DK2879719T3 - Intratekal administration af rekombinant adenoassocieret virus 9 - Google Patents
Intratekal administration af rekombinant adenoassocieret virus 9 Download PDFInfo
- Publication number
- DK2879719T3 DK2879719T3 DK13747755.0T DK13747755T DK2879719T3 DK 2879719 T3 DK2879719 T3 DK 2879719T3 DK 13747755 T DK13747755 T DK 13747755T DK 2879719 T3 DK2879719 T3 DK 2879719T3
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- DK
- Denmark
- Prior art keywords
- composition
- polynucleotide
- raav9
- contrast agent
- use according
- Prior art date
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Classifications
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- 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
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- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/04—X-ray contrast preparations
- A61K49/0433—X-ray contrast preparations containing an organic halogenated X-ray contrast-enhancing agent
- A61K49/0438—Organic X-ray contrast-enhancing agent comprising an iodinated group or an iodine atom, e.g. iopamidol
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
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- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
Claims (14)
1. Sammensætning, der omfatter en rekombinant AAV9 (rAAV9) og et ikke-ionisk, lav-osmolært kontrastmiddel til anvendelse i en fremgangsmåde til administration af et polynukleotid til centralnervesystemet hos en patient, der har brug for det, hvilken fremgangsmåde omfatter intratekal administration af sammensætningen til patienten, hvor rAAV9'en omfatter et selvkomplementært genom, der indbefatter polynukleotidet, og hvor det ikke-ioniske, lavosmolære kontrastmiddel er iobitridol, iohexol, iomeprol, iopamidol, iopentol, iopromid, ioversol eller ioxilan.
2. Sammensætning til anvendelse ifølge krav 1, hvor polynukleotidet administreres til hjernen.
3. Sammensætning til anvendelse ifølge krav 1, hvor polynukleotidet administreres til rygmarven.
4. Sammensætning til anvendelse ifølge krav 1, hvor polynukleotidet administreres til en gliacelle.
5. Sammensætning til anvendelse ifølge krav 4, hvor gliacellen er en astrocyt.
6. Sammensætning til anvendelse ifølge krav 1, hvor polynukleotidet administreres til en nedre motorisk neuron.
7. Sammensætning til anvendelse ifølge krav 1, hvor det ikke-ioniske, lavosmolære kontrastmiddel er iohexol.
8. Sammensætning til anvendelse ifølge krav 1, hvor polynukleotidet er et overlevelses-motorisk neuron (SMN)-polynukleotid.
9. Sammensætning, der omfatter en rAAV9 og et ikke-ionisk, lavosmolært kontrastmiddel til anvendelse i en fremgangsmåde til behandling af en neurologisk sygdom hos en patient, der har brug for det, hvilken fremgangsmåde omfatter intratekal administration af sammensætningen til patienten ved hjælp af en fremgangsmåde som defineret i et hvilket som helst af ovennævnte krav, hvor rAAV9 omfatter et selvkomplementært genom, der indbefatter et terapeutisk polynukleotid, og hvor det ikke-ioniske, lavosmolære kontrastmiddel er iobitridol, iohexol, iomeprol, iopamidol, iopentol, iopromid, ioversol eller ioxilan.
10. Sammensætning til anvendelse ifølge krav 9, hvor den neurologiske sygdom er Retts syndrom.
11. Sammensætning til anvendelse ifølge krav 9, hvor den neurologiske sygdom er en neurodegenerativ sygdom.
12. Sammensætning til anvendelse ifølge krav 11, hvor det terapeutiske polynukleotid er et overlevelses-motorisk neuron (SMN)-polynukleotid.
13. Sammensætning til anvendelse ifølge krav 11, hvor den neurodegenerative sygdom er spinal muskelatrofi eller amyotrofisk lateral sklerose.
14. Sammensætning til anvendelse ifølge krav 9, hvor patienten anbringes i Trendelenburg-stillingen efter intratekal administration af rAAV9.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261678458P | 2012-08-01 | 2012-08-01 | |
| PCT/US2013/053065 WO2014022582A1 (en) | 2012-08-01 | 2013-07-31 | Intrathecal delivery of recombinant adeno-associated virus 9 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2879719T3 true DK2879719T3 (da) | 2018-09-03 |
Family
ID=48951610
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK18181263.7T DK3415167T3 (da) | 2012-08-01 | 2013-07-31 | Intratekal administration af rekombinant adenoassocieret virus 9 |
| DK13747755.0T DK2879719T3 (da) | 2012-08-01 | 2013-07-31 | Intratekal administration af rekombinant adenoassocieret virus 9 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK18181263.7T DK3415167T3 (da) | 2012-08-01 | 2013-07-31 | Intratekal administration af rekombinant adenoassocieret virus 9 |
Country Status (11)
| Country | Link |
|---|---|
| US (12) | US20150252384A1 (da) |
| EP (3) | EP2879719B1 (da) |
| JP (5) | JP6314138B2 (da) |
| AU (5) | AU2013296425B2 (da) |
| BR (1) | BR112015002168A2 (da) |
| CA (2) | CA2880653C (da) |
| DK (2) | DK3415167T3 (da) |
| ES (1) | ES2684222T3 (da) |
| FI (1) | FI3415167T3 (da) |
| PT (1) | PT2879719T (da) |
| WO (1) | WO2014022582A1 (da) |
Families Citing this family (36)
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| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| HRP20212024T1 (hr) | 2009-05-02 | 2022-04-01 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| BR112015002168A2 (pt) | 2012-08-01 | 2017-11-07 | Nationwide Childrens Hospital | liberação intratecal de vírus 9 adeno-associado recombinante |
| BR112015028605A8 (pt) * | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
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| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| ES2878451T3 (es) | 2014-11-14 | 2021-11-18 | Voyager Therapeutics Inc | Polinucleótidos moduladores |
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| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| TWI722310B (zh) | 2017-08-03 | 2021-03-21 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| KR20200104852A (ko) | 2017-09-22 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
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| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
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