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
-
- 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)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Rheumatology (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 | |
| PCT/US2015/027598 WO2015164789A1 (en) | 2014-04-24 | 2015-04-24 | Aav-based gene therapy for multiple sclerosis |
| EP15782332.9A EP3133923B1 (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 |
| US12290574B2 (en) * | 2018-08-24 | 2025-05-06 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| CN114555063A (zh) * | 2019-06-04 | 2022-05-27 | 托马斯杰斐逊大学 | 用于治疗多发性硬化症的少突胶质细胞来源的细胞外囊泡 |
| TW202204625A (zh) * | 2020-04-14 | 2022-02-01 | 美商佛羅里達大學研究基金會公司 | 多發性硬化之基因免疫治療與免疫抑制劑之增強功效 |
| WO2021211614A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
| KR20230031329A (ko) * | 2020-06-30 | 2023-03-07 | 칠드런스 내셔널 메디컬 센터 | 골격 근섬유 복구를 개선하기 위한 재조합 인간 산성 스핑고미엘리나제의 용도 |
| 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 |
| WO2010055413A1 (en) * | 2008-11-12 | 2010-05-20 | Fondazione Centro San Raffaele Del Monte Tabor | Gene vector for inducing transgene-specific immune tolerance |
| SG10201600912SA (en) * | 2011-03-04 | 2016-03-30 | Intrexon Corp | Vectors conditionally expressing protein |
| US9592259B2 (en) * | 2011-09-26 | 2017-03-14 | University Of Zurich | APC-mediated tolerance induction for therapy of multiple sclerosis |
| CA2929273C (en) * | 2013-11-22 | 2022-05-31 | 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 HU HUE15782332A patent/HUE050681T2/hu unknown
- 2015-04-24 EP EP15782332.9A patent/EP3133923B1/en active Active
- 2015-04-24 EP EP20167323.3A patent/EP3744177A1/en active Pending
- 2015-04-24 RS RS20201008A patent/RS60816B1/sr unknown
- 2015-04-24 AU AU2015249328A patent/AU2015249328B2/en not_active Ceased
- 2015-04-24 JP JP2016564019A patent/JP6646282B2/ja active Active
- 2015-04-24 HR HRP20201342TT patent/HRP20201342T1/hr unknown
- 2015-04-24 WO PCT/US2015/027598 patent/WO2015164789A1/en not_active Ceased
- 2015-04-24 US US15/306,163 patent/US20170043036A1/en not_active Abandoned
- 2015-04-24 SM SM20200453T patent/SMT202000453T1/it unknown
- 2015-04-24 DK DK15782332.9T patent/DK3133923T3/da active
- 2015-04-24 CA CA2945984A patent/CA2945984C/en active Active
- 2015-04-24 PT PT157823329T patent/PT3133923T/pt unknown
- 2015-04-24 SI SI201531336T patent/SI3133923T1/sl unknown
- 2015-04-24 ES ES15782332T patent/ES2814280T3/es active Active
- 2015-04-24 LT LTEP15782332.9T patent/LT3133923T/lt unknown
- 2015-04-24 PL PL15782332.9T patent/PL3133923T3/pl unknown
-
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 |
|---|---|
| EP3744177A1 (en) | 2020-12-02 |
| EP3133923B1 (en) | 2020-06-03 |
| EP3133923A4 (en) | 2017-12-27 |
| RS60816B1 (sr) | 2020-10-30 |
| PL3133923T3 (pl) | 2021-02-22 |
| DK3133923T3 (da) | 2020-08-10 |
| CA2945984C (en) | 2023-09-26 |
| HUE050681T2 (hu) | 2020-12-28 |
| EP3133923A1 (en) | 2017-03-01 |
| JP7430898B2 (ja) | 2024-02-14 |
| JP2020062038A (ja) | 2020-04-23 |
| JP2017513500A (ja) | 2017-06-01 |
| AU2019202958A1 (en) | 2019-05-16 |
| LT3133923T (lt) | 2020-08-25 |
| CY1123312T1 (el) | 2021-12-31 |
| US20200138978A1 (en) | 2020-05-07 |
| SI3133923T1 (sl) | 2020-11-30 |
| SMT202000453T1 (it) | 2020-09-10 |
| US20170043036A1 (en) | 2017-02-16 |
| CA2945984A1 (en) | 2015-10-29 |
| JP6646282B2 (ja) | 2020-02-14 |
| WO2015164789A1 (en) | 2015-10-29 |
| AU2015249328B2 (en) | 2019-01-17 |
| US20230381342A1 (en) | 2023-11-30 |
| AU2015249328A1 (en) | 2016-10-27 |
| JP2022081549A (ja) | 2022-05-31 |
| AU2019202958B2 (en) | 2021-08-05 |
| PT3133923T (pt) | 2020-09-04 |
| ES2814280T3 (es) | 2021-03-26 |
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