BR112022003389A2 - Proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico - Google Patents

Proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico

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Publication number
BR112022003389A2
BR112022003389A2 BR112022003389A BR112022003389A BR112022003389A2 BR 112022003389 A2 BR112022003389 A2 BR 112022003389A2 BR 112022003389 A BR112022003389 A BR 112022003389A BR 112022003389 A BR112022003389 A BR 112022003389A BR 112022003389 A2 BR112022003389 A2 BR 112022003389A2
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Brazil
Prior art keywords
capsid
isolated
aav5
adeno
associated virus
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BR112022003389A
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English (en)
Inventor
Pavel Mikhailovich Gershovich
Elena Victorovna Iurlova
Aleksandr Vladimirovich Karabelskii
Dmitrij Aleksandrovich Madera
Dmitry Valentinovich Morozov
Mariya Pavlovna Perepelkina
Alexandr Vladimirovich Prokofyev
Anna Nikolaevna Strelkova
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Llc «Anabion»
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Application filed by Llc «Anabion» filed Critical Llc «Anabion»
Publication of BR112022003389A2 publication Critical patent/BR112022003389A2/pt

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    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
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    • C40B40/06Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
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    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • 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

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Abstract

proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico. este pedido refere-se aos campos de terapia genética e biologia molecular. mais especificamente, a presente invenção refere-se a uma proteína vp1 alterada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5) compreendendo uma ou mais substituições de aminoácidos em comparação com a proteína vp1 do capsídeo do aav5 de tipo selvagem, que aumenta a eficiência de transdução, bem como a um capsídeo e um vetor nele baseados.
BR112022003389A 2019-08-22 2020-08-21 Proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico BR112022003389A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2019126509A RU2751592C2 (ru) 2019-08-22 2019-08-22 Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
PCT/RU2020/000445 WO2021034222A1 (ru) 2019-08-22 2020-08-21 Выделенный модифицированный белок vp1 капсида aav5

Publications (1)

Publication Number Publication Date
BR112022003389A2 true BR112022003389A2 (pt) 2022-05-17

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BR112022003389A BR112022003389A2 (pt) 2019-08-22 2020-08-21 Proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico

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US (1) US20220306696A1 (pt)
EP (1) EP4019642A4 (pt)
JP (1) JP2022545121A (pt)
KR (1) KR20220158674A (pt)
CN (1) CN114981444A (pt)
AR (1) AR119869A1 (pt)
AU (1) AU2020331882A1 (pt)
BR (1) BR112022003389A2 (pt)
CA (1) CA3148964A1 (pt)
CO (1) CO2022001833A2 (pt)
EC (1) ECSP22021725A (pt)
IL (1) IL290802A (pt)
JO (1) JOP20220043A1 (pt)
MA (1) MA56142B1 (pt)
MX (1) MX2022002184A (pt)
PE (1) PE20221254A1 (pt)
RU (1) RU2751592C2 (pt)
TW (1) TW202120690A (pt)
WO (1) WO2021034222A1 (pt)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
CN113121654B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
AR126840A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta

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Publication number Priority date Publication date Assignee Title
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
AU2002360291A1 (en) * 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
RS60207B1 (sr) 2011-04-22 2020-06-30 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
IN2014DN08812A (pt) 2012-04-18 2015-05-22 Philadelphia Children Hospital
KR102380265B1 (ko) * 2013-07-22 2022-03-29 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
PT3356390T (pt) * 2015-09-28 2021-04-21 Univ Florida Métodos e composições para vetores virais que se evadem a anticorpos
WO2017201121A1 (en) * 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs

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KR20220158674A (ko) 2022-12-01
US20220306696A1 (en) 2022-09-29
CN114981444A (zh) 2022-08-30
WO2021034222A9 (ru) 2021-04-22
RU2751592C2 (ru) 2021-07-15
MX2022002184A (es) 2022-05-24
AR119869A1 (es) 2022-01-19
JP2022545121A (ja) 2022-10-25
CO2022001833A2 (es) 2022-08-09
RU2019126509A (ru) 2021-06-04
MA56142A1 (fr) 2023-05-31
RU2019126509A3 (pt) 2021-06-04
JOP20220043A1 (ar) 2023-01-30
EP4019642A1 (en) 2022-06-29
TW202120690A (zh) 2021-06-01
CA3148964A1 (en) 2021-02-25
ECSP22021725A (es) 2022-08-31
PE20221254A1 (es) 2022-08-16
EP4019642A4 (en) 2023-09-06
WO2021034222A1 (ru) 2021-02-25
IL290802A (en) 2022-04-01
MA56142B1 (fr) 2023-09-27
AU2020331882A1 (en) 2022-11-03

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