DK3024497T3 - Fremgangsmåder og præparater til behandling af hjernesygdomme - Google Patents
Fremgangsmåder og præparater til behandling af hjernesygdomme Download PDFInfo
- Publication number
- DK3024497T3 DK3024497T3 DK14829704.7T DK14829704T DK3024497T3 DK 3024497 T3 DK3024497 T3 DK 3024497T3 DK 14829704 T DK14829704 T DK 14829704T DK 3024497 T3 DK3024497 T3 DK 3024497T3
- Authority
- DK
- Denmark
- Prior art keywords
- preparations
- treatment
- methods
- brain diseases
- brain
- Prior art date
Links
- 208000014644 Brain disease Diseases 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 238000002360 preparation method Methods 0.000 title 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
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
- A61K31/343—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
-
- 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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
- 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)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Obesity (AREA)
- Rheumatology (AREA)
- Hematology (AREA)
- Transplantation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361859157P | 2013-07-26 | 2013-07-26 | |
| PCT/US2014/047338 WO2015013148A2 (en) | 2013-07-26 | 2014-07-20 | Methods and compositions for treating brain diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3024497T3 true DK3024497T3 (da) | 2021-04-12 |
Family
ID=52393945
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14829704.7T DK3024497T3 (da) | 2013-07-26 | 2014-07-20 | Fremgangsmåder og præparater til behandling af hjernesygdomme |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US10391184B2 (da) |
| EP (1) | EP3024497B1 (da) |
| JP (2) | JP6752142B2 (da) |
| KR (2) | KR20220106852A (da) |
| CN (1) | CN105764532B (da) |
| AU (1) | AU2014293460B2 (da) |
| BR (1) | BR112016001592A2 (da) |
| CA (1) | CA2918902C (da) |
| DK (1) | DK3024497T3 (da) |
| ES (1) | ES2859605T3 (da) |
| IL (1) | IL243758B (da) |
| MX (1) | MX373332B (da) |
| PT (1) | PT3024497T (da) |
| RU (2) | RU2664471C2 (da) |
| WO (1) | WO2015013148A2 (da) |
| ZA (1) | ZA201601024B (da) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| RU2020108189A (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| TWI752907B (zh) | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| HRP20220050T1 (hr) * | 2015-05-15 | 2022-10-14 | Regents Of The University Of Minnesota | Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav |
| CN108601771B (zh) * | 2015-10-23 | 2023-02-21 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics, Inc. | composições e métodos de tratamento da doença de huntington |
| KR102523237B1 (ko) * | 2016-08-03 | 2023-04-18 | 유니버시티 오브 사우스 플로리다 | 신경 장애의 치료를 위한 리일린 조성물 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP7610923B2 (ja) * | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Cns障害を治療するための方法及びベクター |
| CA3041548A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| IL316926A (en) | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| EP3635009B1 (en) | 2017-06-07 | 2026-02-25 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| IL274430B2 (en) | 2017-11-08 | 2025-01-01 | Novartis Gene Therapies Inc | Means and method for preparing viral vectors and their uses |
| IL276464B2 (en) | 2018-02-07 | 2026-01-01 | Regeneron Pharma | Methods and compositions for administering therapeutic protein |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| MA52626A (fr) | 2018-05-17 | 2021-03-24 | Regeneron Pharma | Anticorps anti-cd63, conjugués et leurs utilisations |
| WO2019236949A1 (en) | 2018-06-08 | 2019-12-12 | Avexis Inc. | Cell-based assay for measuring drug product potency |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US20210386870A1 (en) * | 2018-11-26 | 2021-12-16 | Universitat Autònoma De Barcelona | Fibroblast growth factor 21 (FGF21) gene therapy |
