HK1257801A1 - 用於基因治療的腺伴隨病毒載體的鞘內給藥 - Google Patents

用於基因治療的腺伴隨病毒載體的鞘內給藥

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Publication number
HK1257801A1
HK1257801A1 HK18114021.4A HK18114021A HK1257801A1 HK 1257801 A1 HK1257801 A1 HK 1257801A1 HK 18114021 A HK18114021 A HK 18114021A HK 1257801 A1 HK1257801 A1 HK 1257801A1
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HK
Hong Kong
Prior art keywords
adeno
gene therapy
viral vectors
intrathecal administration
intrathecal
Prior art date
Application number
HK18114021.4A
Other languages
English (en)
Inventor
J‧M‧威爾遜
C‧辛德勒
W‧T‧羅斯維爾
Original Assignee
賓夕法尼亞州大學信託人
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Publication date
Application filed by 賓夕法尼亞州大學信託人 filed Critical 賓夕法尼亞州大學信託人
Publication of HK1257801A1 publication Critical patent/HK1257801A1/zh

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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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/10—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from RNA viruses
    • C07K16/1036—Retroviridae, e.g. leukemia viruses
    • C07K16/1045—Lentiviridae, e.g. HIV, FIV, SIV
    • C07K16/1063—Lentiviridae, e.g. HIV, FIV, SIV env, e.g. gp41, gp110/120, gp160, V3, PND, CD4 binding site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
HK18114021.4A 2015-10-28 2018-11-02 用於基因治療的腺伴隨病毒載體的鞘內給藥 HK1257801A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562247498P 2015-10-28 2015-10-28
PCT/US2016/058968 WO2017075119A1 (en) 2015-10-28 2016-10-27 Intrathecal administration of adeno-associated-viral vectors for gene therapy

Publications (1)

Publication Number Publication Date
HK1257801A1 true HK1257801A1 (zh) 2019-11-01

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HK18114021.4A HK1257801A1 (zh) 2015-10-28 2018-11-02 用於基因治療的腺伴隨病毒載體的鞘內給藥

Country Status (11)

Country Link
US (2) US11135313B2 (zh)
EP (2) EP4316512A3 (zh)
JP (2) JP2019501115A (zh)
KR (1) KR20180100304A (zh)
CN (2) CN116196410A (zh)
AU (2) AU2016344065A1 (zh)
BR (1) BR112018008407A2 (zh)
CA (1) CA3003435A1 (zh)
HK (1) HK1257801A1 (zh)
MX (1) MX2018005353A (zh)
WO (1) WO2017075119A1 (zh)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
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
CA3051839A1 (en) 2017-02-17 2018-08-23 Bristol-Myers Squibb Company Antibodies to alpha-synuclein and uses thereof
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
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
US11819539B2 (en) * 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
KR20200067195A (ko) * 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
EP3790979A4 (en) * 2018-05-08 2022-04-27 Neuracle Science Co., Ltd RELEASE OF ADENO-ASSOCIATED VIRUS (AAV) FROM ANTI-FAM19A5 ANTIBODIES
US20220118226A1 (en) 2019-01-22 2022-04-21 The Trustees Of The University Of Pennsylvania Microcatheter for therapeutic and/or diagnostic interventions in the subarachnoid space
US11541086B2 (en) * 2019-03-14 2023-01-03 Northwestern Univeristy Compositions and methods for treating Alzheimer's disease
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
AU2021359874A1 (en) 2020-10-18 2023-05-25 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4236975A1 (en) 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Aav capsids and compositions containing same
KR20220109347A (ko) * 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
AR125467A1 (es) 2021-04-27 2023-07-19 Univ Pennsylvania Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
WO2023287932A1 (en) * 2021-07-15 2023-01-19 The Regents Of The University Of Michigan Compositions and methods for treating alpha-synucleinopathies
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
AU781958C (en) 1999-08-09 2006-03-30 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
CN101670105B (zh) 2000-02-24 2014-08-06 华盛顿大学 螯合淀粉样蛋白β肽的人源化抗体
DE60139471D1 (de) 2000-06-01 2009-09-17 Univ North Carolina Verfahren und zusammensetzungen zur kontrollierter abgabe von rekombinant parvovirus vektoren
US6946292B2 (en) 2000-10-06 2005-09-20 Kyowa Hakko Kogyo Co., Ltd. Cells producing antibody compositions with increased antibody dependent cytotoxic activity
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
JP4769417B2 (ja) 2001-12-17 2011-09-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
CN1297567C (zh) * 2003-06-13 2007-01-31 马菁 具有抗肿瘤功能的双功能融合蛋白及其制备方法和应用
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
EP1697415A1 (en) 2003-11-12 2006-09-06 Biogen Idec MA Inc. NEONATAL Fc RECEPTOR (FcRn)-BINDING POLYPEPTIDE VARIANTS, DIMERIC Fc BINDING PROTEINS AND METHODS RELATED THERETO
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
GB0513766D0 (en) * 2005-07-05 2005-08-10 Glaxo Group Ltd Immunoglobulins
US20070027438A1 (en) 2005-07-26 2007-02-01 Frieder Loesel System and method for compensating a corneal dissection
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
IL199534A (en) * 2007-01-05 2013-01-31 Univ Zuerich An isolated human antibody capable of detecting a neoepitope in a disease-related protein, a polynucleotide encoding an antibody, a vector containing the polynucleotide, a host cell containing the polynucleotide or vector, a preparation containing the antibody and related methods and uses.
JP5291093B2 (ja) * 2007-05-16 2013-09-18 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド シヌクレイノパチーの治療
AU2009269099B2 (en) 2008-07-09 2016-03-10 Biogen Ma Inc. Compositions comprising antibodies to LINGO or fragments thereof
CA2793633A1 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
TW201817745A (zh) 2011-09-30 2018-05-16 日商中外製藥股份有限公司 具有促進抗原清除之ＦｃＲｎ結合域的治療性抗原結合分子
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
PT2839014T (pt) 2012-04-18 2021-03-19 Childrens Hospital Philadelphia ¿composição e métodos para transferência de genes altamente eficiente com a utilização de variantes de capsídeo de aav
US10287333B2 (en) * 2012-09-27 2019-05-14 University Of British Columbia Peptide directed protein knockdown
EP2928494A4 (en) 2012-12-07 2016-11-02 Biogen Internat Neuroscience Gmbh METHOD FOR REDUCING AMYLOIDER PLAQUE OF BRAIN WITH ANTI-ASS ANTIBODIES
CN103160530B (zh) * 2013-03-19 2017-10-20 苏州工业园区唯可达生物科技有限公司 一种融合基因及其应用
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
RU2724190C2 (ru) 2014-02-08 2020-06-23 Дженентек, Инк. Способы лечения болезни альцгеймера
MX2016010237A (es) * 2014-02-08 2017-04-27 Genentech Inc Metodos de tratamiento de enfermedad de alzheimer.

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US20210379202A1 (en) 2021-12-09
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