CO2019007776A2 - Composiciones y métodos para tratar la deficiencia de alfa-1 antitripsina - Google Patents

Composiciones y métodos para tratar la deficiencia de alfa-1 antitripsina

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Publication number
CO2019007776A2
CO2019007776A2 CONC2019/0007776A CO2019007776A CO2019007776A2 CO 2019007776 A2 CO2019007776 A2 CO 2019007776A2 CO 2019007776 A CO2019007776 A CO 2019007776A CO 2019007776 A2 CO2019007776 A2 CO 2019007776A2
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CO
Colombia
Prior art keywords
methods
compositions
antitrypsin deficiency
treating alpha
antitrypsin
Prior art date
Application number
CONC2019/0007776A
Other languages
English (en)
Inventor
Walter Strapps
Shobu Odate
Reynald Michael Lescarbeau
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.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CO2019007776A2 publication Critical patent/CO2019007776A2/es

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Abstract

RESUMEN Se proporcionan composiciones y métodos para introducir rupturas de doble cadena dentro del gen de SERPINA1. Se proporcionan composiciones y métodos para reducir y eliminar las formas mutantes de α1-antitripsina (AAT), tal como se observa en los sujetos que tienen deficiencia de α1-antitripsina (AATD).
CONC2019/0007776A 2016-12-22 2019-07-19 Composiciones y métodos para tratar la deficiencia de alfa-1 antitripsina CO2019007776A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

Publications (1)

Publication Number Publication Date
CO2019007776A2 true CO2019007776A2 (es) 2019-10-09

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ID=61054487

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CONC2019/0007776A CO2019007776A2 (es) 2016-12-22 2019-07-19 Composiciones y métodos para tratar la deficiencia de alfa-1 antitripsina

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US (2) US11549107B2 (es)
EP (1) EP3559232A1 (es)
JP (2) JP7383478B2 (es)
KR (1) KR102551664B1 (es)
CN (2) CN110382697B (es)
AU (2) AU2017379073B2 (es)
BR (1) BR112019012825A2 (es)
CA (1) CA3047415A1 (es)
CO (1) CO2019007776A2 (es)
EA (1) EA201991455A1 (es)
IL (1) IL267448A (es)
MX (1) MX2019007594A (es)
NZ (1) NZ754739A (es)
PH (1) PH12019501457A1 (es)
SG (1) SG10202106412RA (es)
TW (1) TW201833331A (es)
WO (1) WO2018119182A1 (es)
ZA (1) ZA202007632B (es)

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* Cited by examiner, † Cited by third party
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NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
CA3077413A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. Formulations
WO2020006708A1 (en) * 2018-07-04 2020-01-09 Yale University Compositions and methods for enhancement of homology-directed repair mediated precise gene editing by programming dna repair with a single rna-guided endonuclease
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
JP7472121B2 (ja) * 2018-10-18 2024-04-22 インテリア セラピューティクス，インコーポレーテッド アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination

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NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020082047A1 (en) * 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey

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NZ754739A (en) 2024-01-26
ZA202007632B (en) 2023-03-29
IL267448A (en) 2019-08-29
SG10202106412RA (en) 2021-07-29
JP2020501582A (ja) 2020-01-23
MX2019007594A (es) 2019-12-16
PH12019501457A1 (en) 2020-03-09
US11549107B2 (en) 2023-01-10
JP7383478B2 (ja) 2023-11-20
WO2018119182A1 (en) 2018-06-28
TW201833331A (zh) 2018-09-16
KR102551664B1 (ko) 2023-07-05
EA201991455A1 (ru) 2020-01-15
EP3559232A1 (en) 2019-10-30
CN117737062A (zh) 2024-03-22
CA3047415A1 (en) 2018-06-28
AU2024201464A1 (en) 2024-03-28
US20230212575A1 (en) 2023-07-06
KR20190100943A (ko) 2019-08-29
BR112019012825A2 (pt) 2019-11-26
AU2017379073B2 (en) 2023-12-14
US20190316129A1 (en) 2019-10-17
JP2023075164A (ja) 2023-05-30
AU2017379073A1 (en) 2019-07-11
CN110382697A (zh) 2019-10-25
CN110382697B (zh) 2023-12-29

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