HRP20201342T1 - Genetska terapija za multiplu sklerozu bazirana na aav - Google Patents
Genetska terapija za multiplu sklerozu bazirana na aav Download PDFInfo
- Publication number
- HRP20201342T1 HRP20201342T1 HRP20201342TT HRP20201342T HRP20201342T1 HR P20201342 T1 HRP20201342 T1 HR P20201342T1 HR P20201342T T HRP20201342T T HR P20201342TT HR P20201342 T HRP20201342 T HR P20201342T HR P20201342 T1 HRP20201342 T1 HR P20201342T1
- Authority
- HR
- Croatia
- Prior art keywords
- nucleic acid
- raav
- acid vector
- seq
- protein
- Prior art date
Links
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
- A61K48/005—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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/0005—Vertebrate antigens
- A61K39/0008—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
-
- 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
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
-
- 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
-
- 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
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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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/53—DNA (RNA) vaccination
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/57—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
- A61K2039/577—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Rheumatology (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Mycology (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Neurosurgery (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461983924P | 2014-04-24 | 2014-04-24 | |
| EP15782332.9A EP3133923B1 (en) | 2014-04-24 | 2015-04-24 | Aav-based gene therapy for multiple sclerosis |
| PCT/US2015/027598 WO2015164789A1 (en) | 2014-04-24 | 2015-04-24 | Aav-based gene therapy for multiple sclerosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20201342T1 true HRP20201342T1 (hr) | 2020-11-27 |
Family
ID=54333299
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20201342TT HRP20201342T1 (hr) | 2014-04-24 | 2015-04-24 | Genetska terapija za multiplu sklerozu bazirana na aav |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US20170043036A1 (https=) |
| EP (2) | EP3133923B1 (https=) |
| JP (3) | JP6646282B2 (https=) |
| AU (2) | AU2015249328B2 (https=) |
| CA (1) | CA2945984C (https=) |
| CY (1) | CY1123312T1 (https=) |
| DK (1) | DK3133923T3 (https=) |
| ES (1) | ES2814280T3 (https=) |
| HR (1) | HRP20201342T1 (https=) |
| HU (1) | HUE050681T2 (https=) |
| LT (1) | LT3133923T (https=) |
| PL (1) | PL3133923T3 (https=) |
| PT (1) | PT3133923T (https=) |
| RS (1) | RS60816B1 (https=) |
| SI (1) | SI3133923T1 (https=) |
| SM (1) | SMT202000453T1 (https=) |
| WO (1) | WO2015164789A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7270946B2 (ja) * | 2017-08-08 | 2023-05-11 | 国立大学法人浜松医科大学 | 自己免疫疾患の治療又は予防剤 |
| BR112021003399A2 (pt) * | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| CA3142487A1 (en) * | 2019-06-04 | 2020-12-10 | Thomas Jefferson University | Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis |
| WO2021211614A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
| WO2021211640A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis |
| JP2023532923A (ja) * | 2020-06-30 | 2023-08-01 | チルドレンズ ナショナル メディカル センター | 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用 |
| WO2024081570A1 (en) * | 2022-10-10 | 2024-04-18 | Eli Lilly And Company | Methods and systems for using causal networks to develop models for evaluating biological processes |
| WO2024258925A1 (en) | 2023-06-12 | 2024-12-19 | Children's Hospital Medical Center | Aav-cftr vectors and methods of using same |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR960705038A (ko) * | 1993-09-03 | 1996-10-09 | 스테시 엘. 채닝 | 자기 면역 질환에 관련된 프로토콜에서 미엘린 올리고덴드로시트 당 단백질 및 그의 펩타이드 부분들의 용도(users of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune desease) |
