HK1225305A1 - 治療腦疾病的方法和組合物 - Google Patents
治療腦疾病的方法和組合物Info
- Publication number
- HK1225305A1 HK1225305A1 HK16113695A HK16113695A HK1225305A1 HK 1225305 A1 HK1225305 A1 HK 1225305A1 HK 16113695 A HK16113695 A HK 16113695A HK 16113695 A HK16113695 A HK 16113695A HK 1225305 A1 HK1225305 A1 HK 1225305A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- compositions
- methods
- brain diseases
- treating brain
- treating
- Prior art date
Links
- 208000014644 Brain disease Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
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
-
- 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
- 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)
- 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 (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361859157P | 2013-07-26 | 2013-07-26 |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1225305A1 true HK1225305A1 (zh) | 2017-09-08 |
Family
ID=52393945
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK16113695A HK1225305A1 (zh) | 2013-07-26 | 2016-12-01 | 治療腦疾病的方法和組合物 |
Country Status (17)
Country | Link |
---|---|
US (2) | US10391184B2 (zh) |
EP (1) | EP3024497B1 (zh) |
JP (2) | JP6752142B2 (zh) |
KR (2) | KR102423069B1 (zh) |
CN (1) | CN105764532B (zh) |
AU (1) | AU2014293460B2 (zh) |
BR (1) | BR112016001592A2 (zh) |
CA (1) | CA2918902C (zh) |
DK (1) | DK3024497T3 (zh) |
ES (1) | ES2859605T3 (zh) |
HK (1) | HK1225305A1 (zh) |
IL (1) | IL243758B (zh) |
MX (1) | MX2016001026A (zh) |
PT (1) | PT3024497T (zh) |
RU (2) | RU2018128780A (zh) |
WO (1) | WO2015013148A2 (zh) |
ZA (1) | ZA201601024B (zh) |
Families Citing this family (35)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SG11201509419QA (en) * | 2013-05-15 | 2015-12-30 | Univ 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 |
AU2015343037B2 (en) | 2014-11-05 | 2019-01-17 | Voyager Therapeutics, Inc. | AADC polynucleotides for the treatment of parkinson's disease |
KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
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 |
EP3364970B1 (en) * | 2015-10-23 | 2024-02-07 | University of Iowa Research Foundation | Gene therapy for use in treating lysosomal storage disease |
BR112018071156A2 (pt) | 2016-04-15 | 2019-03-12 | The Trustees Of The University Of Pennsylvania | terapia de gene para tratar mucopolissacaridose tipo ii |
SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
WO2018027037A2 (en) * | 2016-08-03 | 2018-02-08 | 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 |
WO2018045347A1 (en) * | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
US20190269797A1 (en) * | 2016-11-04 | 2019-09-05 | 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 |
AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
IL270422B1 (en) * | 2017-05-11 | 2024-12-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
IL271193B2 (en) | 2017-06-07 | 2025-01-01 | Regeneron Pharma | Preparations and methods for internalizing enzymes |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array 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 |
US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
EP3697905A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
CA3082136A1 (en) | 2017-11-08 | 2019-05-16 | Avexis, Inc. | Means and method for preparing viral vectors and uses of same |
JP2022518931A (ja) * | 2019-02-01 | 2022-03-17 | スパーク セラピューティクス インコーポレイテッド | 遅発型小児性神経セロイドリポフスチン沈着症2型に対するaavベクター治療法 |
CA3130221A1 (en) * | 2019-02-28 | 2020-09-03 | Benitec IP Holdings Inc. | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) |
KR20220007601A (ko) * | 2019-04-12 | 2022-01-18 | 엔코디드 테라퓨틱스, 인크. | 치료제 투여를 위한 조성물 및 방법 |
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 |
JP2023517340A (ja) * | 2020-03-11 | 2023-04-25 | シャンハイ・ビリーフ-デリバリー・バイオメッド・カンパニー・リミテッド | 核酸発現を増加させることにおけるアスピリン化合物の新規な使用 |
US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
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 |
WO2000023587A2 (en) * | 1998-10-16 | 2000-04-27 | 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 |
EP1827502A4 (en) | 2004-12-01 | 2008-01-16 | Genzyme Corp | PROCESS FOR THE TARGETED DELIVERY OF GENETIC MATERIAL TO THE LIVER |
BRPI0711965A2 (pt) | 2006-06-07 | 2012-01-24 | Genzyme Corp | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
ES2605305T3 (es) * | 2010-04-23 | 2017-03-13 | University Of Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
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 |
US9464293B2 (en) * | 2012-04-10 | 2016-10-11 | City Of Hope | RNA aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells |
-
2014
- 2014-07-20 US US14/907,776 patent/US10391184B2/en active Active
- 2014-07-20 CA CA2918902A patent/CA2918902C/en active Active
- 2014-07-20 CN CN201480052788.9A patent/CN105764532B/zh active Active
- 2014-07-20 PT PT148297047T patent/PT3024497T/pt unknown
- 2014-07-20 RU RU2018128780A patent/RU2018128780A/ru unknown
- 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 WO PCT/US2014/047338 patent/WO2015013148A2/en active Application Filing
- 2014-07-20 AU AU2014293460A patent/AU2014293460B2/en active Active
- 2014-07-20 EP EP14829704.7A patent/EP3024497B1/en active Active
- 2014-07-20 DK DK14829704.7T patent/DK3024497T3/da active
- 2014-07-20 RU RU2016106652A patent/RU2664471C2/ru active
- 2014-07-20 KR KR1020227024561A patent/KR20220106852A/ko not_active Application Discontinuation
- 2014-07-20 BR BR112016001592A patent/BR112016001592A2/pt not_active Application Discontinuation
- 2014-07-20 ES ES14829704T patent/ES2859605T3/es active Active
- 2014-07-20 JP JP2016529801A patent/JP6752142B2/ja active Active
-
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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