DK3119797T3 - Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose - Google Patents
Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose Download PDFInfo
- Publication number
- DK3119797T3 DK3119797T3 DK15764861.9T DK15764861T DK3119797T3 DK 3119797 T3 DK3119797 T3 DK 3119797T3 DK 15764861 T DK15764861 T DK 15764861T DK 3119797 T3 DK3119797 T3 DK 3119797T3
- Authority
- DK
- Denmark
- Prior art keywords
- raav
- treatment
- methods
- lateral sclerosis
- amyotrophic lateral
- Prior art date
Links
- CTYIYTSFKAYTGY-UHFFFAOYSA-N C1C(C2)CCC3=C2C13 Chemical compound C1C(C2)CCC3=C2C13 CTYIYTSFKAYTGY-UHFFFAOYSA-N 0.000 description 1
- MMUZTSAHJFINEX-UHFFFAOYSA-N CC1C(C)(C2)CC2C1 Chemical compound CC1C(C)(C2)CC2C1 MMUZTSAHJFINEX-UHFFFAOYSA-N 0.000 description 1
- RIRARCHMRDHZAR-UHFFFAOYSA-N CC1C(C)CCC1 Chemical compound CC1C(C)CCC1 RIRARCHMRDHZAR-UHFFFAOYSA-N 0.000 description 1
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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
- C12N15/11—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
- C12N15/1137—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 enzymes
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
-
- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461955189P | 2014-03-18 | 2014-03-18 | |
PCT/US2015/021321 WO2015143078A1 (en) | 2014-03-18 | 2015-03-18 | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
Publications (1)
Publication Number | Publication Date |
---|---|
DK3119797T3 true DK3119797T3 (da) | 2021-03-15 |
Family
ID=54145287
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK15764861.9T DK3119797T3 (da) | 2014-03-18 | 2015-03-18 | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose |
Country Status (8)
Country | Link |
---|---|
US (6) | US10280418B2 (da) |
EP (2) | EP3119797B1 (da) |
JP (4) | JP6756700B2 (da) |
AU (3) | AU2015231294B2 (da) |
CA (1) | CA2942515A1 (da) |
DK (1) | DK3119797T3 (da) |
ES (1) | ES2856403T3 (da) |
WO (1) | WO2015143078A1 (da) |
Families Citing this family (59)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
SG177564A1 (en) | 2009-07-06 | 2012-02-28 | Ontorii Inc | Novel nucleic acid prodrugs and methods of use thereof |
CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のaavベースの治療 |
WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
AU2015207773B2 (en) | 2014-01-16 | 2021-06-17 | Wave Life Sciences Ltd. | Chiral design |
US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
AU2015231294B2 (en) | 2014-03-18 | 2020-10-29 | University Of Massachusetts | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
JP6619822B2 (ja) | 2015-05-29 | 2019-12-11 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | C9orf72遺伝子座における破壊を有する非ヒト動物 |
EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
EP4094780A3 (en) | 2016-02-12 | 2023-02-08 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
WO2017165490A1 (en) * | 2016-03-22 | 2017-09-28 | President And Fellows Of Harvard College | Biocompatible adhesives and methods of use thereof |
EP4008356A1 (en) * | 2016-03-31 | 2022-06-08 | University of Cincinnati | Methods and compositions for the treatment of als |
CA3019832C (en) | 2016-04-02 | 2023-05-09 | Research Institute At Nationwide Children's Hospital | Modified u6 promoter system for tissue specific expression |
EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN |
WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
WO2017214471A1 (en) * | 2016-06-10 | 2017-12-14 | Saunders, Ann M. | Methods for detecting structural variants in neurodegenerative disease |
WO2017218852A1 (en) | 2016-06-15 | 2017-12-21 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
SG10202102997UA (en) * | 2016-09-30 | 2021-04-29 | Regeneron Pharma | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
AU2017361521B2 (en) | 2016-11-21 | 2020-08-27 | Nanostring Technologies, Inc. | Chemical compositions and methods of using same |
CA3061365A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
US20210108199A1 (en) * | 2017-09-06 | 2021-04-15 | The Trustees Of Columbia University In The City Of New York | Treatment for aggressive cancers by targeting C9ORF72 |
