CA3173012A1 - Compositions and methods using snrna components - Google Patents
Compositions and methods using snrna components Download PDFInfo
- Publication number
- CA3173012A1 CA3173012A1 CA3173012A CA3173012A CA3173012A1 CA 3173012 A1 CA3173012 A1 CA 3173012A1 CA 3173012 A CA3173012 A CA 3173012A CA 3173012 A CA3173012 A CA 3173012A CA 3173012 A1 CA3173012 A1 CA 3173012A1
- Authority
- CA
- Canada
- Prior art keywords
- bases
- engineered polynucleotide
- nucleotides
- engineered
- rna
- 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
Links
Classifications
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- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- 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)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- 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)
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Steroid Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063013774P | 2020-04-22 | 2020-04-22 | |
| US63/013,774 | 2020-04-22 | ||
| US202063030118P | 2020-05-26 | 2020-05-26 | |
| US63/030,118 | 2020-05-26 | ||
| US202063086434P | 2020-10-01 | 2020-10-01 | |
| US63/086,434 | 2020-10-01 | ||
| US202063112486P | 2020-11-11 | 2020-11-11 | |
| US63/112,486 | 2020-11-11 | ||
| US202063119878P | 2020-12-01 | 2020-12-01 | |
| US63/119,878 | 2020-12-01 | ||
| US202163153817P | 2021-02-25 | 2021-02-25 | |
| US63/153,817 | 2021-02-25 | ||
| PCT/US2021/028618 WO2021216853A1 (en) | 2020-04-22 | 2021-04-22 | Compositions and methods using snrna components |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3173012A1 true CA3173012A1 (en) | 2021-10-28 |
Family
ID=78270014
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3173012A Pending CA3173012A1 (en) | 2020-04-22 | 2021-04-22 | Compositions and methods using snrna components |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240011028A9 (https=) |
| EP (1) | EP4139453A1 (https=) |
| JP (1) | JP2023523237A (https=) |
| CN (1) | CN115777020A (https=) |
| AU (1) | AU2021261374A1 (https=) |
| CA (1) | CA3173012A1 (https=) |
| WO (1) | WO2021216853A1 (https=) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| MX2022006991A (es) * | 2019-12-09 | 2022-08-25 | Astellas Pharma Inc | Arn guía antisentido con región funcional añadida para editar arn blanco. |
| WO2022103852A1 (en) * | 2020-11-11 | 2022-05-19 | Shape Therapeutics Inc. | Rna-editing compositions and methods of use |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| US20250084408A1 (en) * | 2021-10-27 | 2025-03-13 | Shape Therapeutics Inc. | Engineered rnas |
| EP4441219A2 (en) * | 2021-12-01 | 2024-10-09 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides for rna editing targeting lrrk2 |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| JP2025507958A (ja) | 2022-03-04 | 2025-03-21 | ロックアネイビオ, インコーポレイテッド | 操作された核内低分子RNA(snRNA)を含む組成物および方法 |
| WO2023183825A2 (en) * | 2022-03-21 | 2023-09-28 | Ptc Therapeutics Gt, Inc. | Gene therapy of ush2a-associated diseases |
| GB202208387D0 (en) * | 2022-06-08 | 2022-07-20 | Ucl Business Ltd | Modified U7 snRNA construct |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| CN120112640A (zh) * | 2022-08-24 | 2025-06-06 | 塑造治疗公司 | 用于提高rna有效载荷转录的工程化构建体 |
| CN120129747A (zh) * | 2022-10-11 | 2025-06-10 | 广州瑞风生物科技有限公司 | snRNA核酸分子及其应用 |
| WO2024081411A1 (en) * | 2022-10-13 | 2024-04-18 | Shape Therapeutics Inc. | Engineered constructs for enhanced stability or localization of rna payloads |
| AU2023364059A1 (en) * | 2022-10-18 | 2025-05-08 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising programmable snrnas for rna editing |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| AR131145A1 (es) | 2022-11-24 | 2025-02-19 | Proqr Therapeutics Ii Bv | Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| CA3276262A1 (en) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| WO2024137991A2 (en) * | 2022-12-23 | 2024-06-27 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| JP2026504808A (ja) * | 2023-01-18 | 2026-02-10 | アプター バイオ,インク. | 神経変性疾患の処置のためのポリヌクレオチド組成物および方法 |