| AU5588298A (en) * | 1996-12-02 | 1998-06-29 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| KR100449454B1 (ko) * | 2000-10-02 | 2004-09-21 | 이현철 | 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터 |
| EP1193272B1 (en) * | 2000-10-02 | 2004-06-30 | Yonsei University | Single-chain insulin analogs |
| CA2361462A1 (en) * | 2001-11-07 | 2003-05-08 | Katherine A. High | Induction of tolerance to a therapeutic polypeptide |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20090304726A1 (en) * | 2006-02-15 | 2009-12-10 | Ramot At Tel Aviv University Ltd. | Viral display vehicles for treating multiple sclerosis |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2364362B1 (en) * | 2008-11-12 | 2015-10-21 | Ospedale San Raffaele S.r.l. | Gene vector for inducing transgene-specific immune tolerance |
| JP6189754B2 (ja) * | 2011-03-04 | 2017-08-30 | イントレキソン コーポレーション | タンパク質を条件的に発現するベクター |
| JP2015502135A (ja) * | 2011-09-26 | 2015-01-22 | ウニヴェルジテート チューリッヒ プロレクトラート エムエヌヴェーＵｎｉｖｅｒｓｉｔａｅｔ Ｚｕｅｒｉｃｈ Ｐｒｏｒｅｋｔｏｒａｔ Ｍｎｗ | 多発性硬化症の治療のためのａｐｃ媒介寛容誘導 |
| US20160287685A1 (en) * | 2013-11-22 | 2016-10-06 | Amarna Holding B.V. | Method for restoring immune tolerance in vivo |
| WO2021211614A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
-
2015
- 2015-04-24 EP EP15782332.9A patent/EP3133923B1/en active Active
- 2015-04-24 HU HUE15782332A patent/HUE050681T2/hu unknown
- 2015-04-24 PL PL15782332.9T patent/PL3133923T3/pl unknown
- 2015-04-24 RS RS20201008A patent/RS60816B1/sr unknown
- 2015-04-24 SM SM20200453T patent/SMT202000453T1/it unknown
- 2015-04-24 EP EP20167323.3A patent/EP3744177A1/en active Pending
- 2015-04-24 DK DK15782332.9T patent/DK3133923T3/da active
- 2015-04-24 AU AU2015249328A patent/AU2015249328B2/en not_active Ceased
- 2015-04-24 SI SI201531336T patent/SI3133923T1/sl unknown
- 2015-04-24 CA CA2945984A patent/CA2945984C/en active Active
- 2015-04-24 LT LTEP15782332.9T patent/LT3133923T/lt unknown
- 2015-04-24 WO PCT/US2015/027598 patent/WO2015164789A1/en not_active Ceased
- 2015-04-24 HR HRP20201342TT patent/HRP20201342T1/hr unknown
- 2015-04-24 JP JP2016564019A patent/JP6646282B2/ja active Active
- 2015-04-24 US US15/306,163 patent/US20170043036A1/en not_active Abandoned
- 2015-04-24 PT PT157823329T patent/PT3133923T/pt unknown
- 2015-04-24 ES ES15782332T patent/ES2814280T3/es active Active
-
2019
- 2019-04-17 AU AU2019202958A patent/AU2019202958B2/en not_active Ceased
- 2019-11-08 US US16/679,102 patent/US20200138978A1/en not_active Abandoned
- 2019-12-25 JP JP2019235107A patent/JP7430898B2/ja active Active
-
2020
- 2020-09-02 CY CY20201100820T patent/CY1123312T1/el unknown
-
2022
- 2022-03-01 JP JP2022031064A patent/JP2022081549A/ja active Pending
-
2023
- 2023-03-28 US US18/191,790 patent/US20230381342A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| PT3133923T (pt) | 2020-09-04 |
| AU2019202958B2 (en) | 2021-08-05 |
| US20170043036A1 (en) | 2017-02-16 |
| AU2015249328B2 (en) | 2019-01-17 |
| PL3133923T3 (pl) | 2021-02-22 |
| LT3133923T (lt) | 2020-08-25 |
| JP7430898B2 (ja) | 2024-02-14 |
| SMT202000453T1 (it) | 2020-09-10 |
| CA2945984A1 (en) | 2015-10-29 |
| AU2015249328A1 (en) | 2016-10-27 |
| US20200138978A1 (en) | 2020-05-07 |
| US20230381342A1 (en) | 2023-11-30 |
| JP2017513500A (ja) | 2017-06-01 |
| JP2022081549A (ja) | 2022-05-31 |
| JP6646282B2 (ja) | 2020-02-14 |
| JP2020062038A (ja) | 2020-04-23 |
| EP3133923A1 (en) | 2017-03-01 |
| AU2019202958A1 (en) | 2019-05-16 |
| ES2814280T3 (es) | 2021-03-26 |
| EP3744177A1 (en) | 2020-12-02 |
| CY1123312T1 (el) | 2021-12-31 |
| RS60816B1 (sr) | 2020-10-30 |
| DK3133923T3 (da) | 2020-08-10 |
| HUE050681T2 (hu) | 2020-12-28 |
| SI3133923T1 (sl) | 2020-11-30 |
| EP3133923B1 (en) | 2020-06-03 |
| WO2015164789A1 (en) | 2015-10-29 |
| EP3133923A4 (en) | 2017-12-27 |
| CA2945984C (en) | 2023-09-26 |
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