EP3684937A4 (en) * | 2017-09-22 | 2021-06-02 | University of Massachusetts | DUAL EXPRESSION VECTORS OF SOD1 AND THEIR USES |
AU2018352236A1 (en) | 2017-10-16 | 2020-04-23 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
CN111819281A (zh) * | 2017-10-23 | 2020-10-23 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
EP3773606A4 (en) * | 2018-04-03 | 2021-12-08 | Academia Sinica | MIR-17 ~ 92 AS A THERAPEUTIC OR DIAGNOSTIC TARGET OF MOTOR NEURON (MN) DEGENERATIONAL DISEASES |
SG11202011274YA (en) | 2018-05-14 | 2020-12-30 | Nanostring Technologies Inc | Chemical compositions and methods of using same |
EP3850098A1 (en) * | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
KR20210105914A (ko) | 2018-12-20 | 2021-08-27 | 리제너론 파마슈티칼스 인코포레이티드 | 뉴클레아제-매개 반복부 팽창 |
EP4037695A4 (en) * | 2019-10-06 | 2024-03-27 | Wave Life Sciences Ltd | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF |
WO2021159008A2 (en) | 2020-02-07 | 2021-08-12 | Maze Therapeutics, Inc. | Compositions and methods for treating neurodegenerative diseases |
AR123524A1 (es) * | 2020-09-16 | 2022-12-14 | Biogen Ma Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1 |
WO2022174000A2 (en) | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
CA3213590A1 (en) | 2021-04-06 | 2022-10-13 | Maze Therapeutics, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
CA3234939A1 (en) * | 2021-10-18 | 2023-04-27 | Michael Goren | Eukaryotic cells comprising adenovirus-associated virus polynucleotides |
WO2024077109A1 (en) | 2022-10-05 | 2024-04-11 | Maze Therapeutics, Inc. | Unc13a antisense oligonucleotides and uses thereof |
Family Cites Families (159)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
US5797898A (en) | 1996-07-02 | 1998-08-25 | Massachusetts Institute Of Technology | Microchip drug delivery devices |
US5783208A (en) | 1996-07-19 | 1998-07-21 | Theratech, Inc. | Transdermal drug delivery matrix for coadministering estradiol and another steroid |
US5779708A (en) | 1996-08-15 | 1998-07-14 | Cyberdent, Inc. | Intraosseous drug delivery device and method |
US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
US6544523B1 (en) | 1996-11-13 | 2003-04-08 | Chiron Corporation | Mutant forms of Fas ligand and uses thereof |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
US20030110526A1 (en) | 1998-08-25 | 2003-06-12 | Robert H. Brown | Dysferlin mutations |
JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
US6544786B1 (en) | 1999-10-15 | 2003-04-08 | University Of Pittsburgh Of The Commonwealth Of Higher Education | Method and vector for producing and transferring trans-spliced peptides |
US7638120B2 (en) | 2000-03-14 | 2009-12-29 | Thomas Jefferson University | High transgene expression of a pseudotyped adeno-associated virus type |
US8202846B2 (en) | 2000-03-16 | 2012-06-19 | Cold Spring Harbor Laboratory | Methods and compositions for RNA interference |
WO2001075164A2 (en) | 2000-03-30 | 2001-10-11 | Whitehead Institute For Biomedical Research | Rna sequence-specific mediators of rna interference |
US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
US20050019915A1 (en) | 2001-06-21 | 2005-01-27 | Bennett C. Frank | Antisense modulation of superoxide dismutase 1, soluble expression |
CA2790034A1 (en) | 2001-06-21 | 2003-01-03 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
CA2452980A1 (en) | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
CA2921821A1 (en) | 2001-07-12 | 2003-01-23 | University Of Massachusetts | In vivo production of small interfering rnas that mediate gene silencing |
WO2003006616A2 (en) | 2001-07-13 | 2003-01-23 | University Of Iowa Research Foundation | Pseudotyped adeno-associated viruses and uses thereof |
ES2258601T3 (es) | 2001-11-13 | 2006-09-01 | The Trustees Of The University Of Pennsylvania | Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo. |
EP2573170B1 (en) | 2001-12-17 | 2017-12-20 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor |
US20050137153A1 (en) | 2002-02-20 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA) |
AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
US20070003518A1 (en) | 2002-04-19 | 2007-01-04 | Atkinson Mark A | Raav vector-based compositions and methods for the prevention and treatment of mammalian diseases |
US7235393B2 (en) | 2002-04-29 | 2007-06-26 | The Trustees Of The University Of Pennsylvania | Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues |
US20060093589A1 (en) | 2004-02-19 | 2006-05-04 | Warrington Kenneth H | Vp2-modified raav vector compositions and uses therefor |
US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
AU2003261449A1 (en) | 2002-08-07 | 2004-02-25 | Compositions for rna interference and methods of use thereof | |
US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
JP2006507841A (ja) | 2002-11-14 | 2006-03-09 | ダーマコン, インコーポレイテッド | 機能的siRNAおよび超機能的siRNA |
US20060206949A1 (en) | 2003-01-28 | 2006-09-14 | Sylvain Arnould | Custom-made meganuclease and use thereof |
US20040219528A1 (en) | 2003-04-15 | 2004-11-04 | Morris David W. | Novel therapeutic targets in cancer |
US7387896B2 (en) | 2003-03-26 | 2008-06-17 | The Regents Of The University Of Michigan | MicroRNA vectors |
US20060228800A1 (en) | 2003-05-15 | 2006-10-12 | Shi-Lung Lin | Novel Transgenic Methods Using intronic RNA |
US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
EP3502252B1 (en) | 2003-06-02 | 2023-04-05 | University of Massachusetts | Methods and compositions for controlling efficacy of rna silencing |
CA2533701A1 (en) | 2003-07-31 | 2005-02-17 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and compositions for use in modulation of small non-coding rnas |
CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
US20060134787A1 (en) | 2004-12-22 | 2006-06-22 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
WO2005062937A2 (en) | 2003-12-22 | 2005-07-14 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna |
JP4763681B2 (ja) | 2004-03-05 | 2011-08-31 | ベニテック インコーポレイテッド | RNAi作用媒介物の同時デリバリーのためのマルチプロモーター発現カセット |
WO2005096781A2 (en) | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
CA2566286A1 (en) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
WO2005116224A2 (en) | 2004-05-18 | 2005-12-08 | Children's Memorial Hospital | Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system |
US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
CN101124328A (zh) | 2004-12-15 | 2008-02-13 | 北卡罗来纳查佩尔山大学 | 嵌合载体 |
WO2006066203A2 (en) * | 2004-12-16 | 2006-06-22 | Alsgen, Llc | Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod) |
ATE524546T1 (de) | 2005-03-25 | 2011-09-15 | Medtronic Inc | Verwendung von anti-tnf oder anti-il1-rnai zur unterdrückung der wirkung entzündungsfördernder cytokine zur lokalen schmerzbehandlung |
EP2359865B1 (en) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
EP3002330A1 (en) | 2005-05-27 | 2016-04-06 | Ospedale San Raffaele S.r.l. | Gene vector |
US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
JP5523705B2 (ja) | 2005-08-29 | 2014-06-18 | レグルス・セラピューティクス・インコーポレイテッド | Mir−122aをモジュレートする使用方法 |
EP1979485A2 (en) | 2006-01-31 | 2008-10-15 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
EP2514824A3 (en) | 2006-03-03 | 2013-01-23 | Amorfix Life Sciences Ltd. | Methods and compositions to treat and detect misfolded-SOD1 mediated diseases |
EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
WO2008118212A2 (en) | 2006-11-08 | 2008-10-02 | Veritas, Llc | In vivo delivery of double stranded rna to a target cell |
CA2683469C (en) | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
WO2008147839A1 (en) | 2007-05-23 | 2008-12-04 | Dharmacon, Inc. | Micro-rna scaffolds and non-naturally occurring micro-rnas |
EP2530152B1 (en) | 2007-05-31 | 2017-12-27 | University of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
WO2009079592A2 (en) | 2007-12-17 | 2009-06-25 | California Institute Of Technology | Modulating immune system development and function through microrna mir-146 |
WO2009102427A2 (en) * | 2008-02-11 | 2009-08-20 | Rxi Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
