CA3177749A1 - Compositions comprising a cas12i2 variant polypeptide and uses thereof - Google Patents

Compositions comprising a cas12i2 variant polypeptide and uses thereof

Info

Publication number
CA3177749A1
CA3177749A1 CA3177749A CA3177749A CA3177749A1 CA 3177749 A1 CA3177749 A1 CA 3177749A1 CA 3177749 A CA3177749 A CA 3177749A CA 3177749 A CA3177749 A CA 3177749A CA 3177749 A1 CA3177749 A1 CA 3177749A1
Authority
CA
Canada
Prior art keywords
composition
variant
cas12i2
polypeptide
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3177749A
Other languages
English (en)
French (fr)
Inventor
Shaorong Chong
Brendan Jay HILBERT
Quinton Norman WESSELLS
Noah Michael Jakimo
Roy ZIBLAT
Jason Michael CARTE
Tia Marie DITOMMASO
Jeffrey Raymond HASWELL
Anthony James Garrity
Colin Alexander MCGAW
David A. Scott
Derek Michael CERCHIONE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arbor Biotechnologies Inc
Original Assignee
Arbor Biotechnologies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arbor Biotechnologies Inc filed Critical Arbor Biotechnologies Inc
Publication of CA3177749A1 publication Critical patent/CA3177749A1/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
CA3177749A 2020-03-31 2021-03-31 Compositions comprising a cas12i2 variant polypeptide and uses thereof Pending CA3177749A1 (en)

Applications Claiming Priority (37)

Application Number Priority Date Filing Date Title
US202063003090P 2020-03-31 2020-03-31
US202063002999P 2020-03-31 2020-03-31
US202063003064P 2020-03-31 2020-03-31
US63/003,064 2020-03-31
US63/003,090 2020-03-31
US63/002,999 2020-03-31
US202063012007P 2020-04-17 2020-04-17
US63/012,007 2020-04-17
US202063026562P 2020-05-18 2020-05-18
US202063026277P 2020-05-18 2020-05-18
US202063026523P 2020-05-18 2020-05-18
US202063026606P 2020-05-18 2020-05-18
US63/026,523 2020-05-18
US63/026,606 2020-05-18
US63/026,277 2020-05-18
US63/026,562 2020-05-18
US202063063819P 2020-08-10 2020-08-10
US202063063859P 2020-08-10 2020-08-10
US202063063831P 2020-08-10 2020-08-10
US202063063879P 2020-08-10 2020-08-10
US63/063,819 2020-08-10
US63/063,879 2020-08-10
US63/063,831 2020-08-10
US63/063,859 2020-08-10
US202063085752P 2020-09-30 2020-09-30
US202063085792P 2020-09-30 2020-09-30
US202063085895P 2020-09-30 2020-09-30
US202063085862P 2020-09-30 2020-09-30
US63/085,792 2020-09-30
US63/085,895 2020-09-30
US63/085,752 2020-09-30
US63/085,862 2020-09-30
US202163147850P 2021-02-10 2021-02-10
US202163147968P 2021-02-10 2021-02-10
US63/147,968 2021-02-10
US63/147,850 2021-02-10
PCT/US2021/025257 WO2021202800A1 (en) 2020-03-31 2021-03-31 Compositions comprising a cas12i2 variant polypeptide and uses thereof

Publications (1)

Publication Number Publication Date
CA3177749A1 true CA3177749A1 (en) 2021-10-07

Family

ID=75640000

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3177749A Pending CA3177749A1 (en) 2020-03-31 2021-03-31 Compositions comprising a cas12i2 variant polypeptide and uses thereof

Country Status (12)

