CA3134271A1 - Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent - Google Patents

Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent Download PDF

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Publication number
CA3134271A1
CA3134271A1 CA3134271A CA3134271A CA3134271A1 CA 3134271 A1 CA3134271 A1 CA 3134271A1 CA 3134271 A CA3134271 A CA 3134271A CA 3134271 A CA3134271 A CA 3134271A CA 3134271 A1 CA3134271 A1 CA 3134271A1
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Canada
Prior art keywords
composition
lipid
seq
ttr
rna
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Pending
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CA3134271A
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English (en)
French (fr)
Inventor
Yong Chang
Seth C. Alexander
Kristy M. WOOD
Arti Mahendra Prakash KANJOLIA
Shobu ODATE
Jessica Lynn SEITZER
Reynald Michael LESCARBEAU
Walter Strapps
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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Publication of CA3134271A1 publication Critical patent/CA3134271A1/en
Pending legal-status Critical Current

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
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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
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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 [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2800/00—Nucleic acids vectors
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CA3134271A 2019-03-28 2020-03-27 Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent Pending CA3134271A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962825637P 2019-03-28 2019-03-28
US62/825,637 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

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CA3134271A1 true CA3134271A1 (en) 2020-10-01

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CA3134271A Pending CA3134271A1 (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

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US (2) US20230044994A1 (enExample)
EP (1) EP3946598A1 (enExample)
JP (2) JP7631215B2 (enExample)
KR (1) KR20220004648A (enExample)
CN (1) CN113874076A (enExample)
AU (1) AU2020244887A1 (enExample)
BR (1) BR112021019196A2 (enExample)
CA (1) CA3134271A1 (enExample)
CO (1) CO2021014559A2 (enExample)
EA (1) EA202192636A1 (enExample)
IL (1) IL286524A (enExample)
MX (1) MX2021011565A (enExample)
PH (1) PH12021552301A1 (enExample)
SG (1) SG11202110434PA (enExample)
WO (1) WO2020198697A1 (enExample)

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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
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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
EP4627083A1 (en) * 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
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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EP3946598A1 (en) 2022-02-09
PH12021552301A1 (en) 2022-07-04
JP2025072483A (ja) 2025-05-09
SG11202110434PA (en) 2021-10-28
BR112021019196A2 (pt) 2022-01-18
WO2020198697A1 (en) 2020-10-01
AU2020244887A1 (en) 2021-11-11
US20230044994A1 (en) 2023-02-09
KR20220004648A (ko) 2022-01-11
MX2021011565A (es) 2021-11-12
US20240124897A1 (en) 2024-04-18
IL286524A (en) 2021-10-31
CO2021014559A2 (es) 2021-11-19
JP7631215B2 (ja) 2025-02-18
EA202192636A1 (ru) 2022-03-17
CN113874076A (zh) 2021-12-31
JP2022525428A (ja) 2022-05-13

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