CA2659103C - Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups - Google Patents
Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups Download PDFInfo
- Publication number
- CA2659103C CA2659103C CA2659103A CA2659103A CA2659103C CA 2659103 C CA2659103 C CA 2659103C CA 2659103 A CA2659103 A CA 2659103A CA 2659103 A CA2659103 A CA 2659103A CA 2659103 C CA2659103 C CA 2659103C
- Authority
- CA
- Canada
- Prior art keywords
- substituted
- alkyl
- alkenyl
- aryl
- heterocyclic
- 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.)
- Expired - Fee Related
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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US83057206P | 2006-07-12 | 2006-07-12 | |
| US60/830,572 | 2006-07-12 | ||
| PCT/US2007/015966 WO2008008476A2 (en) | 2006-07-12 | 2007-07-11 | Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2659103A1 CA2659103A1 (en) | 2008-01-17 |
| CA2659103C true CA2659103C (en) | 2019-05-21 |
Family
ID=38923923
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2659103A Expired - Fee Related CA2659103C (en) | 2006-07-12 | 2007-07-11 | Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20090093425A1 (enExample) |
| EP (1) | EP2046964B1 (enExample) |
| JP (2) | JP6125741B2 (enExample) |
| CN (1) | CN101506368B (enExample) |
| AU (1) | AU2007272906B2 (enExample) |
| CA (1) | CA2659103C (enExample) |
| WO (1) | WO2008008476A2 (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2638915A1 (en) | 2006-02-10 | 2007-08-23 | The Regents Of The University Of California | Transducible delivery of sirna by dsrna binding domain fusions to ptd/cpps |
| US10022454B2 (en) | 2008-09-23 | 2018-07-17 | Liposciences, Llc | Functionalized phosphorodiamites for therapeutic oligonucleotide synthesis |
| EP2346883B1 (en) * | 2008-09-23 | 2016-03-23 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference |
| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| WO2010129853A2 (en) * | 2009-05-07 | 2010-11-11 | The Regents Of The University Of California | TRANSDUCIBLE DELIVERY OF NUCLEIC ACIDS USING MODIFIED dsRNA BINDING DOMAINS |
| EP2438079A4 (en) * | 2009-06-01 | 2013-05-22 | Univ California | NUCLEIC ACID DELIVERY COMPOSITIONS AND METHOD OF USE THEREOF |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| EP3252068B1 (en) | 2009-10-12 | 2025-07-02 | Larry J. Smith | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
| WO2011109677A2 (en) * | 2010-03-04 | 2011-09-09 | University Of Louisville Research Foundation, Inc. | Methods of increasing macropinocytosis in cancer cells |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| WO2012138846A2 (en) * | 2011-04-05 | 2012-10-11 | The Regents Of The University Of California | Method and compositions comprising small rna agonist and antagonists to modulate inflammation |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| JP5435016B2 (ja) | 2011-12-01 | 2014-03-05 | 株式会社デンソー | 半導体装置の製造方法 |
| EP3919620A1 (en) | 2012-05-02 | 2021-12-08 | Sirna Therapeutics, Inc. | Short interfering nucleic acid (sina) compositions |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| CA2880869A1 (en) | 2012-08-20 | 2014-02-27 | The Regents Of The University Of California | Polynucleotides having bioreversible groups |
| CA2880290C (en) | 2012-09-12 | 2020-10-27 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules targeting p53 and methods of use thereof |
| ES2704855T3 (es) | 2012-09-12 | 2019-03-20 | Quark Pharmaceuticals Inc | Moléculas de oligonucleótido de doble cadena para p53 y métodos de uso de las mismas |
| AU2013356386B2 (en) * | 2012-12-06 | 2017-09-07 | Merck Sharp & Dohme Corp. | Nucleoside kinase bypass compositions and methods |