Country Link
US (1) US20230332119A1 (enExample)
EP (1) EP4127154A1 (enExample)
JP (1) JP2023520504A (enExample)
KR (1) KR20230009379A (enExample)
CN (2) CN121380024A (enExample)
AU (1) AU2021249119A1 (enExample)
BR (1) BR112022019713A2 (enExample)
CA (1) CA3177749A1 (enExample)
CO (1) CO2022015170A2 (enExample)
IL (1) IL296791A (enExample)
MX (1) MX2022012189A (enExample)
WO (1) WO2021202800A1 (enExample)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US20230193243A1 (en) * 2020-05-29 2023-06-22 Arbor Biotechnologies, Inc. Compositions comprising a cas12i2 polypeptide and uses thereof
EP4237557A1 (en) * 2020-10-30 2023-09-06 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting pdcd1 and uses thereof
EP4347818A2 (en) * 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
IL308803A (en) 2021-06-04 2024-01-01 Arbor Biotechnologies Inc Gene editing systems comprising guide RNA targeting hydroxy acid oxidase 1 (HAO1) and uses thereof
US20230034581A1 (en) * 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
WO2022256619A2 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
WO2023018856A1 (en) 2021-08-11 2023-02-16 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting polypyrimidine tract binding protein 1 (ptbp1) and uses thereof
US20230203539A1 (en) 2021-08-11 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
WO2023034475A1 (en) 2021-09-01 2023-03-09 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
EP4399293A4 (en) * 2021-09-10 2025-10-15 Arbor Biotechnologies Inc COMPOSITIONS COMPRISING A CAS12I POLYPEPTIDE AND USES THEREOF
CA3237337A1 (en) * 2021-11-02 2023-05-11 Huidagene Therapeutics (Singapore) Pte. Ltd. Novel crispr-cas12i systems and uses thereof
TW202334421A (zh) * 2021-11-05 2023-09-01 美商阿伯生物技術公司 包含靶向ciita之rna引導之組合物及其用途
WO2023122433A1 (en) 2021-12-22 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha)
CN118749029A (zh) * 2022-01-24 2024-10-08 辉大(上海)生物科技有限公司 新型CRISPR-Cas12i系统及其用途
CN118995668A (zh) * 2022-04-25 2024-11-22 辉大基因治疗(新加坡)私人有限公司 新型CRISPR-Cas12i系统及其用途
EP4627083A1 (en) 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
WO2025096594A2 (en) 2023-10-30 2025-05-08 Allogene Therapeutics, Inc. Cells expressing anti-cd19 chimeric antigen receptors and methods of use thereof
CN117431235A (zh) * 2023-11-06 2024-01-23 微光基因(苏州)有限公司 CRISPR-Cas系统及其应用
WO2025147604A1 (en) 2024-01-05 2025-07-10 Arbor Biotechnologies, Inc. Methods for treating primary hyperoxaluria via genetic editing of hydroxyacid oxidase 1
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such
CN120424908B (zh) * 2025-07-10 2025-09-12 南京农业大学 一种基于LAMP与CRISPR/Cas的检测方法和应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CN105492611A (zh) * 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
WO2019178427A1 (en) * 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
JP7693552B2 (ja) * 2019-02-13 2025-06-17 ビーム セラピューティクス インク. アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
WO2024178144A1 (en) * 2023-02-22 2024-08-29 Prime Medicine, Inc. Methods and compositions for editing nucleotide sequences

Also Published As

Publication number Publication date
WO2021202800A1 (en) 2021-10-07
CN121380024A (zh) 2026-01-23
CO2022015170A2 (es) 2022-11-08
IL296791A (en) 2022-11-01
JP2023520504A (ja) 2023-05-17
BR112022019713A2 (pt) 2022-12-20
MX2022012189A (es) 2023-01-05
CN115698278A (zh) 2023-02-03
KR20230009379A (ko) 2023-01-17
EP4127154A1 (en) 2023-02-08
CN115698278B (zh) 2025-10-17
US20230332119A1 (en) 2023-10-19
AU2021249119A1 (en) 2022-10-20

Similar Documents

Publication Publication Date Title
CN115698278B (zh) 包含Cas12i2变体多肽的组合物及其用途
US20240174997A1 (en) Compositions comprising a variant polypeptide and uses thereof
US20240035010A1 (en) Compositions comprising a variant polypeptide and uses thereof
US20250290053A1 (en) Compositions comprising a crispr nuclease and uses thereof
US11946045B2 (en) Compositions comprising a variant polypeptide and uses thereof
US11866746B2 (en) Compositions comprising a variant Cas12i4 polypeptide and uses thereof
US20240352434A1 (en) Compositions comprising a variant cas12i3 polypeptide and uses thereof
WO2024020557A1 (en) Compositions comprising a variant nuclease and uses thereof
WO2022150608A1 (en) Compositions comprising a variant crispr nuclease polypeptide and uses thereof
CN117136233A (zh) 包含变体Cas12i4多肽的组合物及其用途
CN117043326A (zh) 包含变体多肽的组合物及其用途

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220928

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250226

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250819

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250912

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20260109

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20260126

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20260126

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260126

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260219

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260219