BR112023003929A2 - Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado - Google Patents

Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado

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Publication number
BR112023003929A2
BR112023003929A2 BR112023003929A BR112023003929A BR112023003929A2 BR 112023003929 A2 BR112023003929 A2 BR 112023003929A2 BR 112023003929 A BR112023003929 A BR 112023003929A BR 112023003929 A BR112023003929 A BR 112023003929A BR 112023003929 A2 BR112023003929 A2 BR 112023003929A2
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BR
Brazil
Prior art keywords
expression cassette
replacement therapy
targeted gene
gene replacement
pku
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Application number
BR112023003929A
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English (en)
Inventor
R M Kyostio-Moore Sirkka
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Genzyme Corp
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of BR112023003929A2 publication Critical patent/BR112023003929A2/pt

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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/51—Lyases (4)
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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Abstract

CASSETE DE EXPRESSÃO DE PAH HUMANO PARA TRATAMENTO DE PKU POR TERAPIA DE REPOSIÇÃO GÊNICA DIRECIONADA AO FÍGADO. A presente invenção refere-se a cassetes de expressão para expressar um transgene em uma célula hepática, em que o transgene codifica um polipeptídeo da PAH. Também são fornecidos métodos para tratar a fenilcetonúria (PKU) e/ou para reduzir os níveis de fenilalanina em um indivíduo em necessidade dos mesmos. Além disso, são fornecidos neste documento vetores (por exemplo, vetores do rAAV), partículas virais, composições farmacêuticas e kits para expressar um polipeptídeo da PAH em um indivíduo em necessidade dos mesmos.
BR112023003929A 2020-10-01 2021-09-30 Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado BR112023003929A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063086537P 2020-10-01 2020-10-01
US202063121797P 2020-12-04 2020-12-04
PCT/US2021/052913 WO2022072657A1 (en) 2020-10-01 2021-09-30 Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy

Publications (1)

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BR112023003929A2 true BR112023003929A2 (pt) 2023-04-11

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Country Status (12)

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US (1) US20220112520A1 (pt)
EP (1) EP4222251A1 (pt)
JP (1) JP2023544165A (pt)
KR (1) KR20230079172A (pt)
AU (1) AU2021355481A1 (pt)
BR (1) BR112023003929A2 (pt)
CA (1) CA3193866A1 (pt)
CO (1) CO2023003418A2 (pt)
IL (1) IL301677A (pt)
MX (1) MX2023003805A (pt)
TW (1) TW202229559A (pt)
WO (1) WO2022072657A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3863662A1 (en) * 2018-10-12 2021-08-18 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
CN115896135B (zh) * 2022-11-02 2024-03-01 苏州诺洁贝生物技术有限公司 优化的pah基因和表达盒及其用途

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US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
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EP1204739B1 (en) 1999-08-09 2008-08-06 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
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
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
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EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US8586006B2 (en) * 2006-08-09 2013-11-19 Institute For Systems Biology Organ-specific proteins and methods of their use
KR101812813B1 (ko) 2009-06-16 2017-12-27 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
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EP3863662A1 (en) 2018-10-12 2021-08-18 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy

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Publication number Publication date
EP4222251A1 (en) 2023-08-09
AU2021355481A1 (en) 2023-06-08
CA3193866A1 (en) 2022-04-07
JP2023544165A (ja) 2023-10-20
AU2021355481A9 (en) 2024-07-25
KR20230079172A (ko) 2023-06-05
US20220112520A1 (en) 2022-04-14
MX2023003805A (es) 2023-04-12
TW202229559A (zh) 2022-08-01
WO2022072657A1 (en) 2022-04-07
IL301677A (en) 2023-05-01
CO2023003418A2 (es) 2023-03-27

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