WO2009108274A2 (en) | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
CA2717792A1 (en) | 2008-03-07 | 2009-09-11 | Santaris Pharma A/S | Pharmaceutical compositions for treatment of microrna related diseases |
WO2009146178A1 (en) | 2008-04-15 | 2009-12-03 | President And Fellows Of Harvard College | Angiogenin and amyotrophic lateral sclerosis |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
EP2297322A1 (en) | 2008-06-04 | 2011-03-23 | The Board of Regents of The University of Texas System | Modulation of gene expression through endogenous small rna targeting of gene promoters |
EP2297185A1 (en) | 2008-06-17 | 2011-03-23 | Amsterdam Molecular Therapeutics (AMT) B.V. | Parvoviral capsid with incorporated gly-ala repeat region |
US20110172293A1 (en) | 2008-07-08 | 2011-07-14 | Fish Jason E | Methods and Compositions for Modulating Angiogenesis |
KR20110073494A (ko) | 2008-09-04 | 2011-06-29 | 갈레네아 코포레이션 | 시냅스 소포 순환 검정 및 시스템 |
WO2010034314A1 (en) | 2008-09-24 | 2010-04-01 | Aarhus Universitet | Retroviral delivery of synthectic gene cassettes |
WO2010071454A1 (en) | 2008-12-17 | 2010-06-24 | Auckland Uniservices Limited | Adeno-associated viral vectors and uses thereof |
US20110305772A1 (en) | 2009-02-26 | 2011-12-15 | The Johns Hopkins University | Compositions and methods for ex vivo hepatic nucleic acid delivery |
EP3421603B1 (en) | 2009-05-02 | 2021-10-06 | Genzyme Corporation | Gene therapy for neurodegenerative disorders |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
US20120270930A1 (en) | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
CA2779437A1 (en) | 2009-11-09 | 2011-05-12 | Genepod Therapeutics Ab | Novel viral vector construct for neuron specific continuous dopa synthesis in vivo |
WO2011062298A1 (ja) | 2009-11-19 | 2011-05-26 | 国立大学法人岡山大学 | 遺伝子発現を上昇させるシステム及び該システムを保持したベクター |
EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
ES2680915T3 (es) | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
AU2011223820B2 (en) | 2010-03-01 | 2016-01-14 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the HCV genome |
CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のaavベースの治療 |
WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
US8906607B2 (en) * | 2010-04-30 | 2014-12-09 | Cellectis | Method for modulating double-strand break-induced homologous recombination |
US8785413B2 (en) | 2010-09-27 | 2014-07-22 | Florida Atlantic University Research Corporation | Materials and methods for the treatment of pathological neovascularization in the eye |
US9181544B2 (en) | 2011-02-12 | 2015-11-10 | University Of Iowa Research Foundation | Therapeutic compounds |
WO2012142434A2 (en) | 2011-04-15 | 2012-10-18 | THE UNITED STATES OF AMERICA, as represented by the secretary, Depart.of Healtth and Human Services | Aav mediated ctla-4 gene transfer to treat sjogren's syndrome |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
US8609088B2 (en) | 2011-05-10 | 2013-12-17 | Regents Of The University Of Minnesota | Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases |
EP2751284B1 (en) | 2011-08-31 | 2017-01-11 | The University Of Manchester | Method for diagnosing a neurodegenerative disease. |
US20140255936A1 (en) * | 2011-09-09 | 2014-09-11 | Mayo Foundation For Medical Education And Research | Detecting frontotemporal dementia and amyotrophic lateral sclerosis |
AU2012322788B2 (en) * | 2011-10-11 | 2018-01-04 | The Brigham And Women's Hospital, Inc. | Micrornas in neurodegenerative disorders |
US20130142861A1 (en) | 2011-12-05 | 2013-06-06 | National Yang Ming University | Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis |
AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
EP2847337A4 (en) | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | PLASMIDS AND VIRAL VECTORS ASSOCIATED WITH ADENOVIRUS |
CN104704123A (zh) * | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
CA2888486A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
ES2883590T3 (es) | 2012-12-12 | 2021-12-09 | Broad Inst Inc | Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas |
CN105142396A (zh) | 2013-01-14 | 2015-12-09 | 重组股份有限公司 | 缺角家畜 |
