WO2006086667A2 - Antisense composition and method for treating muscle atrophy - Google Patents
Antisense composition and method for treating muscle atrophy Download PDFInfo
- Publication number
- WO2006086667A2 WO2006086667A2 PCT/US2006/004797 US2006004797W WO2006086667A2 WO 2006086667 A2 WO2006086667 A2 WO 2006086667A2 US 2006004797 W US2006004797 W US 2006004797W WO 2006086667 A2 WO2006086667 A2 WO 2006086667A2
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- myostatin
- transcript
- effective
- compound
- antisense
- Prior art date
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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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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- 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
- A61K31/66—Phosphorus compounds
- A61K31/675—Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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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
- 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
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
- Y10T—TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
- Y10T436/00—Chemistry: analytical and immunological testing
- Y10T436/14—Heterocyclic carbon compound [i.e., O, S, N, Se, Te, as only ring hetero atom]
- Y10T436/142222—Hetero-O [e.g., ascorbic acid, etc.]
- Y10T436/143333—Saccharide [e.g., DNA, etc.]
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- Chemical & Material Sciences (AREA)
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- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Analytical Chemistry (AREA)
- Endocrinology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Physical Education & Sports Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
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Priority Applications (11)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
AU2006213686A AU2006213686A1 (en) | 2005-02-09 | 2006-02-09 | Antisense composition and method for treating muscle atrophy |
EP06734779.9A EP1855694B1 (en) | 2005-02-09 | 2006-02-09 | Antisense composition for treating muscle atrophy |
CA2596506A CA2596506C (en) | 2005-02-09 | 2006-02-09 | Antisense composition and method for treating muscle atrophy |
ES06734779T ES2852549T3 (en) | 2005-02-09 | 2006-02-09 | Antisense composition for treatment of muscle atrophy |
PCT/US2006/004797 WO2006086667A2 (en) | 2005-02-09 | 2006-02-09 | Antisense composition and method for treating muscle atrophy |
US11/433,724 US7888012B2 (en) | 2005-02-09 | 2006-05-11 | Antisense composition and method for treating muscle atrophy |
US12/983,798 US8785410B2 (en) | 2005-02-09 | 2011-01-03 | Antisense composition and method for treating muscle atrophy |
US14/323,349 US20150119316A1 (en) | 2005-02-09 | 2014-07-03 | Antisense composition and method for treating muscle atrophy |
US15/177,244 US10006031B2 (en) | 2005-02-09 | 2016-06-08 | Antisense composition and method for treating muscle atrophy |
US15/986,746 US10626396B2 (en) | 2005-02-09 | 2018-05-22 | Antisense composition and method for treating muscle atrophy |
US16/814,861 US20210139911A1 (en) | 2005-02-09 | 2020-03-10 | Antisense composition and method for treating muscle atrophy |
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US65157405P | 2005-02-09 | 2005-02-09 | |
US60/651,574 | 2005-02-09 | ||
PCT/US2006/004797 WO2006086667A2 (en) | 2005-02-09 | 2006-02-09 | Antisense composition and method for treating muscle atrophy |
Related Child Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US11/433,724 Continuation-In-Part US7888012B2 (en) | 2005-02-09 | 2006-05-11 | Antisense composition and method for treating muscle atrophy |
US11/433,724 Continuation US7888012B2 (en) | 2005-02-09 | 2006-05-11 | Antisense composition and method for treating muscle atrophy |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2006086667A2 true WO2006086667A2 (en) | 2006-08-17 |
WO2006086667A3 WO2006086667A3 (en) | 2007-02-22 |
Family
ID=36793777
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2006/004797 WO2006086667A2 (en) | 2005-02-09 | 2006-02-09 | Antisense composition and method for treating muscle atrophy |
Country Status (6)
Country | Link |
---|---|
US (6) | US7888012B2 (en) |
EP (1) | EP1855694B1 (en) |
AU (1) | AU2006213686A1 (en) |
CA (1) | CA2596506C (en) |
ES (1) | ES2852549T3 (en) |
WO (1) | WO2006086667A2 (en) |
Cited By (24)
