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 nucleic acids [NA]
-
- 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 nucleic acids [NA]
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Physical Education & Sports Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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 (3) | EP4410805A3 (da) |
| JP (4) | JP6756700B2 (da) |
| AU (3) | AU2015231294B2 (da) |
| CA (2) | CA3276269A1 (da) |
| DK (1) | DK3119797T3 (da) |
| ES (1) | ES2856403T3 (da) |
| WO (1) | WO2015143078A1 (da) |
Families Citing this family (64)
| 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 |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| US9272053B2 (en) | 2010-04-23 | 2016-03-01 | University Of Massachusetts | AAV-based treatment of cholesterol-related disorders |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| CA3276269A1 (en) | 2014-03-18 | 2026-03-02 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | 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 |
| RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| EP3689139B1 (en) | 2015-05-29 | 2025-01-01 | Regeneron Pharmaceuticals, Inc. | Rodent cells having a disruption in a c9orf72 locus |
| WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| 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 |
| WO2017139643A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| EP3432933B1 (en) * | 2016-03-22 | 2022-10-19 | President and Fellows of Harvard College | Biocompatible adhesives and methods of use thereof |
| EP3436078B1 (en) * | 2016-03-31 | 2021-10-27 | University of Cincinnati | Methods and compositions for the treatment of als |
| WO2017173411A1 (en) | 2016-04-02 | 2017-10-05 | Research Institute At Nation Wide Children's Hospital | Modified u6 promoter system for tissue specific expression |
| WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| KR20220108216A (ko) * | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| CA3026103A1 (en) * | 2016-06-10 | 2017-12-14 | Saunders, Ann M. | Methods for detecting structural variants in neurodegenerative disease |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | 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 |
| EP3518667B1 (en) * | 2016-09-30 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Rodents having a hexanucleotide repeat expansion in a c9orf72 locus |
| CA3040483A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
| KR102476709B1 (ko) | 2016-11-21 | 2022-12-09 | 나노스트링 테크놀로지스, 인크. | 화학적 조성물 및 이것을 사용하는 방법 |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP2020518266A (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． | 調節性ポリヌクレオチド |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| WO2019051025A2 (en) * | 2017-09-06 | 2019-03-14 | The Trustees Of Columbia University In The City Of New York | TREATMENT OF AGGRESSIVE CANCERS BY TARGETING C9ORF72 |
| US11739330B2 (en) * | 2017-09-22 | 2023-08-29 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN111819281B (zh) * | 2017-10-23 | 2024-12-31 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
| CN112292134A (zh) * | 2018-04-03 | 2021-01-29 | 中央研究院 | Mir-17~92作为运动神经元(mn)退化疾病的治疗或诊断目标 |
| JP7525402B2 (ja) | 2018-05-14 | 2024-07-30 | ナノストリング テクノロジーズ，インコーポレイティド | 化学的組成物とそれを利用する方法 |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| EP3850098A1 (en) * | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
| AU2019389047A1 (en) * | 2018-11-30 | 2021-05-20 | Novartis Ag | AAV viral vectors and uses thereof |
| US11690362B2 (en) | 2018-12-20 | 2023-07-04 | Regeneran Pharmaceuticals, Inc. | Nuclease-mediated repeat expansion |
| WO2021055703A1 (en) | 2019-09-20 | 2021-03-25 | President And Fellows Of Harvard College | Tough gel-based drug delivery compositions and methods thereof |
| WO2021067613A1 (en) * | 2019-10-01 | 2021-04-08 | Children's Medical Center Corporation | Compositions and methods for treating amyotrophic lateral sclerosis |
| CA3156176A1 (en) * | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US20230070049A1 (en) * | 2020-02-03 | 2023-03-09 | Rutgers, The State University Of New Jersey | Microrna-7 compositions for promoting functional recovery following spinal cord injury 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 |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | 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 |
| AU2022255175A1 (en) | 2021-04-06 | 2023-11-23 | Trace Neuroscience, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
