JP2023518541A5 - - Google Patents

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Publication number
JP2023518541A5
JP2023518541A5 JP2022556229A JP2022556229A JP2023518541A5 JP 2023518541 A5 JP2023518541 A5 JP 2023518541A5 JP 2022556229 A JP2022556229 A JP 2022556229A JP 2022556229 A JP2022556229 A JP 2022556229A JP 2023518541 A5 JP2023518541 A5 JP 2023518541A5
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JP
Japan
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JP2022556229A
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Japanese (ja)
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JP7847853B2 (ja
JP2023518541A (ja
JPWO2021188729A5 (https=
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Priority claimed from PCT/US2021/022840 external-priority patent/WO2021188729A1/en
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Publication of JPWO2021188729A5 publication Critical patent/JPWO2021188729A5/ja
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JP2022556229A 2020-03-18 2021-03-17 C9orf72の標的化のための組成物及び方法 Active JP7847853B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062991403P 2020-03-18 2020-03-18
US62/991,403 2020-03-18
PCT/US2021/022840 WO2021188729A1 (en) 2020-03-18 2021-03-17 Compositions and methods for the targeting of c9orf72

Publications (4)

Publication Number Publication Date
JP2023518541A JP2023518541A (ja) 2023-05-02
JP2023518541A5 true JP2023518541A5 (https=) 2024-03-28
JPWO2021188729A5 JPWO2021188729A5 (https=) 2024-03-28
JP7847853B2 JP7847853B2 (ja) 2026-04-20

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ID=75478206

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JP2022556229A Active JP7847853B2 (ja) 2020-03-18 2021-03-17 C9orf72の標的化のための組成物及び方法

Country Status (12)

Country Link
US (1) US20240309344A1 (https=)
EP (1) EP4121535A1 (https=)
JP (1) JP7847853B2 (https=)
KR (1) KR20230002401A (https=)
CN (1) CN116096885A (https=)
AU (1) AU2021237633A1 (https=)
BR (1) BR112022018673A2 (https=)
CA (1) CA3172178A1 (https=)
CO (1) CO2022014598A2 (https=)
IL (1) IL296477A (https=)
MX (1) MX2022011460A (https=)
WO (1) WO2021188729A1 (https=)

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PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
EP4288541A1 (en) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
EP4423263A4 (en) * 2021-10-29 2026-03-04 Mammoth Biosciences Inc EFFECTOR PROTEINS, COMPOSITIONS, SYSTEMS, DEVICES, KITS AND METHODS FOR THERECOMMENDATION
US20250011766A1 (en) * 2021-11-09 2025-01-09 Prime Medicine, Inc. Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis
WO2023235725A2 (en) * 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024173479A2 (en) * 2023-02-15 2024-08-22 The Trustees Of Columbia University In The City Of New York Compositions and methods for treating motor neuron diseases
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2025024285A1 (en) * 2023-07-21 2025-01-30 Mammoth Biosciences, Inc. Compositions for the modification of the human c9orf72 gene
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
BR112018012894A2 (en) * 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
CA3038960A1 (en) * 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
WO2018208972A1 (en) * 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2019030306A1 (en) * 2017-08-08 2019-02-14 Depixus ISOLATION AND IN VITRO ENRICHMENT OF NUCLEIC ACIDS USING SITE-SPECIFIC NUCLEASES
US11578334B2 (en) * 2017-10-25 2023-02-14 Monsanto Technology Llc Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems

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