BR112021021979A2 - Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas - Google Patents

Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas

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Publication number
BR112021021979A2
BR112021021979A2 BR112021021979A BR112021021979A BR112021021979A2 BR 112021021979 A2 BR112021021979 A2 BR 112021021979A2 BR 112021021979 A BR112021021979 A BR 112021021979A BR 112021021979 A BR112021021979 A BR 112021021979A BR 112021021979 A2 BR112021021979 A2 BR 112021021979A2
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methods
treatment
dna molecule
dna
disease
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BR112021021979A
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English (en)
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R Edgell David
A Mcmurrough Thomas
E Stead Brent
K Israel Odisho
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Specific Biologics Inc
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Publication of BR112021021979A2 publication Critical patent/BR112021021979A2/pt

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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
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    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
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    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • A—HUMAN NECESSITIES
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    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract

POLIPEPTÍDEO, NUCLEASES QUIMÉRICAS, FORMULAÇÕES FARMACEUTICAMENTE ACEITÁVEIS, MÉTODOS PARA EDITAR DNA GENÔMICO, PARA DELETAR COMPRIMENTOS DEFINIDOS DE UMA MOLÉCULA DE DNA E PARA SUBSTITUIR SEQUÊNCIAS SELECIONADAS DE UMA MOLÉCULA DE DNA, PROCESSO, MÉTODOS DE ADMINISTRAÇÃO DA FORMULAÇÃO, DE TRATAMENTO DE UMA DOENÇA RELACIONADA AO PULMÃO E DE TRATAMENTO DE DOENÇAS MONOGENÉTICAS E INFECCIOSAS, LIGANTE E MOLÉCULAS DE DNA DOADORAS MODIFICADAS. Métodos para editar genes através da administração de uma nuclease quimérica a uma célula ou organismo sem o uso de um vetor viral.
BR112021021979A 2019-05-03 2020-05-04 Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas BR112021021979A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962842586P 2019-05-03 2019-05-03
US202063019423P 2020-05-03 2020-05-03
PCT/IB2020/054229 WO2020225719A1 (en) 2019-05-03 2020-05-04 Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing

Publications (1)

Publication Number Publication Date
BR112021021979A2 true BR112021021979A2 (pt) 2022-12-06

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BR112021021979A BR112021021979A2 (pt) 2019-05-03 2020-05-04 Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas

Country Status (13)

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US (3) US20220195404A1 (pt)
EP (1) EP3963062A4 (pt)
KR (1) KR20220115860A (pt)
CN (1) CN114761550A (pt)
AU (1) AU2020268959A1 (pt)
BR (1) BR112021021979A2 (pt)
CA (1) CA3139581A1 (pt)
GB (1) GB2600568B (pt)
IL (1) IL287788A (pt)
MX (1) MX2021013400A (pt)
PE (1) PE20221123A1 (pt)
SG (1) SG11202112161TA (pt)
WO (1) WO2020225719A1 (pt)

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EP3963062A4 (en) * 2019-05-03 2023-09-06 Specific Biologics Inc. LIPID ENCAPSULATED DOUBLE-CLEVISING ENDONUCLEASE FOR DNA AND GENE EDITING
CN116669775A (zh) * 2020-11-04 2023-08-29 特定生物制品公司 用修饰的核酸内切酶进行的基因编辑
AU2022242086A1 (en) * 2021-03-26 2023-10-19 Specific Biologics Inc. Targeting oncogentic mutations with dual-cleaving endonuclease
WO2023154451A1 (en) * 2022-02-10 2023-08-17 Christiana Care Gene Editing Institute, Inc. Methods for lipid nanoparticle delivery of crispr/cas system

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WO2013068845A2 (en) * 2011-11-07 2013-05-16 The University Of Western Ontario Endonuclease for genome editing
DK3064585T3 (da) 2012-12-12 2020-04-27 Broad Inst Inc Konstruering og optimering af forbedrede systemer, fremgangsmåder og enzymsammensætninger til sekvensmanipulation
SG11201504523UA (en) * 2012-12-12 2015-07-30 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014121222A1 (en) * 2013-02-01 2014-08-07 The University Of Western Ontario Endonuclease for genome editing
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EP3963062A4 (en) * 2019-05-03 2023-09-06 Specific Biologics Inc. LIPID ENCAPSULATED DOUBLE-CLEVISING ENDONUCLEASE FOR DNA AND GENE EDITING
CN116669775A (zh) * 2020-11-04 2023-08-29 特定生物制品公司 用修饰的核酸内切酶进行的基因编辑

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GB202117443D0 (en) 2022-01-19
US11814658B2 (en) 2023-11-14
WO2020225719A1 (en) 2020-11-12
GB2600568A (en) 2022-05-04
US20230016280A1 (en) 2023-01-19
MX2021013400A (es) 2022-04-20
IL287788A (en) 2022-01-01
GB2600568B (en) 2024-07-31
KR20220115860A (ko) 2022-08-19
US20230203464A1 (en) 2023-06-29
EP3963062A1 (en) 2022-03-09
AU2020268959A1 (en) 2021-12-09
GB2600568A8 (en) 2022-05-18
EP3963062A4 (en) 2023-09-06
PE20221123A1 (es) 2022-07-13
CA3139581A1 (en) 2020-11-12
CN114761550A (zh) 2022-07-15
US20220195404A1 (en) 2022-06-23
SG11202112161TA (en) 2021-12-30

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