| CA3127889A1 (en) * | 2019-02-01 | 2020-08-06 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
| BR112021017054A2 (pt) * | 2019-02-28 | 2021-11-09 | Benitec Ip Holdings Inc | Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) |
| JP2022526425A (ja) * | 2019-04-12 | 2022-05-24 | エンコーデッド セラピューティクス, インコーポレイテッド | 治療剤の投与のための組成物および方法 |
| KR102272858B1 (ko) | 2019-05-22 | 2021-07-05 | 경희대학교 산학협력단 | 뇌 이상 단백질 치료를 위한 방사선 시스템 및 이의 사용 방법 |
| RU2716013C2 (ru) * | 2019-05-27 | 2020-03-05 | Федеральное государственное бюджетное образовательное учреждение высшего образования "Казанский Государственный медицинский университет" Министерства здравоохранения Российской Федерации | Способ изготовления средства для клеточно-опосредованной генной терапии и средство для клеточно-опосредованной генной терапии |
| US12509705B2 (en) | 2019-10-31 | 2025-12-30 | Universität Bern | AAV vector variants for ocular gene delivery |
| CN115244181B (zh) * | 2020-03-11 | 2025-08-08 | 上海信致医药科技有限公司 | 阿司匹林化合物在增加核酸表达中的新型用途 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| WO2025084951A1 (en) * | 2023-10-16 | 2025-04-24 | Joint Stock Company "Axelgen" | Recombinant genetic construct, adeno-associated virus for the treatment of metachromatic leukodystrophy |
| WO2025221877A1 (en) * | 2024-04-17 | 2025-10-23 | Five Prime Sciences Incorporated | Tripeptidyl peptidase 1-related compositions and methods for the treatment and diagnosis of amyotrophic lateral sclerosis |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH09505561A (ja) * | 1993-10-01 | 1997-06-03 | アメリカ合衆国 | 神経系の遺伝子治療 |
| EP1121429A2 (en) * | 1998-10-16 | 2001-08-08 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e |
| US7037493B2 (en) | 2000-05-01 | 2006-05-02 | Cornell Research Foundation, Inc. | Method of inducing neuronal production in the brain and spinal cord |
| MX2007006524A (es) | 2004-12-01 | 2007-06-22 | Genzyme Corp | Metodos para el suministro dirigido de material genetico al higado. |
| BRPI0711965A2 (pt) * | 2006-06-07 | 2012-01-24 | Genzyme Corporation | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
| DK2826860T3 (da) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS-targeting AAV-vektorer og fremgangsmåder til anvendelse deraf |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CA2832151C (en) * | 2011-03-31 | 2021-06-15 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP2836501B1 (en) * | 2012-04-10 | 2018-03-07 | City of Hope | Rna aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells |
-
2014
- 2014-07-20 BR BR112016001592A patent/BR112016001592A2/pt not_active Application Discontinuation
- 2014-07-20 EP EP14829704.7A patent/EP3024497B1/en active Active
- 2014-07-20 US US14/907,776 patent/US10391184B2/en active Active
- 2014-07-20 AU AU2014293460A patent/AU2014293460B2/en active Active
- 2014-07-20 CA CA2918902A patent/CA2918902C/en active Active
- 2014-07-20 DK DK14829704.7T patent/DK3024497T3/da active
- 2014-07-20 PT PT148297047T patent/PT3024497T/pt unknown
- 2014-07-20 RU RU2016106652A patent/RU2664471C2/ru active
- 2014-07-20 WO PCT/US2014/047338 patent/WO2015013148A2/en not_active Ceased
- 2014-07-20 CN CN201480052788.9A patent/CN105764532B/zh active Active
- 2014-07-20 JP JP2016529801A patent/JP6752142B2/ja active Active
- 2014-07-20 KR KR1020227024561A patent/KR20220106852A/ko not_active Ceased
- 2014-07-20 RU RU2018128780A patent/RU2018128780A/ru unknown
- 2014-07-20 ES ES14829704T patent/ES2859605T3/es active Active
- 2014-07-20 KR KR1020167004644A patent/KR102423069B1/ko active Active
- 2014-07-20 MX MX2016001026A patent/MX373332B/es active IP Right Grant
-
2016
- 2016-01-24 IL IL24375816A patent/IL243758B/en active IP Right Grant
- 2016-02-15 ZA ZA2016/01024A patent/ZA201601024B/en unknown
-
2019
- 2019-05-07 JP JP2019087662A patent/JP6872579B2/ja active Active
- 2019-06-28 US US16/457,332 patent/US20200009267A1/en not_active Abandoned
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