| EP4652278A1 (en) * | 2023-01-18 | 2025-11-26 | Aptah Bio, Inc. | Polynucleotide compositions and methods for treatment of cancer |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| EP4669753A1 (en) | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2024226860A2 (en) * | 2023-04-26 | 2024-10-31 | Shape Therapeutics Inc. | Small nuclear rna processing hairpins for small rna payloads |
| EP4713450A1 (en) * | 2023-05-15 | 2026-03-25 | Shape Therapeutics Inc. | Engineered constructs with cassette arrangements for increased transcription of rna payloads |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| WO2025007937A1 (zh) * | 2023-07-04 | 2025-01-09 | 广州瑞风生物科技有限公司 | 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用 |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025080844A1 (en) * | 2023-10-11 | 2025-04-17 | Emugen Therapeutics Llc | Methods and compositions for altering mecp2 expression |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025126158A2 (en) * | 2023-12-15 | 2025-06-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) targeting sod1 |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| WO2025190385A1 (zh) * | 2024-03-14 | 2025-09-18 | 上海拓界生物医药科技有限公司 | 靶向LRRK2的RNAi剂及其医药用途 |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025211949A1 (ko) * | 2024-04-05 | 2025-10-09 | 재단법인대구경북과학기술원 | SerpinA1e 단백질을 유효성분으로 포함하는 기억력, 인지기능 개선용 또는 뇌신경질환의 예방 또는 치료용 조성물 |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
| GB202410081D0 (en) | 2024-07-11 | 2024-08-28 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of cardiovascular disease |
| WO2026022136A1 (en) | 2024-07-23 | 2026-01-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2026060374A2 (en) | 2024-09-16 | 2026-03-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030036519A1 (en) * | 1998-04-21 | 2003-02-20 | Ryszard Kole | Stable alteration of pre-mrna splicing patterns by modified rnas |
| CA2957661A1 (en) * | 2014-08-09 | 2016-02-18 | Kevin FLANIGAN | Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene |
| US10676737B2 (en) * | 2014-12-17 | 2020-06-09 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| DE102015012522B3 (de) * | 2015-09-26 | 2016-06-02 | Eberhard Karls Universität Tübingen | Verfahren und Substanzen zur gerichteten RNA-Editierung |
| WO2020001793A1 (en) * | 2018-06-29 | 2020-01-02 | Eberhard-Karls-Universität Tübingen | Artificial nucleic acids for rna editing |
| AU2019336793B2 (en) * | 2018-09-06 | 2024-11-21 | The Regents Of The University Of California | RNA and DNA base editing via engineered ADAR recruitment |
| EP4306116A3 (en) * | 2018-10-12 | 2024-04-17 | Peking University | Methods and compositions for editing rnas |
| EP4008784A1 (en) * | 2019-08-01 | 2022-06-08 | Astellas Pharma Inc. | Guide rna for targeted-editing with functional base sequence added thereto |
| WO2021113270A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| MX2022006991A (es) * | 2019-12-09 | 2022-08-25 | Astellas Pharma Inc | Arn guía antisentido con región funcional añadida para editar arn blanco. |
-
2021
- 2021-04-22 CN CN202180042856.3A patent/CN115777020A/zh active Pending
- 2021-04-22 AU AU2021261374A patent/AU2021261374A1/en active Pending
- 2021-04-22 JP JP2022564250A patent/JP2023523237A/ja active Pending
- 2021-04-22 EP EP21726256.7A patent/EP4139453A1/en active Pending
- 2021-04-22 CA CA3173012A patent/CA3173012A1/en active Pending
- 2021-04-22 WO PCT/US2021/028618 patent/WO2021216853A1/en not_active Ceased
- 2021-04-22 US US17/920,065 patent/US20240011028A9/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4139453A1 (en) | 2023-03-01 |
| AU2021261374A1 (en) | 2022-10-13 |
| WO2021216853A1 (en) | 2021-10-28 |
| JP2023523237A (ja) | 2023-06-02 |
| US20240011028A9 (en) | 2024-01-11 |
| US20230203483A1 (en) | 2023-06-29 |
| CN115777020A (zh) | 2023-03-10 |
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