CA3165462A1 - Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia - Google Patents
Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia Download PDFInfo
- Publication number
- CA3165462A1 CA3165462A1 CA3165462A CA3165462A CA3165462A1 CA 3165462 A1 CA3165462 A1 CA 3165462A1 CA 3165462 A CA3165462 A CA 3165462A CA 3165462 A CA3165462 A CA 3165462A CA 3165462 A1 CA3165462 A1 CA 3165462A1
- Authority
- CA
- Canada
- Prior art keywords
- promoter
- seq
- functional fragment
- viral vector
- sequence identity
- 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
Links
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- 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
- A61K38/19—Cytokines; Lymphokines; Interferons
-
- 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
- A61K38/19—Cytokines; Lymphokines; Interferons
- A61K38/193—Colony stimulating factors [CSF]
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/53—Colony-stimulating factor [CSF]
- C07K14/535—Granulocyte CSF; Granulocyte-macrophage CSF
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/5434—IL-12
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/5443—IL-15
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/55—IL-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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/56—IFN-alpha
-
- 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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/57—IFN-gamma
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Toxicology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Psychiatry (AREA)
- Developmental Biology & Embryology (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20176939 | 2020-05-27 | ||
| EP20176939.5 | 2020-05-27 | ||
| PCT/EP2021/059070 WO2021239308A1 (en) | 2020-05-27 | 2021-04-07 | Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3165462A1 true CA3165462A1 (en) | 2021-12-02 |
Family
ID=70918223
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3165462A Pending CA3165462A1 (en) | 2020-05-27 | 2021-04-07 | Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230227847A1 (https=) |
| EP (1) | EP4158035A1 (https=) |
| JP (2) | JP7820823B2 (https=) |
| KR (1) | KR20230018378A (https=) |
| CN (1) | CN115552019A (https=) |
| AU (1) | AU2021280943A1 (https=) |
| BR (1) | BR112022016715A2 (https=) |
| CA (1) | CA3165462A1 (https=) |
| IL (1) | IL298093A (https=) |
| MX (1) | MX2022014766A (https=) |
| WO (1) | WO2021239308A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12071633B2 (en) | 2020-10-13 | 2024-08-27 | Kriya Therapeutics, Inc. | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| WO2023109911A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Microglia having car and use thereof |
| WO2024036106A1 (en) * | 2022-08-08 | 2024-02-15 | The Board Of Trustees Of The Leland Stanford Junior University | Embryo microglia complementation for in vivo microglia manipulation and production of a non-human animal model for validation of gene function and therapeutic screening |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6379674B1 (en) | 1997-08-12 | 2002-04-30 | Georgetown University | Use of herpes vectors for tumor therapy |
| US20110223635A1 (en) | 2008-08-11 | 2011-09-15 | Karl Deisseroth | Method and Composition for Controlling Gene Expression |
| WO2016026854A2 (en) | 2014-08-18 | 2016-02-25 | Apceth Gmbh & Co. Kg | Genetically modified mesenchymal stem cells expressing an immune response-stimulating cytokine to attract and/or activate immune cells |
| US20190328906A1 (en) | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| US20180186855A1 (en) * | 2016-03-23 | 2018-07-05 | Alector Llc | Chimeric receptors and methods of use thereof |
| WO2018169901A1 (en) * | 2017-03-13 | 2018-09-20 | Massachusetts Institute Of Technology | Synthetic promoters |
| BR112020001060A2 (pt) | 2017-07-27 | 2020-07-14 | Novartis Ag | variantes de trem2 resistentes a shedase |
| CN109868286B (zh) * | 2017-12-05 | 2022-09-16 | 深圳先进技术研究院 | 一种慢病毒载体、慢病毒及其制备方法和应用 |
| JP2021517168A (ja) | 2018-03-09 | 2021-07-15 | アブロバイオ,インコーポレーテッド | パーキンソン病を処置するための組成物及び方法 |
| US20190300870A1 (en) | 2018-04-03 | 2019-10-03 | The Broad Institute, Inc. | Target recognition motifs and uses thereof |
| WO2020160458A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| MX2022003934A (es) * | 2019-10-02 | 2022-04-25 | Univ Zuerich | Tratamiento de la enfermedad granulomatosa cronica. |
-
2021
- 2021-04-07 US US17/998,587 patent/US20230227847A1/en active Pending
- 2021-04-07 BR BR112022016715A patent/BR112022016715A2/pt unknown
- 2021-04-07 MX MX2022014766A patent/MX2022014766A/es unknown
- 2021-04-07 KR KR1020227041496A patent/KR20230018378A/ko active Pending
- 2021-04-07 CA CA3165462A patent/CA3165462A1/en active Pending
- 2021-04-07 IL IL298093A patent/IL298093A/en unknown
- 2021-04-07 CN CN202180034310.3A patent/CN115552019A/zh active Pending
- 2021-04-07 JP JP2022564746A patent/JP7820823B2/ja active Active
- 2021-04-07 AU AU2021280943A patent/AU2021280943A1/en active Pending
- 2021-04-07 WO PCT/EP2021/059070 patent/WO2021239308A1/en not_active Ceased
- 2021-04-07 EP EP21716740.2A patent/EP4158035A1/en active Pending
-
2025
- 2025-12-08 JP JP2025239487A patent/JP2026062601A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230018378A (ko) | 2023-02-07 |
| JP7820823B2 (ja) | 2026-02-26 |
| JP2023526773A (ja) | 2023-06-23 |
| EP4158035A1 (en) | 2023-04-05 |
| CN115552019A (zh) | 2022-12-30 |
| US20230227847A1 (en) | 2023-07-20 |
| BR112022016715A2 (pt) | 2022-11-16 |
| JP2026062601A (ja) | 2026-04-09 |
| AU2021280943A1 (en) | 2022-07-28 |
| IL298093A (en) | 2023-01-01 |
| MX2022014766A (es) | 2023-02-22 |
| WO2021239308A1 (en) | 2021-12-02 |
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