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EP2124968A1 (en) * | 2006-06-30 | 2009-12-02 | Oligos Etc. Inc. | Compositions and methods for the treatment of muscle wasting |
GB2441581B (en) * | 2005-06-24 | 2011-01-19 | Roderic M K Dale | Compositions and methods for the treatment of muscle wasting |
US7989608B2 (en) | 2007-12-28 | 2011-08-02 | Avi Biopharma Inc. | Immunomodulatory agents and methods of use |
US8067569B2 (en) | 2000-05-04 | 2011-11-29 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
WO2011150408A2 (en) | 2010-05-28 | 2011-12-01 | Avi Biopharma, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
US8097596B2 (en) | 2006-06-30 | 2012-01-17 | Lakewood-Amedex, Inc. | Compositions and methods for the treatment of muscle wasting |
WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
WO2013142087A1 (en) | 2012-03-20 | 2013-09-26 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
US8741863B2 (en) | 2007-06-29 | 2014-06-03 | Sarepta Therapeutics, Inc. | Compound and method for treating myotonic dystrophy |
US8785410B2 (en) | 2005-02-09 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense composition and method for treating muscle atrophy |
US8877725B2 (en) | 2004-05-24 | 2014-11-04 | Sarepta Therapeutics, Inc. | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
US9249243B2 (en) | 2005-07-13 | 2016-02-02 | Sarepta Therapeutics, Inc. | Antibacterial antisense oligonucleotide and method |
US9278987B2 (en) | 2011-11-18 | 2016-03-08 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
US9572899B2 (en) | 2003-04-29 | 2017-02-21 | Avi Biopharma, Inc. | Compositions for enhancing transport of molecules into cells |
WO2017047741A1 (en) * | 2015-09-16 | 2017-03-23 | 日本新薬株式会社 | Antisense nucleic acid for treating amyotrophy |
AU2015200600B2 (en) * | 2007-06-29 | 2017-04-20 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
JP2018518148A (en) * | 2015-03-18 | 2018-07-12 | サレプタ セラピューティクス,インコーポレイテッド | Elimination of antisense-induced exons in myostatin |
WO2018162931A1 (en) * | 2017-03-10 | 2018-09-13 | Ucl Business Plc | Method relating to myostatin pathway inhibition |
US10106796B2 (en) | 2009-06-26 | 2018-10-23 | Sarepta Therapeutics, Inc. | Compound and method for treating myotonic dystrophy |
US10106795B2 (en) | 2011-10-04 | 2018-10-23 | Royal Holloway And Bedford New College | Oligomers |
US10888578B2 (en) | 2016-12-19 | 2021-01-12 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
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US20050222068A1 (en) * | 2003-10-23 | 2005-10-06 | Mourich Dan V | Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells |
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US7807816B2 (en) | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
ES2694726T3 (en) * | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
EP2350281B1 (en) | 2008-10-24 | 2014-05-14 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
CA2746508A1 (en) * | 2008-12-17 | 2010-07-15 | Avi Biopharma, Inc. | Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis |
HUE040445T2 (en) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antisense molecules and methods for treating pathologies |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
JP6336755B2 (en) | 2010-11-12 | 2018-06-06 | ザ ジェネラル ホスピタル コーポレイション | Non-coding RNA related to polycomb |
EP2750715B1 (en) | 2011-08-30 | 2018-10-31 | The Regents of The University of California | Identification of small molecules that enhance therapeutic exon skipping |
WO2013040429A1 (en) | 2011-09-14 | 2013-03-21 | Rana Therapeutics Inc. | Multimeric oligonucleotide compounds |
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JP2015523854A (en) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Compositions and methods for modulating SMN gene family expression |
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WO2006086667A3 (en) | 2007-02-22 |
US8785410B2 (en) | 2014-07-22 |
CA2596506A1 (en) | 2006-08-17 |
US7888012B2 (en) | 2011-02-15 |
US20110166082A1 (en) | 2011-07-07 |
US20210139911A1 (en) | 2021-05-13 |
US20070122821A1 (en) | 2007-05-31 |
EP1855694A4 (en) | 2010-10-27 |
US20160281092A1 (en) | 2016-09-29 |
ES2852549T3 (en) | 2021-09-13 |
AU2006213686A1 (en) | 2006-08-17 |
US10006031B2 (en) | 2018-06-26 |
US20180327749A1 (en) | 2018-11-15 |
EP1855694B1 (en) | 2020-12-02 |
US10626396B2 (en) | 2020-04-21 |
CA2596506C (en) | 2021-04-06 |
US20150119316A1 (en) | 2015-04-30 |
EP1855694A2 (en) | 2007-11-21 |
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