AU2017379073B2 - Compositions and methods for treating alpha-1 antitrypsin deficiency - Google Patents

Compositions and methods for treating alpha-1 antitrypsin deficiency Download PDF

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AU2017379073B2
AU2017379073B2 AU2017379073A AU2017379073A AU2017379073B2 AU 2017379073 B2 AU2017379073 B2 AU 2017379073B2 AU 2017379073 A AU2017379073 A AU 2017379073A AU 2017379073 A AU2017379073 A AU 2017379073A AU 2017379073 B2 AU2017379073 B2 AU 2017379073B2
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seq
rna
composition
sequence
guide
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AU2017379073A1 (en
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Reynald Michael LESCARBEAU
Shobu ODATE
Walter Strapps
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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    • C12N2310/35—Nature of the modification
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AU2017379073A 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency Active AU2017379073B2 (en)

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AU2024201464A AU2024201464A1 (en) 2016-12-22 2024-03-06 Compositions and methods for treating alpha-1 antitrypsin deficiency

Applications Claiming Priority (3)

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US201662438219P 2016-12-22 2016-12-22
US62/438,219 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

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AU2017379073B2 true AU2017379073B2 (en) 2023-12-14

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US (3) US11549107B2 (enExample)
EP (1) EP3559232A1 (enExample)
JP (2) JP7383478B2 (enExample)
KR (1) KR102551664B1 (enExample)
CN (2) CN117737062A (enExample)
AU (2) AU2017379073B2 (enExample)
BR (1) BR112019012825A2 (enExample)
CA (1) CA3047415A1 (enExample)
CO (1) CO2019007776A2 (enExample)
EA (1) EA201991455A1 (enExample)
IL (1) IL267448B2 (enExample)
MX (2) MX2019007594A (enExample)
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KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
MX2021004276A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina.
BR112021007343A2 (pt) * 2018-10-18 2021-08-03 Intellia Therapeutics, Inc. composições e métodos para expressão de transgene a partir de um lócus de albumina
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
WO2022060707A1 (en) * 2020-09-15 2022-03-24 Rutgers, The State University Of New Jersey Systems for gene editing and methods of use thereof
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
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
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

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