MX2016001026A - Metodos y composiciones para tratar enfermedades del cerebro. - Google Patents
Metodos y composiciones para tratar enfermedades del cerebro.Info
- Publication number
- MX2016001026A MX2016001026A MX2016001026A MX2016001026A MX2016001026A MX 2016001026 A MX2016001026 A MX 2016001026A MX 2016001026 A MX2016001026 A MX 2016001026A MX 2016001026 A MX2016001026 A MX 2016001026A MX 2016001026 A MX2016001026 A MX 2016001026A
- Authority
- MX
- Mexico
- Prior art keywords
- therapeutic agent
- compositions
- methods
- csf
- mammal
- Prior art date
Links
- 208000014644 Brain disease Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 abstract 3
- 239000003814 drug Substances 0.000 abstract 3
- 229940124597 therapeutic agent Drugs 0.000 abstract 3
- 241000124008 Mammalia Species 0.000 abstract 2
- 210000004556 brain Anatomy 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 210000003703 cisterna magna Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 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
- 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
- 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
- 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)
- Veterinary Medicine (AREA)
- Public Health (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)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Transplantation (AREA)
Abstract
La presente descripción proporciona métodos para tratar una enfermedad o suministrar un agente terapéutico a un mamífero, que comprende administrar a la cisterna magna y/o ventrículo del mamífero una partícula rAAV que contiene un vector que comprende un ácido nucleico que codifica una proteína terapéutica insertada entre un par de repeticiones terminales invertidas AAV de tal forma que las células que tienen acceso al fluido cerebroespinal (CSF) expresan el agente terapéutico y en ciertas modalidades secreta el agente terapéutico al CSF para su distribución en el cerebro.
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 |
|---|---|
| MX2016001026A true MX2016001026A (es) | 2016-08-03 |
Family
ID=52393945
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016001026A MX2016001026A (es) | 2013-07-26 | 2014-07-20 | Metodos y composiciones para tratar enfermedades del cerebro. |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US10391184B2 (es) |
| EP (1) | EP3024497B1 (es) |
| JP (2) | JP6752142B2 (es) |
| KR (2) | KR102423069B1 (es) |
| CN (1) | CN105764532B (es) |
| AU (1) | AU2014293460B2 (es) |
| BR (1) | BR112016001592A2 (es) |
| CA (1) | CA2918902C (es) |
| DK (1) | DK3024497T3 (es) |
| ES (1) | ES2859605T3 (es) |
| HK (1) | HK1225305A1 (es) |
| IL (1) | IL243758B (es) |
| MX (1) | MX2016001026A (es) |
| PT (1) | PT3024497T (es) |
| RU (2) | RU2018128780A (es) |
| WO (1) | WO2015013148A2 (es) |
| ZA (1) | ZA201601024B (es) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| 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 |
| RS63927B1 (sr) * | 2015-05-15 | 2023-02-28 | Regenxbio Inc | Povezani adenovirus za terapijsku isporuku u centralni nervni sistem |
| US20180236105A1 (en) * | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| AU2017306558A1 (en) * | 2016-08-03 | 2019-02-21 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| EP3506930A4 (en) * | 2016-09-02 | 2020-09-23 | Spark Therapeutics, Inc. | METHODS AND VECTORS FOR TREATMENT OF CNS DISEASES |
| WO2018085688A1 (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 |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| WO2018209205A1 (en) * | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal 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 |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| JP7502991B2 (ja) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症（ａｌｓ）の治療 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US20220184188A1 (en) * | 2019-02-01 | 2022-06-16 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
| EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
| 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 |
| CN115244181A (zh) * | 2020-03-11 | 2022-10-25 | 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 |
Family Cites Families (8)
| 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 |
| 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 |
| EP1827502A4 (en) | 2004-12-01 | 2008-01-16 | Genzyme Corp | PROCESS FOR THE TARGETED DELIVERY OF GENETIC MATERIAL TO THE LIVER |
| CA2654292C (en) * | 2006-06-07 | 2022-01-11 | Genzyme Corporation | Gene therapy for motor neuron disorders |
| EP2826860B1 (en) * | 2010-04-23 | 2018-08-22 | 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 |
-
2014
- 2014-07-20 RU RU2018128780A patent/RU2018128780A/ru unknown
- 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 DK DK14829704.7T patent/DK3024497T3/da 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 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 active Application Filing
- 2014-07-20 CA CA2918902A patent/CA2918902C/en active Active
- 2014-07-20 JP JP2016529801A patent/JP6752142B2/ja active Active
- 2014-07-20 KR KR1020167004644A patent/KR102423069B1/ko active IP Right Grant
- 2014-07-20 MX MX2016001026A patent/MX2016001026A/es active IP Right Grant
- 2014-07-20 BR BR112016001592A patent/BR112016001592A2/pt not_active Application Discontinuation
- 2014-07-20 CN CN201480052788.9A patent/CN105764532B/zh active Active
- 2014-07-20 KR KR1020227024561A patent/KR20220106852A/ko 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
- 2016-12-01 HK HK16113695A patent/HK1225305A1/zh unknown
-
2019
- 2019-05-07 JP JP2019087662A patent/JP6872579B2/ja active Active
- 2019-06-28 US US16/457,332 patent/US20200009267A1/en active Pending
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