CA3219795A1 - Recombinant tert-encoding viral genomes and vectors - Google Patents

Recombinant tert-encoding viral genomes and vectors Download PDF

Info

Publication number
CA3219795A1
CA3219795A1 CA3219795A CA3219795A CA3219795A1 CA 3219795 A1 CA3219795 A1 CA 3219795A1 CA 3219795 A CA3219795 A CA 3219795A CA 3219795 A CA3219795 A CA 3219795A CA 3219795 A1 CA3219795 A1 CA 3219795A1
Authority
CA
Canada
Prior art keywords
aav9
aav
promoter
spb
cmv
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
CA3219795A
Other languages
English (en)
French (fr)
Inventor
Maria BLASCO
Paula MARTINEZ
Maria Fatima Bosch Tubert
Veronica Jimenez Cenzano
Miquel Garcia Martinez
Estefania Casana Lorente
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.)
Telomere Therapeutics Sl
Universitat Autonòma de Barcelona
Fundacion del Sector Publico Estatal Centro Nacional de Investigaciones Oncologicas Carlos III FSP CNIO
Original Assignee
Telomere Therapeutics Sl
Universitat Autonòma de Barcelona
Fundacion del Sector Publico Estatal Centro Nacional de Investigaciones Oncologicas Carlos III FSP CNIO
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 Telomere Therapeutics Sl, Universitat Autonòma de Barcelona, Fundacion del Sector Publico Estatal Centro Nacional de Investigaciones Oncologicas Carlos III FSP CNIO filed Critical Telomere Therapeutics Sl
Publication of CA3219795A1 publication Critical patent/CA3219795A1/en
Pending 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
    • 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
    • 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
    • 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)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3219795A 2021-05-12 2022-05-12 Recombinant tert-encoding viral genomes and vectors Pending CA3219795A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21382441.0A EP4089171A1 (en) 2021-05-12 2021-05-12 Recombinant tert-encoding viral genomes and vectors
EP21382441.0 2021-05-12
PCT/EP2022/062990 WO2022238557A1 (en) 2021-05-12 2022-05-12 Recombinant tert-encoding viral genomes and vectors

Publications (1)

Publication Number Publication Date
CA3219795A1 true CA3219795A1 (en) 2022-11-17

Family

ID=75919286

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3219795A Pending CA3219795A1 (en) 2021-05-12 2022-05-12 Recombinant tert-encoding viral genomes and vectors

Country Status (9)

Country Link
US (2) US20250099618A1 (enExample)
EP (2) EP4089171A1 (enExample)
JP (1) JP2024521063A (enExample)
CN (1) CN117836420A (enExample)
AU (1) AU2022273863A1 (enExample)
CA (1) CA3219795A1 (enExample)
ES (1) ES3073712T3 (enExample)
MX (1) MX2023013385A (enExample)
WO (1) WO2022238557A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12668817B1 (en) * 2025-07-10 2026-06-30 Telomere Therapeutics S.L. Recombinant adeno-associated viral vectors for delivering a therapeutic gene to lungs

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
CA2188157A1 (en) * 1994-05-18 1995-11-23 Children's Hospital Medical Center Nucleic acid sequences controlling lung cell-specific gene expression
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
EP1007637B1 (en) 1997-04-14 2004-06-30 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
CN1247788C (zh) 1997-05-13 2006-03-29 北卡罗来纳-查佩尔山大学 基于慢病毒的基因转移载体
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
DE19909769A1 (de) 1999-03-05 2000-09-07 Bundesrepublik Deutschland Let Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
ES2590461T3 (es) * 2010-07-02 2016-11-22 Fundación Centro Nacional De Investigaciones Oncológicas Carlos Iii La transcriptasa inversa de la telomerasa como protección frente al envejecimiento
US9453209B2 (en) * 2012-12-27 2016-09-27 Sierra Sciences, Llc Enhancing health in mammals using telomerase reverse transcriptase gene therapy
ES2936383T3 (es) 2014-08-08 2023-03-16 Fundacion Del Sector Publico Estatal Centro Nac De Investigaciones Oncologicas Carlos Iii F S P Cnio Terapias basadas en transcriptasa inversa de la telomerasa para el tratamiento de afecciones asociadas con el infarto de miocardio
EP3485914B1 (en) 2014-08-08 2021-01-13 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Telomerase reverse transcriptase-based therapies

Also Published As

Publication number Publication date
US20260115322A1 (en) 2026-04-30
WO2022238557A1 (en) 2022-11-17
ES3073712T3 (en) 2026-07-14
EP4337780A1 (en) 2024-03-20
MX2023013385A (es) 2024-02-06
AU2022273863A1 (en) 2023-12-07
US20250099618A1 (en) 2025-03-27
EP4337780B1 (en) 2026-04-01
CN117836420A (zh) 2024-04-05
EP4089171A1 (en) 2022-11-16
JP2024521063A (ja) 2024-05-28
EP4337780C0 (en) 2026-04-01

Similar Documents

Publication Publication Date Title
JP7761686B2 (ja) 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
KR102743219B1 (ko) 아데노-관련 바이러스 변이체 캡시드 및 그 용도
CN114127089A (zh) 重组腺相关病毒及其用途
US20250001006A1 (en) Recombinant adeno-associated viruses for targeted delivery
WO2022076750A2 (en) Recombinant adeno-associated viruses for cns or muscle delivery
AU2018350700A1 (en) AAV vectors
AU2023202289A1 (en) Adeno-Associated Variants, Formulations And Methods For Pulmonary Delivery
JP2022505816A (ja) 小型化ジストロフィンおよびそれらの使用
IL293431A (en) Transgene cassettes designed to express human mecp2 gene
US20260055144A1 (en) Recombinant adeno-associated viruses and uses thereof
WO2026001133A1 (zh) 腺相关病毒突变体及其应用
US20260115322A1 (en) Recombinant tert-encoding viral genomes and vectors
KR20250120301A (ko) 심장 질환 및 심장 기능 장애 관련 질환 치료를 위해 간 친화성 감소 및 심장 세포 형질도입이 향상된 재조합-아데노 관련 바이러스 유전자 치료 벡터
KR20230082613A (ko) Cns 또는 근육 전달을 위한 재조합 아데노-연관 바이러스
HK40109651A (en) Recombinant tert-encoding viral genomes and vectors
HK40119094A (en) Adeno-associated virus variant capsids and methods of use thereof
WO2025023319A1 (ja) 脳指向性aav変異体
HK40121415A (en) Adeno-associated virus variant capsids with improved lung tropism and uses thereof
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用
HK40036161A (zh) 腺相关病毒变异衣壳和其使用方法
HK40036161B (en) Adeno-associated virus variant capsids and methods of use thereof

Legal Events

Date Code Title Description
P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20240916

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250507

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250507

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20260218

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260218

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20260304

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20260304

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260304