AU2022342168A1 - Pah-modulating compositions and methods - Google Patents

Pah-modulating compositions and methods Download PDF

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Publication number
AU2022342168A1
AU2022342168A1 AU2022342168A AU2022342168A AU2022342168A1 AU 2022342168 A1 AU2022342168 A1 AU 2022342168A1 AU 2022342168 A AU2022342168 A AU 2022342168A AU 2022342168 A AU2022342168 A AU 2022342168A AU 2022342168 A1 AU2022342168 A1 AU 2022342168A1
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Australia
Prior art keywords
pah
methods
modulating compositions
modulating
compositions
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AU2022342168A
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Inventor
Robert Charles ALTSHULER
Luciano Henrique APPONI
Anne Helen Bothmer
Daniel Raymond CHEE
Robert James Citorik
Cecilia Giovanna Silvia COTTA-RAMUSINO
Kyusik Kim
Randi Michelle KOTLAR
Gregory David MCALLISTER
William Querbes
Ananya RAY
Nathaniel Roquet
William Edward Salomon
Carlos Sanchez
Barrett Ethan Steinberg
Zhan Wang
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Flagship Pioneering Innovations VI Inc
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Flagship Pioneering Innovations VI Inc
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Publication of AU2022342168A1 publication Critical patent/AU2022342168A1/en
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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
    • C12N15/1137—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 enzymes
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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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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • C12N9/14—Hydrolases (3)
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    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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AU2022342168A 2021-09-08 2022-09-07 Pah-modulating compositions and methods Pending AU2022342168A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241897P 2021-09-08 2021-09-08
US63/241,897 2021-09-08
US202263303927P 2022-01-27 2022-01-27
US63/303,927 2022-01-27
US202263367025P 2022-06-24 2022-06-24
US63/367,025 2022-06-24
PCT/US2022/076058 WO2023039435A2 (en) 2021-09-08 2022-09-07 Pah-modulating compositions and methods

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AU2022342168A1 true AU2022342168A1 (en) 2024-03-28

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US (1) US12544458B2 (https=)
EP (1) EP4399306A4 (https=)
JP (1) JP2024533315A (https=)
KR (1) KR20240099164A (https=)
AU (1) AU2022342168A1 (https=)
CA (1) CA3231712A1 (https=)
IL (1) IL311218A (https=)
TW (1) TW202320810A (https=)
WO (1) WO2023039435A2 (https=)

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KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
AU2022342169A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
WO2023070062A2 (en) 2021-10-21 2023-04-27 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
US20250011766A1 (en) 2021-11-09 2025-01-09 Prime Medicine, Inc. Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis
EP4430185A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
EP4430189A1 (en) 2021-11-11 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fragile x syndrome
EP4437106A2 (en) 2021-11-24 2024-10-02 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
JP2024545947A (ja) 2021-11-24 2024-12-16 プライム メディシン， インコーポレイテッド 修飾されたプライム編集ガイドｒｎａ
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
EP4504921A4 (en) 2022-04-01 2026-04-15 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTID SEQUENCES

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CA3231712A1 (en) 2023-03-16
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