AU2020221355A1 - Compositions and methods for treating glycogen storage disease type 1a - Google Patents

Compositions and methods for treating glycogen storage disease type 1a Download PDF

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Publication number
AU2020221355A1
AU2020221355A1 AU2020221355A AU2020221355A AU2020221355A1 AU 2020221355 A1 AU2020221355 A1 AU 2020221355A1 AU 2020221355 A AU2020221355 A AU 2020221355A AU 2020221355 A AU2020221355 A AU 2020221355A AU 2020221355 A1 AU2020221355 A1 AU 2020221355A1
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Australia
Prior art keywords
tada
adenosine deaminase
domain
cas9
variant
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AU2020221355A
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Inventor
Yvonne ARATYN
David A. BORN
Nicole GAUDELLI
Francine Gregoire
Seung-Joo Lee
Genesis LUNG
Michael Packer
Ian SLAYMAKER
Yi Yu
Bernd ZETSCHE
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • 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
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    • 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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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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)
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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]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)

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AU2020221355A 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a Pending AU2020221355A1 (en)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962852228P 2019-05-23 2019-05-23
US201962852224P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962876354P 2019-07-19 2019-07-19
US62/876,354 2019-07-19
US201962912992P 2019-10-09 2019-10-09
US62/912,992 2019-10-09
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018124 WO2020168088A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a

Publications (1)

Publication Number Publication Date
AU2020221355A1 true AU2020221355A1 (en) 2021-08-12

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AU2020221355A Pending AU2020221355A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a

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US (1) US20220127594A1 (https=)
EP (1) EP3924478A4 (https=)
JP (2) JP7631209B2 (https=)
KR (1) KR20210129108A (https=)
CN (2) CN120138047A (https=)
AU (1) AU2020221355A1 (https=)
CA (1) CA3128886A1 (https=)
WO (1) WO2020168088A1 (https=)

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EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
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CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3198671A1 (en) * 2020-10-14 2022-04-21 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
EP4504947A4 (en) * 2022-04-07 2026-03-25 Univ Duke COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
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CA3128886A1 (en) 2020-08-20
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CN114026237A (zh) 2022-02-08
JP7631209B2 (ja) 2025-02-18
CN120138047A (zh) 2025-06-13
JP2022519882A (ja) 2022-03-25
EP3924478A1 (en) 2021-12-22
US20220127594A1 (en) 2022-04-28
WO2020168088A1 (en) 2020-08-20
KR20210129108A (ko) 2021-10-27

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