WO2014114303A1 (en) | 2013-01-22 | 2014-07-31 | Deutsches Zentrum Für Neurodegenerative Erkrankungen | Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion |
AU2014244167A1 (en) | 2013-03-13 | 2015-10-08 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
AU2014227766B2 (en) | 2013-03-15 | 2018-10-04 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV vectors |
US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
US10006049B2 (en) | 2013-05-16 | 2018-06-26 | University Of Florida Research Foundation, Incorporated | Hairpin mRNA elements and methods for the regulation of protein translation |
KR20230136697A (ko) | 2013-06-05 | 2023-09-26 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
RU2716420C2 (ru) | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
US10155794B2 (en) | 2013-07-16 | 2018-12-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to CEP290 |
CA2919995A1 (en) | 2013-08-05 | 2015-02-12 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
ES2936220T3 (es) * | 2013-08-27 | 2023-03-15 | Res Inst Nationwide Childrens Hospital | Productos y métodos para el tratamiento de la esclerosis lateral amiotrófica |
SG11201602597YA (en) * | 2013-10-11 | 2016-05-30 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
JP6712948B2 (ja) | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法 |
GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
HUE054748T2 (hu) | 2014-03-17 | 2021-09-28 | Hospital For Sick Children | Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére |
AU2015231294B2 (en) | 2014-03-18 | 2020-10-29 | University Of Massachusetts | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
EP3137497B1 (en) | 2014-05-02 | 2021-04-07 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
CA2949437C (en) | 2014-05-20 | 2023-08-15 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
MX2016016833A (es) | 2014-06-16 | 2017-10-04 | Univ Johns Hopkins | Composiciones y metodos para la expresion de arns guias crispr que usan el promotor h1. |
WO2016016449A1 (en) | 2014-07-31 | 2016-02-04 | Association Institut De Myologie | Treatment of amyotrophic lateral sclerosis |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
US11104897B2 (en) | 2015-04-27 | 2021-08-31 | Genethon | Compositions and methods for the treatment of nucleotide repeat expansion disorders |
US10016514B2 (en) | 2015-05-15 | 2018-07-10 | New Hope Research Foundation | Polynucleotides, vectors and methods for insertion and expression of transgenes |
PT3298134T (pt) | 2015-05-16 | 2023-08-18 | Genzyme Corp | Edição génica de mutações intrónicas profundas |
EP3302677A4 (en) | 2015-06-01 | 2019-02-20 | University of Massachusetts | CATHETER SETS |
WO2016210372A2 (en) | 2015-06-25 | 2016-12-29 | University Of Southern California | Methods to treat neurological diseases |
US20180214576A1 (en) | 2015-07-28 | 2018-08-02 | University Of Massachusetts | Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector |
US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
CA3009308A1 (en) | 2015-12-23 | 2017-06-29 | Chad Albert COWAN | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
SG10202102997UA (en) | 2016-09-30 | 2021-04-29 | Regeneron Pharma | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
EP3684937A4 (en) | 2017-09-22 | 2021-06-02 | University of Massachusetts | DUAL EXPRESSION VECTORS OF SOD1 AND THEIR USES |
-
2015
- 2015-03-18 AU AU2015231294A patent/AU2015231294B2/en active Active
- 2015-03-18 DK DK15764861.9T patent/DK3119797T3/da active
- 2015-03-18 ES ES15764861T patent/ES2856403T3/es active Active
- 2015-03-18 US US15/126,688 patent/US10280418B2/en active Active
- 2015-03-18 EP EP15764861.9A patent/EP3119797B1/en active Active
- 2015-03-18 JP JP2017501101A patent/JP6756700B2/ja active Active
- 2015-03-18 WO PCT/US2015/021321 patent/WO2015143078A1/en active Application Filing
- 2015-03-18 EP EP20183218.5A patent/EP3750907A3/en active Pending
- 2015-03-18 CA CA2942515A patent/CA2942515A1/en active Pending
-
2019
- 2019-03-07 US US16/295,621 patent/US10954518B2/en active Active