| US12234472B2 (en) | 2021-10-18 | 2025-02-25 | Regeneron Pharmaceuticals, Inc. | Eukaryotic cells comprising adenovirus-associated virus polynucleotides |
| CN120283053A (zh) | 2022-10-05 | 2025-07-08 | 追踪神经科学公司 | Unc13a反义寡核苷酸和其用途 |
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 |
| AU762220B2 (en) | 1998-05-28 | 2003-06-19 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | 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 |
| WO2001029243A1 (en) | 1999-10-15 | 2001-04-26 | Dalhousie University | 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 |
| ATE450621T2 (de) | 2000-03-30 | 2009-12-15 | Whitehead Biomedical Inst | Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | 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 |
| AU2002315393A1 (en) | 2001-06-21 | 2003-01-08 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| WO2003004660A1 (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 |
| ES2609014T3 (es) | 2001-07-12 | 2017-04-18 | University Of Massachusetts | Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico |
| JP2004534543A (ja) | 2001-07-13 | 2004-11-18 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | シュードタイプ化アデノ関連ウイルスおよびその使用 |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2526341T3 (es) | 2001-12-17 | 2015-01-09 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas |
| 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) |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
| AU2003221745A1 (en) | 2002-04-19 | 2003-11-03 | University Of Florida | rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES |
| WO2003093460A1 (en) | 2002-04-29 | 2003-11-13 | 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 |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| WO2004014933A1 (en) | 2002-08-07 | 2004-02-19 | University Of Massachusetts | 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 |
| AU2004208031B2 (en) | 2003-01-28 | 2009-10-08 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application 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 |
| ES2712695T3 (es) | 2003-06-02 | 2019-05-14 | Univ Massachusetts | Métodos y composiciones para controlar la eficacia de la silenciación del ARN |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| EP2530157B1 (en) | 2003-07-31 | 2016-09-28 | Regulus Therapeutics Inc. | Oligomeric compounds and compositions for use in modulation of miRNAs |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| 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 |
| KR101246862B1 (ko) | 2004-03-05 | 2013-03-27 | 베니텍 리미티드 | ＲＮＡｉ 제제의 동시 전달을 위한 다중 프로모터 발현카세트 |
| US7498316B2 (en) | 2004-04-06 | 2009-03-03 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using RNA interference |
| EP1752536A4 (en) * | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE 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 |
| AU2005316476A1 (en) | 2004-12-15 | 2006-06-22 | University Of Florida Research Foundation, Inc. | Chimeric vectors |
| 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 |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| ES2564823T3 (es) | 2005-05-27 | 2016-03-29 | Ospedale San Raffaele S.R.L. | Vector génico que comprende miARN |
| US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| WO2007027775A2 (en) | 2005-08-29 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Methods for use in modulating mir-122a |
| WO2008016391A2 (en) | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| DK2514823T3 (da) | 2006-03-03 | 2018-07-23 | Promis Neurosciences Inc | Fremgangsmåder og sammensætninger til at behandle og detektere fejlfoldet SOD1-medierede sygdomme |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| DK2548438T3 (da) | 2006-11-08 | 2015-10-19 | Veritas Bio LLC | In vivo indgivelse af dobbeltstrenget rna til en celle |
| 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 |
| EP2160477B1 (en) | 2007-05-23 | 2013-08-07 | Dharmacon, Inc. | Micro-RNA scaffolds and non-naturally occurring micro-RNAs |
| WO2008150897A2 (en) | 2007-05-31 | 2008-12-11 | 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 |
| EP2247729B1 (en) | 2008-02-11 | 2019-05-01 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| US20090215879A1 (en) | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| EP2268811A1 (en) | 2008-03-07 | 2011-01-05 | 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 |
| US8222221B2 (en) | 2008-06-04 | 2012-07-17 | 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 |
