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)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (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)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Psychology (AREA)
- Pain & Pain Management (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Transplantation (AREA)
- Orthopedic Medicine & Surgery (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) | KR102423069B1 (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) | RU2018128780A (da) |
| WO (1) | WO2015013148A2 (da) |
| ZA (1) | ZA201601024B (da) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HK1222093A1 (zh) * | 2013-05-15 | 2017-06-23 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| RU2716422C2 (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| CN112410339A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| 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 |
| MX2017014443A (es) * | 2015-05-15 | 2018-08-01 | Univ Minnesota | Adeno-asociado para administracion terapeutica al sistema nervioso central. |
| AU2016341428B2 (en) * | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| SG11201808176TA (en) | 2016-04-15 | 2018-10-30 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| CA3252099A1 (en) | 2016-05-18 | 2025-06-05 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CN109831916B (zh) | 2016-05-18 | 2023-07-21 | 沃雅戈治疗公司 | 治疗亨廷顿氏舞蹈病的组合物和方法 |
| AU2017306558B2 (en) * | 2016-08-03 | 2024-09-19 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| MX2019002518A (es) * | 2016-09-02 | 2019-07-18 | Spark Therapeutics Inc | Metodos y vectores para el tratamiento de trastornos del snc. |
| JP2019537576A (ja) * | 2016-11-04 | 2019-12-26 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | ハンチントン病治療組成物および方法 |
| US11591614B2 (en) | 2017-05-11 | 2023-02-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ceroid lipofuscinoses |
| EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | 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 |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| CN119242711A (zh) | 2017-10-16 | 2025-01-03 | 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) |
| EP3707264A1 (en) | 2017-11-08 | 2020-09-16 | Avexis Inc. | Means and method for preparing viral vectors and uses of same |
| IL322464A (en) | 2018-02-07 | 2025-09-01 | Regeneron Pharma | Methods and compositions for administering therapeutic protein |
| BR112020023082A2 (pt) | 2018-05-15 | 2021-02-09 | Voyager Therapeutics, Inc. | composições e métodos para o tratamento de doença de parkinson |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| US12228571B2 (en) | 2018-06-08 | 2025-02-18 | Novartis Ag | Cell-based assay for measuring drug product potency |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN113227126A (zh) * | 2018-11-26 | 2021-08-06 | 巴塞罗那自治大学 | 成纤维细胞生长因子21(fgf21)基因治疗 |
| CA3127889A1 (en) * | 2019-02-01 | 2020-08-06 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
| MX2021010412A (es) * | 2019-02-28 | 2022-01-24 | Benitec Ip Holdings Inc | Composiciones y metodos para tratar distrofia muscular oculofaringea (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 |
| US20230132582A1 (en) * | 2020-03-11 | 2023-05-04 | Shanghai Belief-Delivery Biomed Co., Ltd. | Novel use of aspirin compound in increasing nucleic acid expression |
| 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 |
|---|---|---|---|---|
| CA2171109A1 (en) * | 1993-10-01 | 1995-04-13 | Zvi Ram | Gene therapy of the nervous system |
| AU6234099A (en) * | 1998-10-16 | 2000-05-08 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apoliprotein |
| US7037493B2 (en) * | 2000-05-01 | 2006-05-02 | Cornell Research Foundation, Inc. | Method of inducing neuronal production in the brain and spinal cord |
| CN101068575A (zh) * | 2004-12-01 | 2007-11-07 | 建新公司 | 向肝脏定向递送遗传物质的方法 |
| PL2489733T3 (pl) | 2006-06-07 | 2019-08-30 | Genzyme Corporation | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
| US9102949B2 (en) * | 2010-04-23 | 2015-08-11 | University Of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| US9849195B2 (en) * | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| CA2870288A1 (en) * | 2012-04-10 | 2013-10-17 | John J. Rossi | Rna aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells |
-
2014
- 2014-07-20 DK DK14829704.7T patent/DK3024497T3/da active
- 2014-07-20 PT PT148297047T patent/PT3024497T/pt unknown
- 2014-07-20 JP JP2016529801A patent/JP6752142B2/ja active Active
- 2014-07-20 KR KR1020167004644A patent/KR102423069B1/ko 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 AU AU2014293460A patent/AU2014293460B2/en active Active
- 2014-07-20 CA CA2918902A patent/CA2918902C/en active Active
- 2014-07-20 RU RU2016106652A patent/RU2664471C2/ru active
- 2014-07-20 ES ES14829704T patent/ES2859605T3/es active Active
- 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 MX MX2016001026A patent/MX373332B/es active IP Right Grant
- 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 BR BR112016001592A patent/BR112016001592A2/pt not_active Application Discontinuation
-
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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