| EP2928877B1 (en) * | 2012-12-06 | 2020-01-22 | Merck Sharp & Dohme Corp. | Disulfide masked prodrug compositions and methods |
| CA2907184C (en) | 2013-03-15 | 2022-12-06 | Sutter West Bay Hospitals | Falz for use as a target for therapies to treat cancer |
| US20160208247A1 (en) | 2013-07-31 | 2016-07-21 | Qbi Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| EP3027222A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| CA2922698C (en) * | 2013-08-29 | 2023-01-03 | City Of Hope | Cell penetrating conjugates comprising non-cell penetrating antibodies covalently attached to one or more phosphorothioate nucleic acids |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| GB201410507D0 (en) * | 2014-06-12 | 2014-07-30 | Univ Bath | Drug delivery enhancement agents |
| KR20190058477A (ko) | 2016-08-17 | 2019-05-29 | 솔스티스 바이올로직스, 리미티드 | 폴리뉴클레오티드 구축물 |
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| JP7282379B2 (ja) * | 2017-08-22 | 2023-05-29 | 国立大学法人東海国立大学機構 | 修飾ポリヌクレオチド |
| US11510939B1 (en) | 2019-04-19 | 2022-11-29 | Apellis Pharmaceuticals, Inc. | RNAs for complement inhibition |
Family Cites Families (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4806463A (en) * | 1986-05-23 | 1989-02-21 | Worcester Foundation For Experimental Biology | Inhibition of HTLV-III by exogenous oligonucleotides |
| US6316003B1 (en) * | 1989-12-21 | 2001-11-13 | Whitehead Institute For Biomedical Research | Tat-derived transport polypeptides |
| US5674980A (en) * | 1989-12-21 | 1997-10-07 | Biogen Inc | Fusion protein comprising tat-derived transport moiety |
| WO1991014696A1 (en) * | 1990-03-29 | 1991-10-03 | Gilead Sciences, Inc. | Oligonucleotide-transport agent disulfide conjugates |
| US5733523A (en) * | 1990-12-10 | 1998-03-31 | Akzo Nobel N.V. | Targeted delivery of a therapeutic entity using complementary oligonucleotides |
| NZ244306A (en) * | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
| US5955591A (en) * | 1993-05-12 | 1999-09-21 | Imbach; Jean-Louis | Phosphotriester oligonucleotides, amidites and method of preparation |
| US6051429A (en) * | 1995-06-07 | 2000-04-18 | Life Technologies, Inc. | Peptide-enhanced cationic lipid transfections |
| US6017700A (en) * | 1995-08-04 | 2000-01-25 | Bayer Corporation | Cationic oligonucleotides, and related methods of synthesis and use |
| US6251433B1 (en) * | 1996-08-13 | 2001-06-26 | Chiron Corporation | Polycationic polymers |
| WO1999029721A1 (en) * | 1997-12-10 | 1999-06-17 | Washington University | Anti-pathogen system and methods of use thereof |
| US6531590B1 (en) * | 1998-04-24 | 2003-03-11 | Isis Pharmaceuticals, Inc. | Processes for the synthesis of oligonucleotide compounds |
| US6903077B1 (en) * | 1999-01-04 | 2005-06-07 | University Of Vermont And State Agricultural College | Methods and products for delivering nucleic acids |
| US20070026394A1 (en) * | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| US7491805B2 (en) * | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US6841535B2 (en) * | 2000-07-31 | 2005-01-11 | Active Motif | Peptide-mediated transfection agents and methods of use |
| EP1345956A2 (en) * | 2000-10-13 | 2003-09-24 | University of Lausanne | Intracellular delivery of biological effectors by novel transporter peptide sequences |
| US20040204377A1 (en) * | 2002-11-26 | 2004-10-14 | University Of Massachusetts | Delivery of siRNAs |
| US20060178297A1 (en) * | 2003-01-28 | 2006-08-10 | Troy Carol M | Systems and methods for silencing expression of a gene in a cell and uses thereof |
| US20040147027A1 (en) * | 2003-01-28 | 2004-07-29 | Troy Carol M. | Complex for facilitating delivery of dsRNA into a cell and uses thereof |
| US7166692B2 (en) * | 2003-03-04 | 2007-01-23 | Canbrex Bio Science Walkersville, Inc. | Intracellular delivery of small molecules, proteins, and nucleic acids |