| WO2010005850A1 (en) | 2008-07-08 | 2010-01-14 | The J. David Gladstone Institutes | Methods and compositions for modulating angiogenesis |
| CA2744804C (en) | 2008-09-04 | 2019-02-05 | Galenea Corp. | Synaptic vesicle cycling assays and systems |
| 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 |
| WO2010099383A2 (en) | 2009-02-26 | 2010-09-02 | The Johns Hopkins University | Compositions and methods for ex vivo hepatic nucleic acid delivery |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| 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 |
| EP2498825B1 (en) | 2009-11-09 | 2017-03-29 | Genepod Therapeutics Ab | Novel viral vector construct for neuron specific continuous dopa synthesis in vivo |
| EP2508603B1 (en) | 2009-11-19 | 2015-05-13 | National University Corporation Okayama University | System for increasing gene expression, and vector supporting said system |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| 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 |
| CA2791974A1 (en) | 2010-03-01 | 2011-09-09 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the hcv genome |
| US9272053B2 (en) | 2010-04-23 | 2016-03-01 | University Of Massachusetts | AAV-based treatment of cholesterol-related disorders |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
| 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 |
| WO2012109667A1 (en) | 2011-02-12 | 2012-08-16 | 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 |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | 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 |
| CA2846307C (en) | 2011-08-31 | 2020-03-10 | University College Cardiff Consultants Limited | Method for diagnosing a neurodegenerative disease |
| WO2013036833A1 (en) * | 2011-09-09 | 2013-03-14 | Mayo Foundation For Medical Education And Research | Detecting frontotemporal dementia and amyotrophic lateral sclerosis |
| EP2766482B1 (en) * | 2011-10-11 | 2016-12-07 | 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 |
| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| JP6417322B2 (ja) * | 2012-06-21 | 2018-11-07 | アソシアシオン・アンスティテュ・ドゥ・ミオロジーＡｓｓｏｃｉａｔｉｏｎ Ｉｎｓｔｉｔｕｔ Ｄｅ Ｍｙｏｌｏｇｉｅ | 遺伝子治療ベクターの広範な遺伝子送達 |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| RU2730677C2 (ru) * | 2012-10-15 | 2020-08-24 | Ионис Фармасьютикалз, Инк. | Соединение для модуляции экспрессии гена c9orf72 и его применение |
| IL239317B (en) | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications |
| CA2897932A1 (en) | 2013-01-14 | 2014-07-17 | Recombinetics, Inc. | Hornless livestock |
| 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 |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | 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 |
| EP2986635B1 (en) | 2013-04-18 | 2018-10-03 | 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 |
| ES3029138T3 (en) | 2013-06-05 | 2025-06-23 | Univ Duke | Rna-guided gene editing and gene regulation |
| MX2015017312A (es) | 2013-06-17 | 2017-04-10 | Broad Inst Inc | Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas. |
| WO2015009575A1 (en) | 2013-07-16 | 2015-01-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to cep290 |
| EP3030665B9 (en) | 2013-08-05 | 2023-10-04 | 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 |
| RS63942B1 (sr) * | 2013-08-27 | 2023-02-28 | Res Inst Nationwide Childrens Hospital | Proizvodi i postupci za lečenje amiotrofične lateralne skleroze |
| SG10201808903UA (en) * | 2013-10-11 | 2018-11-29 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| BR112016013547A2 (pt) | 2013-12-12 | 2017-10-03 | Broad Inst Inc | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
| 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 |
| WO2015150922A2 (en) | 2014-03-17 | 2015-10-08 | The Hospital For Sick Children | β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES |
| CA3276269A1 (en) | 2014-03-18 | 2026-03-02 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
| EP3137497B2 (en) | 2014-05-02 | 2025-03-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| ES2759317T3 (es) | 2014-05-20 | 2020-05-08 | Univ Iowa Res Found | Compuestos terapéuticos para la enfermedad de Huntington |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| EP3674408A1 (en) | 2014-06-16 | 2020-07-01 | The Johns Hopkins University | Compositions and methods for the expression of crispr guide rnas |
| US10590420B2 (en) | 2014-07-31 | 2020-03-17 | Association Institut De Myologie | Treatment of amyotrophic lateral sclerosis |