- 2019-03-25 US US16/364,126 patent/US10711274B2/en active Active
- 2019-05-06 US US16/403,948 patent/US10851375B2/en active Active
- 2019-09-02 JP JP2019159514A patent/JP7051118B2/ja active Active
-
2020
- 2020-11-02 AU AU2020260765A patent/AU2020260765B2/en active Active
-
2021
- 2021-02-12 US US17/174,452 patent/US11760999B2/en active Active
- 2021-06-02 JP JP2021093003A patent/JP2021152030A/ja active Pending
-
2023
- 2023-02-03 AU AU2023200576A patent/AU2023200576A1/en active Pending
- 2023-07-31 US US18/362,190 patent/US20240076668A1/en active Pending
-
2024
- 2024-01-04 JP JP2024000089A patent/JP2024032730A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
AU2020260765B2 (en) | 2022-12-01 |
AU2015231294B2 (en) | 2020-10-29 |
JP2017510298A (ja) | 2017-04-13 |
US10851375B2 (en) | 2020-12-01 |
EP3119797A4 (en) | 2018-04-18 |
US20170114340A1 (en) | 2017-04-27 |
EP3750907A2 (en) | 2020-12-16 |
AU2015231294A1 (en) | 2016-10-06 |
US20240076668A1 (en) | 2024-03-07 |
US20210246450A1 (en) | 2021-08-12 |
WO2015143078A1 (en) | 2015-09-24 |
JP7051118B2 (ja) | 2022-04-11 |
US10711274B2 (en) | 2020-07-14 |
US20200032256A1 (en) | 2020-01-30 |
ES2856403T3 (es) | 2021-09-27 |
AU2020260765A1 (en) | 2020-11-26 |
US10954518B2 (en) | 2021-03-23 |
CA2942515A1 (en) | 2015-09-24 |
JP6756700B2 (ja) | 2020-09-16 |
EP3119797B1 (en) | 2020-12-23 |
EP3750907A3 (en) | 2021-04-28 |
JP2020019772A (ja) | 2020-02-06 |
EP3119797A1 (en) | 2017-01-25 |
US20190276826A1 (en) | 2019-09-12 |
JP2021152030A (ja) | 2021-09-30 |
US10280418B2 (en) | 2019-05-07 |
US11760999B2 (en) | 2023-09-19 |
AU2023200576A1 (en) | 2023-04-27 |
US20190316126A1 (en) | 2019-10-17 |
JP2024032730A (ja) | 2024-03-12 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DK3119797T3 (da) | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose | |
DK3102673T3 (da) | Fremgangsmåder og sammensætninger til behandling af beta-talassæmi | |
DK3185876T3 (da) | Sammensætninger og fremgangsmåder til behandling af neurologiske lidelser | |
DK3234133T3 (da) | Crispr-baserede sammensætninger og fremgangsmåder til anvendelse | |
DK3212226T3 (da) | Sammensætninger og anvendelsesfremgangsmåder til behandling af metaboliske lidelser | |
DK3206493T3 (da) | Sammensætninger og fremgangsmåder til behandling af cns-forstyrrelser | |
DK3149045T3 (da) | Sammensætninger og fremgangsmåder vedrørende universelle glycoformer til forbedret antistofeffekt | |
DK3212778T3 (da) | Fremgangsmåder og sammensætninger til stabilisering af trans-splejsning af intein-modificerede proteaser | |
DK3224269T3 (da) | Sammensætninger og fremgangsmåder til behandling af cns-forstyrrelser | |
DK3092256T3 (da) | Forbindelser og sammensætninger til immunterapi | |
DK3495023T3 (da) | Semifluorerede forbindelser og sammensætninger deraf | |
DK3137605T3 (da) | Sammensætninger og fremgangsmåder til modulering af angiopoietin-lignende-3-ekspression | |
DK3125927T3 (da) | Fremgangsmåder og sammensætninger til immunmodulering | |
DK3466432T3 (da) | Fremgangsmåder til behandling af pulmonale non-tuberkuløse mykobakterielle infektioner | |
DK3134130T3 (da) | Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier | |
DK3277270T3 (da) | Sammensætninger og fremgangsmåder til behandling af anæmi | |
DK3481846T3 (da) | 11-substituerede 24-hydroxysteroler til anvendelse til behandling af nmda-relaterede tilstande | |
DK3102555T3 (da) | Sammensætninger af forbindelser og anvendelser deraf | |
DK3393655T3 (da) | Fenfluramin-sammensætninger og fremgangsmåder til fremstilling af samme | |
DK3144001T3 (da) | Sammensætninger af selenoorganiske forbindelser og fremgangsmåder til anvendelse deraf | |
DK3007695T3 (da) | Sammensætninger og fremgangsmåder til behandling af anæmi | |
DK3209696T3 (da) | Fremgangsmåder og sammensætninger til diagnosticering og behandling af glioblastom | |
DK3189074T3 (da) | Sammensætninger og metoder til behandling og forebyggelse af inflammation | |
DK3102213T3 (da) | Farmaceutiske sammensætninger til behandlingen af inflammatoriske lidelser | |
DK3200815T3 (da) | Fremgangsmåder og sammensætninger til behandling af cancer |