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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
Application number
HK16113695A
Other languages
English (en)
Inventor
Beverly L Davidson
Luis Tecedor
Yong Hong Chen
Original Assignee
Univ Iowa Res Found
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Iowa Res Found filed Critical Univ Iowa Res Found
Publication of HK1225305A1 publication Critical patent/HK1225305A1/zh

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/34Heterocyclic 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
    • A61K31/343Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
HK16113695A 2013-07-26 2016-12-01 治療腦疾病的方法和組合物 HK1225305A1 (zh)

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)

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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
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Also Published As

Publication number Publication date
EP3024497A4 (en) 2017-01-04
JP2016530246A (ja) 2016-09-29
KR20220106852A (ko) 2022-07-29
US20160166709A1 (en) 2016-06-16
EP3024497B1 (en) 2021-01-13
AU2014293460B2 (en) 2019-10-24
WO2015013148A3 (en) 2015-11-05
CA2918902A1 (en) 2015-01-29
KR20160035596A (ko) 2016-03-31
WO2015013148A2 (en) 2015-01-29
RU2016106652A (ru) 2017-08-31
CN105764532B (zh) 2021-08-31
DK3024497T3 (da) 2021-04-12
RU2018128780A (ru) 2018-12-05
US10391184B2 (en) 2019-08-27
MX2016001026A (es) 2016-08-03
ZA201601024B (en) 2020-08-26
IL243758B (en) 2019-11-28
ES2859605T3 (es) 2021-10-04
RU2018128780A3 (zh) 2022-04-13
AU2014293460A1 (en) 2016-02-18
JP6872579B2 (ja) 2021-05-19
BR112016001592A2 (pt) 2017-11-28
KR102423069B1 (ko) 2022-07-21
EP3024497A2 (en) 2016-06-01
CN105764532A (zh) 2016-07-13
JP2019163281A (ja) 2019-09-26
PT3024497T (pt) 2021-08-23
RU2664471C2 (ru) 2018-08-17
CA2918902C (en) 2023-04-04
JP6752142B2 (ja) 2020-09-09
IL243758A0 (en) 2016-04-21
US20200009267A1 (en) 2020-01-09

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