19 Splicebio, Sl Use of split intein for the treatment of scn1a associated disease
WO2025153705A1 (en) 2024-01-18 2025-07-24 Splicebio, Sl Use of split intein for the treatment of myo7a-associated disease
WO2025193938A1 (en) * 2024-03-13 2025-09-18 Oregon Health & Science University Delivery of type iv collagen alpha chain using a split intein dual aav vector
WO2026013288A1 (en) 2024-07-12 2026-01-15 Splicebio, Sl Split intein for use in the treatment of cep290-associated disease
WO2026057767A1 (en) 2024-09-13 2026-03-19 Splicebio, Sl Use of split intein for the treatment of abca3-associated disease

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4945050A (en) 1984-11-13 1990-07-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues and apparatus therefor
US5036006A (en) 1984-11-13 1991-07-30 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues and apparatus therefor
US5100792A (en) 1984-11-13 1992-03-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2727679B1 (fr) 1994-12-05 1997-01-03 Rhone Poulenc Rorer Sa Nouveaux agents de transfection et leurs applications pharmaceutiques
FR2730637B1 (fr) 1995-02-17 1997-03-28 Rhone Poulenc Rorer Sa Composition pharmaceutique contenant des acides nucleiques, et ses utilisations
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
US11203621B2 (en) * 2015-06-19 2021-12-21 Riken PH-responsive proteolysis probe
WO2017132580A2 (en) * 2016-01-29 2017-08-03 The Trustees Of Princeton University Split inteins with exceptional splicing activity
JP2022512718A (ja) * 2018-10-15 2022-02-07 フォンダッツィオーネ・テレソン インテインタンパク質及びその使用

Also Published As

Publication number Publication date
JP2023518596A (ja) 2023-05-02
IL296712A (en) 2022-11-01
CN115698307A (zh) 2023-02-03
BR112022019167A2 (pt) 2022-11-01
US20230116688A1 (en) 2023-04-13
AU2021241899A1 (en) 2022-10-06
EP4127194A1 (en) 2023-02-08
WO2021191447A1 (en) 2021-09-30
EP3885440A1 (en) 2021-09-29
CA3175696A1 (en) 2021-09-30

Similar Documents

Publication Publication Date Title
US20230116688A1 (en) Split inteins and their uses
US11325956B2 (en) Dual-AAV vector-based systems and methods for delivering oversized genes to mammalian cells
AU2019258830B2 (en) Modified AAV capsid polypeptides for treatment of muscular diseases
AU2018290954B2 (en) Adeno-associated virus virions with variant capsids and methods of use thereof
CN113631182B (zh) 二硫键稳定的多肽组合物和使用方法
CN114127089A (zh) 重组腺相关病毒及其用途
WO2019076856A1 (en) AAV VECTORS
JP2008506363A (ja) ベクター内非相同末端パリンドローム配列を有するアデノ随伴ウイルスベクター
CN114008069A (zh) 外来体和aav的组合物
KR20230043794A (ko) 변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법
AU2017223465A1 (en) AAV-IDUA vector for treatment of MPS I-associated blindness
CN111718418B (zh) 一种增强基因编辑的融合蛋白及其应用
US20260055144A1 (en) Recombinant adeno-associated viruses and uses thereof
KR20240000542A (ko) 근이영양증의 유전자 요법을 위한 AAVrh74 벡터
Kochergin-Nikitsky et al. Tissue and cell-type-specific transduction using rAAV vectors in lung diseases
JP2024517957A (ja) ベクター系
CN117003833A (zh) 肝脏特异性的腺相关病毒血清型及应用
EP4337780B1 (en) Recombinant tert-encoding viral genomes and vectors
KR20250096825A (ko) 목적 유전자용 발현 카세트 및 이의 용도
WO2026057767A1 (en) Use of split intein for the treatment of abca3-associated disease
WO2024216244A9 (en) Targeting aav capsids, methods of manufacturing and using same
WO2026090552A1 (en) Methods of treating amyloid pathology
TW202603165A (zh) 用於治療杜興氏肌肉失養症之腺相關病毒組成物
TW202530248A (zh) 用於治療肌肉萎縮症之具有增強的肌動蛋白結合親和力之適應性人類肌肉萎縮蛋白
WO2024044691A9 (en) Modified aav vectors for gene therapy

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902