MX2021011565A - Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn. - Google Patents

Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn.

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Publication number
MX2021011565A
MX2021011565A MX2021011565A MX2021011565A MX2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A
Authority
MX
Mexico
Prior art keywords
rna
compositions
methods
binding agent
polynucleotide encoding
Prior art date
Application number
MX2021011565A
Other languages
English (en)
Inventor
Kristy M Wood
Shobu Odate
Reynald Michael Lescarbeau
Seth C Alexander
Arti Mahendra Prakash Kanjolia
Jessica Lynn Seitzer
Yong Chang
Walter Strapps
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 MX2021011565A publication Critical patent/MX2021011565A/es

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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
    • A—HUMAN NECESSITIES
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    • 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
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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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    • C12N2310/00—Structure or type of the nucleic acid
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Abstract

Se proporcionan composiciones y métodos para editar, por ejemplo, introducir roturas bicatenarias, dentro del gen TTR. Se proporcionan composiciones y métodos para tratar a sujetos que tienen amiloidosis asociada con transtiretina (ATTR).
MX2021011565A 2019-03-28 2020-03-27 Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn. MX2021011565A (es)

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
MX2021011565A true MX2021011565A (es) 2021-11-12

Family

ID=70296147

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MX2021011565A MX2021011565A (es) 2019-03-28 2020-03-27 Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn.

Country Status (14)

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

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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
EA202192636A1 (ru) 2022-03-17
IL286524A (en) 2021-10-31
BR112021019196A2 (pt) 2022-01-18

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