CL2021003201A1 - Vectores aav con el promotor de la proteína cero de mielina y usos de los mismos para el tratamiento de enfermedades asociadas a las células de schwann como la enfermedad de charcot-marie-tooth. - Google Patents
Vectores aav con el promotor de la proteína cero de mielina y usos de los mismos para el tratamiento de enfermedades asociadas a las células de schwann como la enfermedad de charcot-marie-tooth.Info
- Publication number
- CL2021003201A1 CL2021003201A1 CL2021003201A CL2021003201A CL2021003201A1 CL 2021003201 A1 CL2021003201 A1 CL 2021003201A1 CL 2021003201 A CL2021003201 A CL 2021003201A CL 2021003201 A CL2021003201 A CL 2021003201A CL 2021003201 A1 CL2021003201 A1 CL 2021003201A1
- Authority
- CL
- Chile
- Prior art keywords
- treatment
- charcot
- marie
- associated diseases
- myelin
- Prior art date
Links
- 210000004116 schwann cell Anatomy 0.000 title abstract 4
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 title abstract 2
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 102000006386 Myelin Proteins Human genes 0.000 title 1
- 108010083674 Myelin Proteins Proteins 0.000 title 1
- 210000005012 myelin Anatomy 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 230000002265 prevention Effects 0.000 abstract 2
- 230000003210 demyelinating effect Effects 0.000 abstract 1
- 230000007823 neuropathy Effects 0.000 abstract 1
- 201000001119 neuropathy Diseases 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
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
- 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
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- 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
- A61K48/0075—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
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- 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
- C07K14/70503—Immunoglobulin superfamily
-
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cell Biology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1907882.3A GB201907882D0 (en) | 2019-06-03 | 2019-06-03 | Methods |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021003201A1 true CL2021003201A1 (es) | 2023-02-03 |
Family
ID=67385825
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021003201A CL2021003201A1 (es) | 2019-06-03 | 2021-12-01 | Vectores aav con el promotor de la proteína cero de mielina y usos de los mismos para el tratamiento de enfermedades asociadas a las células de schwann como la enfermedad de charcot-marie-tooth. |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12582725B2 (https=) |
| EP (1) | EP3976800A1 (https=) |
| JP (1) | JP7692369B2 (https=) |
| KR (1) | KR20220016485A (https=) |
| CN (1) | CN114026242A (https=) |
| AU (1) | AU2020288605A1 (https=) |
| BR (1) | BR112021024285A2 (https=) |
| CA (1) | CA3142211A1 (https=) |
| CL (1) | CL2021003201A1 (https=) |
| CO (1) | CO2021016487A2 (https=) |
| EA (1) | EA202290001A1 (https=) |
| GB (1) | GB201907882D0 (https=) |
| IL (1) | IL288462A (https=) |
| MX (1) | MX2021014789A (https=) |
| SG (1) | SG11202112889VA (https=) |
| WO (1) | WO2020245169A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240076665A1 (en) * | 2020-12-22 | 2024-03-07 | Wisconsin Alumni Research Foundation | Regulatory elements for schwann cell-specific gene expression |
| CA3234702A1 (en) | 2021-10-07 | 2023-04-13 | Research Institute At Nationwide Children's Hospital | Products and methods for myelin protein zero silencing and treating cmt1b disease |
| JP2025502269A (ja) * | 2022-01-13 | 2025-01-24 | ツールゲン インコーポレイテッド | シュワン細胞特異的プロモーター |
| GB202303495D0 (en) * | 2023-03-09 | 2023-04-26 | Ucl Business Ltd | Therapy for charcot-marie-tooth disease |
| WO2025105734A1 (ko) * | 2023-11-15 | 2025-05-22 | 주식회사 카인사이언스 | 탈수초 질환 치료용 펩타이드 및 이의 용도 |
| WO2025174135A1 (ko) * | 2024-02-16 | 2025-08-21 | 주식회사 엣진 | 샤르코-마리-투스 질환 치료를 위한 징크 핑거 단백질 및 이를 발현시키는 방법 |
| CN118291543A (zh) * | 2024-02-29 | 2024-07-05 | 神拓生物技术(杭州)有限公司 | 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用 |
| KR20260001543A (ko) * | 2024-06-25 | 2026-01-06 | 연세대학교 산학협력단 | 바이러스 벡터를 포함한 난청 치료용 조성물 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL210751B1 (pl) | 2006-09-12 | 2012-02-29 | Anna Jurewicz | Izolowany fragment białka Nogo-A, jego zastosowanie oraz sposób diagnozowania oraz leczenia stwardnienia rozsianego i polineuropatii zapalno-demielinizacyjnej |