| EP1631676A4 (en) * | 2003-06-10 | 2006-12-20 | Toolgen Inc | TRANSDUCIBLE DNA BINDING PROTEINS |
| AU2004316996A1 (en) * | 2003-06-20 | 2005-09-15 | The Regents Of The University Of California | Polypeptide transduction and fusogenic peptides |
| WO2005003315A2 (en) * | 2003-07-01 | 2005-01-13 | Allele Biotechnology & Pharmaceuticals, Inc. | Compositions and methods for peptide-assisted transfection |
| JP4838722B2 (ja) * | 2003-10-24 | 2011-12-14 | ゲンシア コーポレーション | ポリヌクレオチドを送達する方法、及び送達用組成物 |
| US7514530B2 (en) * | 2004-04-26 | 2009-04-07 | Centre National De La Recherche Scientifique | Peptide carrier for delivering siRNA into mammalian cells |
| CA2565685A1 (en) * | 2004-05-04 | 2005-12-15 | Nastech Pharmaceutical Company Inc. | Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells |
| US20060040882A1 (en) * | 2004-05-04 | 2006-02-23 | Lishan Chen | Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells |
| US20060035815A1 (en) * | 2004-05-04 | 2006-02-16 | Nastech Pharmaceutical Company Inc. | Pharmaceutical compositions for delivery of ribonucleic acid to a cell |
| US20060030003A1 (en) * | 2004-05-12 | 2006-02-09 | Simon Michael R | Composition and method for introduction of RNA interference sequences into targeted cells and tissues |
| US20050288246A1 (en) * | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| US7084248B2 (en) * | 2004-07-14 | 2006-08-01 | Gene Tools, Llc | Peptide composition and method for delivering substances into the cytosol of cells |
| CA2574200A1 (en) * | 2004-07-21 | 2006-01-26 | Xiao Huang | Hybrid wicking materials for use in high performance heat pipes |
| EP1789447B1 (en) * | 2004-08-16 | 2012-04-25 | Immune Disease Institute, Inc. | Method of delivering rna interference and uses thereof |
| US20080027025A1 (en) * | 2004-08-27 | 2008-01-31 | The Regents Of The University Of California | Protection From And Treatment Of Prion Protein Infection |
| US20090098049A1 (en) * | 2004-09-07 | 2009-04-16 | The Regents Of The University Of California | Targeting transducible molecules to specific cell types |
| CA2638915A1 (en) * | 2006-02-10 | 2007-08-23 | The Regents Of The University Of California | Transducible delivery of sirna by dsrna binding domain fusions to ptd/cpps |
| CN101421301A (zh) * | 2006-02-10 | 2009-04-29 | 加利福尼亚大学董事会 | 通过dsRNA结合域与PTD/CPPS的融合物转导运输siRNA |
| EP2438079A4 (en) * | 2009-06-01 | 2013-05-22 | Univ California | NUCLEIC ACID DELIVERY COMPOSITIONS AND METHOD OF USE THEREOF |
-
2007
- 2007-07-11 WO PCT/US2007/015966 patent/WO2008008476A2/en not_active Ceased
- 2007-07-11 US US11/776,317 patent/US20090093425A1/en not_active Abandoned
- 2007-07-11 CN CN200780030587.9A patent/CN101506368B/zh not_active Expired - Fee Related
- 2007-07-11 EP EP07796847.7A patent/EP2046964B1/en not_active Not-in-force
- 2007-07-11 CA CA2659103A patent/CA2659103C/en not_active Expired - Fee Related
- 2007-07-11 JP JP2009519541A patent/JP6125741B2/ja active Active
- 2007-07-11 AU AU2007272906A patent/AU2007272906B2/en not_active Ceased
-
2014
- 2014-10-24 JP JP2014217097A patent/JP2015062421A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2007272906A1 (en) | 2008-01-17 |
| JP2009543783A (ja) | 2009-12-10 |
| JP2015062421A (ja) | 2015-04-09 |
| AU2007272906B2 (en) | 2013-01-31 |
| CN101506368A (zh) | 2009-08-12 |
| JP6125741B2 (ja) | 2017-05-17 |
| EP2046964A4 (en) | 2014-05-14 |
| CA2659103A1 (en) | 2008-01-17 |
| WO2008008476A3 (en) | 2008-07-24 |
| EP2046964A2 (en) | 2009-04-15 |
| WO2008008476A2 (en) | 2008-01-17 |
| EP2046964B1 (en) | 2018-07-04 |
| US20090093425A1 (en) | 2009-04-09 |
| CN101506368B (zh) | 2017-02-08 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| MKLA | Lapsed |
Effective date: 20200831 |