MX420023B - Moléculas guía sintéticas, composiciones y métodos relacionados con éstas. - Google Patents

Moléculas guía sintéticas, composiciones y métodos relacionados con éstas.

Info

Publication number
MX420023B
MX420023B MX2019007750A MX2019007750A MX420023B MX 420023 B MX420023 B MX 420023B MX 2019007750 A MX2019007750 A MX 2019007750A MX 2019007750 A MX2019007750 A MX 2019007750A MX 420023 B MX420023 B MX 420023B
Authority
MX
Mexico
Prior art keywords
compositions
related methods
guide molecules
synthetic guide
molecules
Prior art date
Application number
MX2019007750A
Other languages
English (en)
Spanish (es)
Other versions
MX2019007750A (es
Inventor
Bruce Eaton
Cecilia Fernandez
Hariharan Jayaram
Jim Heil
Karin Zemski Berry
Sam Saccomano
Stacy Capehart
Stephanie King
Original Assignee
Editas Medicine 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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2019007750A publication Critical patent/MX2019007750A/es
Publication of MX420023B publication Critical patent/MX420023B/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
MX2019007750A 2016-12-30 2017-12-29 Moléculas guía sintéticas, composiciones y métodos relacionados con éstas. MX420023B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662441046P 2016-12-30 2016-12-30
US201762492001P 2017-04-28 2017-04-28
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto

Publications (2)

Publication Number Publication Date
MX2019007750A MX2019007750A (es) 2019-10-15
MX420023B true MX420023B (es) 2025-01-14

Family

ID=61187807

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019007750A MX420023B (es) 2016-12-30 2017-12-29 Moléculas guía sintéticas, composiciones y métodos relacionados con éstas.

Country Status (9)

Country Link
US (1) US20230111575A1 (enExample)
EP (1) EP3565895A1 (enExample)
JP (3) JP7167029B2 (enExample)
KR (2) KR102618864B1 (enExample)
CN (2) CN110249052B (enExample)
AU (2) AU2017388753A1 (enExample)
CA (1) CA3048434A1 (enExample)
MX (1) MX420023B (enExample)
WO (1) WO2018126176A1 (enExample)

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US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
WO2018041973A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
KR102709603B1 (ko) 2017-02-28 2024-09-27 보르 바이오파마 인크. 계통 특이적 단백질의 억제를 위한 조성물 및 방법
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3681637A4 (en) 2017-09-11 2021-06-02 Synthego Corporation SYSTEM AND PROCEDURE FOR BIOPOLYMER SYNTHESIS
IL277234B2 (en) 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
GB201901873D0 (en) * 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
AU2020280103A1 (en) 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
AU2020338011A1 (en) 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2021041977A1 (en) 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cd123 modification
EP4204564A1 (en) 2020-08-28 2023-07-05 Vor Biopharma Inc. Compositions and methods for cd123 modification
EP4204565A1 (en) 2020-08-28 2023-07-05 Vor Biopharma Inc. Compositions and methods for cll1 modification
WO2022056489A1 (en) 2020-09-14 2022-03-17 Vor Biopharma, Inc. Compositions and methods for cd38 modification
EP4211245A1 (en) 2020-09-14 2023-07-19 Vor Biopharma Inc. Compositions and methods for cd5 modification
WO2022061115A1 (en) 2020-09-18 2022-03-24 Vor Biopharma Inc. Compositions and methods for cd7 modification
WO2022067240A1 (en) 2020-09-28 2022-03-31 Vor Biopharma, Inc. Compositions and methods for cd6 modification
WO2022072643A1 (en) 2020-09-30 2022-04-07 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
CA3199623A1 (en) 2020-10-27 2022-05-05 Vor Biopharma Inc. Compositions and methods for treating hematopoietic malignancy
WO2022094245A1 (en) 2020-10-30 2022-05-05 Vor Biopharma, Inc. Compositions and methods for bcma modification
WO2022104090A1 (en) 2020-11-13 2022-05-19 Vor Biopharma Inc. Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors
AU2021413252A1 (en) 2020-12-31 2023-06-08 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
WO2022217086A1 (en) 2021-04-09 2022-10-13 Vor Biopharma Inc. Photocleavable guide rnas and methods of use thereof
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
EP4381062A1 (en) 2021-08-02 2024-06-12 Vor Biopharma Inc. Compositions and methods for gene modification
EP4408991A2 (en) 2021-09-27 2024-08-07 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
EP4430186A1 (en) 2021-11-09 2024-09-18 Vor Biopharma Inc. Compositions and methods for erm2 modification
US20250179531A1 (en) 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
US20250295695A1 (en) 2022-04-04 2025-09-25 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
WO2024015925A2 (en) 2022-07-13 2024-01-18 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2024112876A2 (en) * 2022-11-23 2024-05-30 Prime Medicine, Inc. Split synthesis of long rnas
WO2024168312A1 (en) 2023-02-09 2024-08-15 Vor Biopharma Inc. Methods for treating hematopoietic malignancy
WO2025006216A2 (en) * 2023-06-29 2025-01-02 Synthego Corporation Methods and systems for automated fraction selection in nucleic acid manufacturing
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome

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US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
EP3981876A1 (en) * 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
CN113930455A (zh) * 2014-10-09 2022-01-14 生命技术公司 Crispr寡核苷酸和基因剪辑
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US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
CN107787367B (zh) * 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
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EP3763814A1 (en) * 2015-05-08 2021-01-13 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
CN107709555A (zh) * 2015-05-15 2018-02-16 达尔马科恩有限公司 用于Cas9介导的基因编辑的合成的单向导RNA
WO2017131237A1 (ja) * 2016-01-30 2017-08-03 株式会社ボナック 人工単一ガイドrna及びその用途
WO2017180711A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof

Also Published As

Publication number Publication date
JP7167029B2 (ja) 2022-11-08
WO2018126176A1 (en) 2018-07-05
JP2020503049A (ja) 2020-01-30
CN110249052A (zh) 2019-09-17
US20230111575A1 (en) 2023-04-13
EP3565895A1 (en) 2019-11-13
KR20230175330A (ko) 2023-12-29
KR102618864B1 (ko) 2024-01-02
AU2017388753A1 (en) 2019-07-11
CA3048434A1 (en) 2018-07-05
JP2022173349A (ja) 2022-11-18
JP7610559B2 (ja) 2025-01-08
CN110249052B (zh) 2024-04-12
AU2024205204A1 (en) 2024-09-05
MX2019007750A (es) 2019-10-15
KR102758180B1 (ko) 2025-01-23
CN118345072A (zh) 2024-07-16
KR20190110554A (ko) 2019-09-30
JP2025016776A (ja) 2025-02-04

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