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
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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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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
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    • 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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    • 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
    • C07K—PEPTIDES
    • 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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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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Application Number Title Priority Date Filing Date
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)

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HRP20240257T1 (hr) 2017-02-28 2024-05-24 The Trustees Of The University Of Pennsylvania Adeno-povezani virusni (aav) clade f vektor i njegova uporaba
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
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JP2023526310A (ja) 2020-05-12 2023-06-21 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 導入遺伝子発現のｄｒｇ特異的低減のための組成物
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang 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
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EP4286521A1 (en) * 2021-01-28 2023-12-06 Abrain Gene therapy for treating neurodegenerative diseases
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EP4370154A1 (en) * 2021-07-15 2024-05-22 The Regents Of The University Of Michigan Compositions and methods for treating alpha-synucleinopathies
TW202325845A (zh) 2021-10-02 2023-07-01 賓州大學委員會 新穎aav衣殼及含其之組成物
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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

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