| EP3200830B1 (en) | 2014-10-03 | 2020-09-09 | University of Massachusetts | High efficiency library-identified aav vectors |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| EP4491733A3 (en) | 2015-04-23 | 2025-03-26 | University of Massachusetts | Modulation of aav vector transgene expression |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| EP3289081B1 (en) | 2015-04-27 | 2019-03-27 | 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 |
| RU2021132397A (ru) | 2015-05-16 | 2022-02-24 | Джензим Корпорейшн | Генное редактирование глубоких интронных мутаций |
| EP3302677A4 (en) | 2015-06-01 | 2019-02-20 | University of Massachusetts | CATHETER ARRANGEMENTS |
| US10758545B2 (en) | 2015-06-25 | 2020-09-01 | 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 |
| MX2018007840A (es) | 2015-12-23 | 2019-05-02 | Crispr Therapeutics Ag | Materiales y metodos para el tratamiento de la esclerosis lateral amiotrofica y/o la degeneracion lobar frontotemporal. |
| EP3518667B1 (en) | 2016-09-30 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Rodents having a hexanucleotide repeat expansion in a c9orf72 locus |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US11739330B2 (en) | 2017-09-22 | 2023-08-29 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
-
2015
- 2015-03-18 CA CA3276269A patent/CA3276269A1/en active Pending
- 2015-03-18 ES ES15764861T patent/ES2856403T3/es active Active
- 2015-03-18 EP EP24170036.8A patent/EP4410805A3/en active Pending
- 2015-03-18 EP EP20183218.5A patent/EP3750907A3/en not_active Withdrawn
- 2015-03-18 JP JP2017501101A patent/JP6756700B2/ja active Active
- 2015-03-18 DK DK15764861.9T patent/DK3119797T3/da active
- 2015-03-18 AU AU2015231294A patent/AU2015231294B2/en active Active
- 2015-03-18 CA CA2942515A patent/CA2942515C/en active Active
- 2015-03-18 WO PCT/US2015/021321 patent/WO2015143078A1/en not_active Ceased
- 2015-03-18 US US15/126,688 patent/US10280418B2/en active Active
- 2015-03-18 EP EP15764861.9A patent/EP3119797B1/en active Active
-
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/AU2023200576B2/en active Active
- 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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK3102673T3 (da) | Fremgangsmåder og sammensætninger til behandling af beta-talassæmi | |
| DK3185876T3 (da) | Sammensætninger og fremgangsmåder til behandling af neurologiske lidelser | |
| DK3224269T3 (da) | Sammensætninger og fremgangsmåder til behandling af cns-forstyrrelser | |
| DK3206493T3 (da) | Sammensætninger og fremgangsmåder til behandling af cns-forstyrrelser | |
| DK3212226T3 (da) | Sammensætninger og anvendelsesfremgangsmåder til behandling af metaboliske lidelser | |
| DK3234133T3 (da) | Crispr-baserede sammensætninger og fremgangsmåder til anvendelse | |
| DK3092256T3 (da) | Forbindelser og sammensætninger til immunterapi | |
| 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 | |
| DK3137605T3 (da) | Sammensætninger og fremgangsmåder til modulering af angiopoietin-lignende-3-ekspression | |
| DK3277270T3 (da) | Sammensætninger og fremgangsmåder til behandling af anæmi | |
| DK3466432T3 (da) | Fremgangsmåder til behandling af pulmonale non-tuberkuløse mykobakterielle infektioner | |
| DK3189074T3 (da) | Sammensætninger og metoder til behandling og forebyggelse af inflammation | |
| DK3144001T3 (da) | Sammensætninger af selenoorganiske forbindelser og fremgangsmåder til anvendelse deraf | |
| DK3134130T3 (da) | Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier | |
| DK3102555T3 (da) | Sammensætninger af forbindelser og anvendelser deraf | |
| DK3102213T3 (da) | Farmaceutiske sammensætninger til behandlingen af inflammatoriske lidelser | |
| DK3481846T3 (da) | 11-substituerede 24-hydroxysteroler til anvendelse til behandling af nmda-relaterede tilstande | |
| DK3007695T3 (da) | Sammensætninger og fremgangsmåder til behandling af anæmi | |
| DK3081263T3 (da) | Sammensætninger og redskaber til behandling af glaukom | |
| DK3200815T3 (da) | Fremgangsmåder og sammensætninger til behandling af cancer | |
| DK3524255T3 (da) | Sammensætning til behandling af acne | |
| DK3242947T3 (da) | Genterapi og elektroporese til behandling af maligniteter | |
| DK3209696T3 (da) | Fremgangsmåder og sammensætninger til diagnosticering og behandling af glioblastom | |
| DK3157534T3 (da) | Acetylcholinesterasehæmmere til behandling af dermatologiske tilstande |