BR112021019196A2 - Composições e métodos compreendendo um rna guia de ttr e um polinucleotídeo codificando um agente de ligação de dna guiado por rna - Google Patents

Composições e métodos compreendendo um rna guia de ttr e um polinucleotídeo codificando um agente de ligação de dna guiado por rna

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Publication number
BR112021019196A2
BR112021019196A2 BR112021019196A BR112021019196A BR112021019196A2 BR 112021019196 A2 BR112021019196 A2 BR 112021019196A2 BR 112021019196 A BR112021019196 A BR 112021019196A BR 112021019196 A BR112021019196 A BR 112021019196A BR 112021019196 A2 BR112021019196 A2 BR 112021019196A2
Authority
BR
Brazil
Prior art keywords
compositions
methods
rna
binding agent
polynucleotide encoding
Prior art date
Application number
BR112021019196A
Other languages
English (en)
Inventor
Mahendra Prakash Kanjolia Arti
Lynn Seitzer Jessica
M Wood Kristy
Michael Lescarbeau Reynald
C Alexander Seth
Shobu Odate
Walter Strapps
Yong Chang
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of BR112021019196A2 publication Critical patent/BR112021019196A2/pt

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • A—HUMAN NECESSITIES
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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Abstract

composições e métodos compreendendo um rna guia de ttr e um polinucleotídeo codificando um agente de ligação de dna guiado por rna. são fornecidas composições e métodos para edição, por exemplo, introdução de quebras de fita dupla, dentro do gene ttr . são fornecidas composições e métodos para tratar sujeitos tendo amiloidose associada à transtirretina (attr).
BR112021019196A 2019-03-28 2020-03-27 Composições e métodos compreendendo um rna guia de ttr e um polinucleotídeo codificando um agente de ligação de dna guiado por rna BR112021019196A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825637P 2019-03-28 2019-03-28
PCT/US2020/025513 WO2020198697A1 (en) 2019-03-28 2020-03-27 Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent

Publications (1)

Publication Number Publication Date
BR112021019196A2 true BR112021019196A2 (pt) 2022-01-18

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BR112021019196A BR112021019196A2 (pt) 2019-03-28 2020-03-27 Composições e métodos compreendendo um rna guia de ttr e um polinucleotídeo codificando um agente de ligação de dna guiado por rna

Country Status (14)

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US (2) US20230044994A1 (pt)
EP (1) EP3946598A1 (pt)
JP (1) JP2022525428A (pt)
KR (1) KR20220004648A (pt)
CN (1) CN113874076A (pt)
AU (1) AU2020244887A1 (pt)
BR (1) BR112021019196A2 (pt)
CA (1) CA3134271A1 (pt)
CO (1) CO2021014559A2 (pt)
EA (1) EA202192636A1 (pt)
IL (1) IL286524A (pt)
MX (1) MX2021011565A (pt)
SG (1) SG11202110434PA (pt)
WO (1) WO2020198697A1 (pt)

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CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
US20220396782A1 (en) * 2021-06-04 2022-12-15 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
EP4359531A1 (en) * 2021-06-22 2024-05-01 Intellia Therapeutics, Inc. Methods for in vivo editing of a liver gene
WO2023185697A2 (en) 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
WO2024061296A2 (en) 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
WO2024118747A1 (en) * 2022-11-30 2024-06-06 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof

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JP2022525428A (ja) 2022-05-13
WO2020198697A1 (en) 2020-10-01
CN113874076A (zh) 2021-12-31
CO2021014559A2 (es) 2021-11-19
CA3134271A1 (en) 2020-10-01
AU2020244887A1 (en) 2021-11-11
US20240124897A1 (en) 2024-04-18
KR20220004648A (ko) 2022-01-11
US20230044994A1 (en) 2023-02-09
EP3946598A1 (en) 2022-02-09
SG11202110434PA (en) 2021-10-28
MX2021011565A (es) 2021-11-12
EA202192636A1 (ru) 2022-03-17
IL286524A (en) 2021-10-31

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