| US20090018031A1 (en) | 2006-12-07 | 2009-01-15 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways tools, and methods |
| US20090215178A1 (en) | 2008-02-22 | 2009-08-27 | Zequn Tang | Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines |
| CA2730785A1 (en) | 2008-07-16 | 2010-01-21 | The Board Of Trustees Of The Leland Stanford Junior University | Modulation of gm98 (mrf) in remyelination |
| AU2010289419A1 (en) | 2009-09-02 | 2012-03-29 | The University Of Chicago | Methods and systems for inducible ablation of neural cells |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| AU2012258558A1 (en) | 2011-05-26 | 2013-05-02 | Biogen Ma Inc. | Methods of treating multiple sclerosis and preserving and/or increasing myelin content |
| DK2820042T3 (da) | 2012-02-29 | 2019-10-28 | Baxalta GmbH | Igg-stimuleret remyelinisering af perifere nerver |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| US9644215B2 (en) * | 2013-04-12 | 2017-05-09 | The General Hospital Corporation | AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors |
| WO2015140351A1 (en) | 2014-03-21 | 2015-09-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for enhancing myelination |
| EP2933335A1 (en) * | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| US10675382B2 (en) | 2014-09-12 | 2020-06-09 | Kyoto Prefectural Public University Corporation | Schwann cells and method for preparing same |
| EP3286318A2 (en) | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| RU2766200C1 (ru) | 2015-10-01 | 2022-02-09 | Хит Байолоджикс, Инк. | Композиции и способы для соединения внеклеточных доменов типа i и типа ii в качестве гетерологичных химерных белков |
| JP7064197B2 (ja) | 2016-09-23 | 2022-05-10 | 国立大学法人大阪大学 | シュワン細胞分化促進剤及び末梢神経再生促進剤 |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| WO2018088694A2 (ko) | 2016-11-14 | 2018-05-17 | 주식회사 툴젠 | 인위적으로 조작된 sc 기능 조절 시스템 |
| WO2018106753A1 (en) * | 2016-12-07 | 2018-06-14 | The General Hospital Corporation | Methods and compositions relating the treatment of tumors |
| EP3635121A1 (en) | 2017-06-02 | 2020-04-15 | Institut National de la Sante et de la Recherche Medicale (INSERM) | Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders |
| AU2018345772B2 (en) * | 2017-10-02 | 2025-04-17 | Research Institute At Nationwide Children's Hospital | MiRNA detargeting system for tissue specific interference |
| AU2018351528B2 (en) * | 2017-10-20 | 2025-02-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| IL299762A (en) | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
-
2019
- 2019-06-03 GB GBGB1907882.3A patent/GB201907882D0/en not_active Ceased
-
2020
- 2020-06-03 SG SG11202112889VA patent/SG11202112889VA/en unknown
- 2020-06-03 BR BR112021024285A patent/BR112021024285A2/pt unknown
- 2020-06-03 WO PCT/EP2020/065312 patent/WO2020245169A1/en not_active Ceased
- 2020-06-03 JP JP2021571874A patent/JP7692369B2/ja active Active
- 2020-06-03 MX MX2021014789A patent/MX2021014789A/es unknown
- 2020-06-03 CA CA3142211A patent/CA3142211A1/en active Pending
- 2020-06-03 CN CN202080040867.3A patent/CN114026242A/zh active Pending
- 2020-06-03 KR KR1020217042344A patent/KR20220016485A/ko not_active Ceased
- 2020-06-03 US US17/616,006 patent/US12582725B2/en active Active
- 2020-06-03 EP EP20732138.1A patent/EP3976800A1/en active Pending
- 2020-06-03 AU AU2020288605A patent/AU2020288605A1/en active Pending
- 2020-06-03 EA EA202290001A patent/EA202290001A1/ru unknown
-
2021
- 2021-11-28 IL IL288462A patent/IL288462A/en unknown
- 2021-12-01 CL CL2021003201A patent/CL2021003201A1/es unknown
- 2021-12-02 CO CONC2021/0016487A patent/CO2021016487A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220016485A (ko) | 2022-02-09 |
| BR112021024285A2 (pt) | 2022-01-11 |
| CN114026242A (zh) | 2022-02-08 |
| JP7692369B2 (ja) | 2025-06-13 |
| SG11202112889VA (en) | 2021-12-30 |
| IL288462A (en) | 2022-01-01 |
| CO2021016487A2 (es) | 2022-01-17 |
| CA3142211A1 (en) | 2020-12-10 |
| EP3976800A1 (en) | 2022-04-06 |
| MX2021014789A (es) | 2022-02-11 |
| WO2020245169A1 (en) | 2020-12-10 |
| US20220323611A1 (en) | 2022-10-13 |
| AU2020288605A1 (en) | 2022-02-03 |
| EA202290001A1 (ru) | 2022-03-28 |
| US12582725B2 (en) | 2026-03-24 |
| JP2022535412A (ja) | 2022-08-08 |
| GB201907882D0 (en) | 2019-07-17 |
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