CA3132714A1 - Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy - Google Patents

Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy Download PDF

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Publication number
CA3132714A1
CA3132714A1 CA3132714A CA3132714A CA3132714A1 CA 3132714 A1 CA3132714 A1 CA 3132714A1 CA 3132714 A CA3132714 A CA 3132714A CA 3132714 A CA3132714 A CA 3132714A CA 3132714 A1 CA3132714 A1 CA 3132714A1
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Canada
Prior art keywords
oncolytic virus
replicon rna
virus
synthetic oncolytic
synthetic
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Pending
Application number
CA3132714A
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English (en)
French (fr)
Inventor
Darrell J. Irvine
Karl Dane Wittrup
Ron Weiss
Yingzhong Li
Noor MOMIN
Yizhou Dong
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.)
Massachusetts Institute of Technology
Ohio State Innovation Foundation
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Massachusetts Institute of Technology
Ohio State University
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Application filed by Massachusetts Institute of Technology, Ohio State University filed Critical Massachusetts Institute of Technology
Publication of CA3132714A1 publication Critical patent/CA3132714A1/en
Pending legal-status Critical Current

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/768Oncolytic viruses not provided for in groups A61K35/761 - A61K35/766
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/20Interleukins [IL]
    • A61K38/208IL-12
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/54Interleukins [IL]
    • C07K14/5434IL-12
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/36011Togaviridae
    • C12N2770/36111Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/36011Togaviridae
    • C12N2770/36111Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Oncology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3132714A 2019-03-08 2020-01-10 Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy Pending CA3132714A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962815611P 2019-03-08 2019-03-08
US62/815,611 2019-03-08
PCT/US2020/013069 WO2020185293A1 (en) 2019-03-08 2020-01-10 Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy

Publications (1)

Publication Number Publication Date
CA3132714A1 true CA3132714A1 (en) 2020-09-17

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CA3132714A Pending CA3132714A1 (en) 2019-03-08 2020-01-10 Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy

Country Status (14)

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US (2) US11717548B2 (en])
EP (1) EP3934673A1 (en])
JP (2) JP7561134B2 (en])
KR (1) KR20220006041A (en])
CN (1) CN113966221A (en])
AU (1) AU2020233788A1 (en])
BR (1) BR112021017637A8 (en])
CA (1) CA3132714A1 (en])
CO (1) CO2021013417A2 (en])
IL (1) IL285963A (en])
MX (1) MX2021010808A (en])
PH (1) PH12021552145A1 (en])
SG (1) SG11202109514VA (en])
WO (1) WO2020185293A1 (en])

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* Cited by examiner, † Cited by third party
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WO2020185293A1 (en) * 2019-03-08 2020-09-17 Massachusetts Institute Of Technology Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy
CN113509542A (zh) * 2021-04-20 2021-10-19 嘉晨西海(杭州)生物技术有限公司 一种基于mRNA的表达白介素12针对肿瘤的药物及其制备方法
CZ310443B6 (cs) * 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
US20250152737A1 (en) 2022-04-26 2025-05-15 Strand Therapeutics Inc. Lipid nanoparticles comprising venezuelan equine encephalitis (vee) replicon and uses thereof
CN115227674B (zh) * 2022-08-05 2023-07-04 武汉滨会生物科技股份有限公司 包封的溶瘤病毒遗传物质及其应用
AU2024208643A1 (en) * 2023-01-12 2025-06-26 Bavarian Nordic A/S RECOMBINANT MODIFIED saRNA (VRP) FOR CANCER VACCINE
WO2024211865A2 (en) * 2023-04-07 2024-10-10 Omega Therapeutics, Inc. Lipids and lipid-like compounds for therapeutic lipid nanoparticle (lnp) delivery
CN118147171B (zh) * 2024-03-08 2024-12-06 上海复诺健生物科技有限公司 具有增强的外源基因表达水平的自扩增mRNA核酸序列
WO2025024704A1 (en) 2023-07-25 2025-01-30 Strand Therapeutics Inc. Polynucleotides comprising a micro rna detargeting sensor and uses thereof
CN119479778B (zh) * 2024-10-16 2025-04-25 北京悦康科创医药科技股份有限公司 一种lnp转染效率预测模型构建方法及装置

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US4544545A (en) 1983-06-20 1985-10-01 Trustees University Of Massachusetts Liposomes containing modified cholesterol for organ targeting
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EP2451475A2 (en) * 2009-07-06 2012-05-16 Novartis AG Self replicating rna molecules and uses thereof
HRP20161352T1 (hr) * 2010-07-06 2016-12-02 Glaxosmithkline Biologicals Sa Čestice nalik na virione za unos samoreplicirajućih molekula rna
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WO2020185293A1 (en) * 2019-03-08 2020-09-17 Massachusetts Institute Of Technology Synthetic oncolytic lnp-replicon rna and uses for cancer immunotherapy
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JP2022524391A (ja) 2022-05-02
US20240024394A1 (en) 2024-01-25
IL285963A (en) 2021-10-31
US12383589B2 (en) 2025-08-12
JP2025011109A (ja) 2025-01-23
BR112021017637A2 (en]) 2021-11-09
JP7561134B2 (ja) 2024-10-03
EP3934673A1 (en) 2022-01-12
KR20220006041A (ko) 2022-01-14
US20200281994A1 (en) 2020-09-10
AU2020233788A1 (en) 2021-10-07
MX2021010808A (es) 2021-12-15
BR112021017637A8 (pt) 2022-08-16
PH12021552145A1 (en) 2022-08-31
US11717548B2 (en) 2023-08-08
CN113966221A (zh) 2022-01-21
WO2020185293A1 (en) 2020-09-17
CO2021013417A2 (es) 2022-01-17
SG11202109514VA (en) 2021-09-29

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