RU2018120484A - Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии - Google Patents
Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии Download PDFInfo
- Publication number
- RU2018120484A RU2018120484A RU2018120484A RU2018120484A RU2018120484A RU 2018120484 A RU2018120484 A RU 2018120484A RU 2018120484 A RU2018120484 A RU 2018120484A RU 2018120484 A RU2018120484 A RU 2018120484A RU 2018120484 A RU2018120484 A RU 2018120484A
- Authority
- RU
- Russia
- Prior art keywords
- abcd1
- vector
- subject
- aav9
- expression
- Prior art date
Links
- 201000011452 Adrenoleukodystrophy Diseases 0.000 title claims 9
- 238000007913 intrathecal administration Methods 0.000 title claims 5
- 150000007523 nucleic acids Chemical group 0.000 title claims 4
- 208000018126 Adrenomyeloneuropathy Diseases 0.000 title 1
- 102000009030 Member 1 Subfamily D ATP Binding Cassette Transporter Human genes 0.000 title 1
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 title 1
- 238000000034 method Methods 0.000 claims 15
- 208000010796 X-linked adrenoleukodystrophy Diseases 0.000 claims 8
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 210000000056 organ Anatomy 0.000 claims 3
- 230000002093 peripheral effect Effects 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 239000013592 cell lysate Substances 0.000 claims 2
- 230000002452 interceptive effect Effects 0.000 claims 2
- 241000649044 Adeno-associated virus 9 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000003204 osmotic effect Effects 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- 238000001890 transfection Methods 0.000 claims 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
- C12N7/02—Recovery or purification
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0002—Galenical forms characterised by the drug release technique; Application systems commanded by energy
- A61K9/0004—Osmotic delivery systems; Sustained release driven by osmosis, thermal energy or gas
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- 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
- 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
- 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
-
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Claims (16)
1. Способ увеличения титров вектора на основе аденоассоциированного вируса 9 (AAV9) в трансфицированных клетках-продуцентах, выращенных в культуре, при этом указанный способ предусматривает стадии:
i) инкубирования последовательности нуклеиновой кислоты, которая комплементарна mRNA, кодирующей представителя 1 надсемейства D АТФ-связывающей кассеты (ABCD1), с клетками и
ii) трансфицирования клеток вектором на основе AAV9, содержащим нуклеотидную последовательность, кодирующую ABCD1 (вектор AAV9-ABCD1), где обеспечивают снижение количества mRNA ABCD1, экспрессируемой из вектора на основе AAV9, вследствие чего достигается увеличение выхода вектора AAV9-ABCD1 в клеточном лизате и/или среде, составляющее от приблизительно 1 раза до приблизительно 50 раз, по сравнению с эталонным стандартом.
2. Способ по п. 1, где последовательность нуклеиновой кислоты, которая комплементарна mRNA, кодирующей ABCD1, представляет собой интерферирующую РНК.
3. Способ по п. 2, где интерферирующая РНК представляет собой shRNA или siRNA.
4. Способ по п. 3, где siRNA содержит SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7 или их комбинацию.
5. Способ по п. 1, где эталонный стандарт предусматривает выход вектора AAV9-ABCD1 в клеточном лизате и/или среде клеток-продуцентов, которые не инкубировали с последовательностью нуклеиновой кислоты, которая комплементарна mRNA, кодирующей ABCD1.
6. Способ лечения X-сцепленной адренолейкодистрофии (X-ALD) у субъекта, нуждающегося в этом, предусматривающий введение субъекту композиции, содержащей очищенный вектор AAV9-ABCD1, полученный из клеток-продуцентов по в п. 1.
7. Способ по п. 6, где композицию, содержащую очищенный вектор AAV9-ABCD1, вводят субъекту посредством интратекального введения.
8. Способ лечения X-сцепленной адренолейкодистрофии (X-ALD) у субъекта, нуждающегося в этом, предусматривающий введение субъекту вектора на основе аденоассоциированного вируса (AAV), кодирующего представителя 1 надсемейства D АТФ-связывающей кассеты (ABCD1), где указанный вектор вводят субъекту посредством интратекального введения.
9. Способ по п. 8, где интратекальное введение опосредовано осмотическим насосом.
10. Способ по п. 8, где доза вектора составляет от приблизительно 1×1013 к. г. до приблизительно 10×1013 к. г.
11. Способ по п. 8, где AAV представляет собой AAV9.
12. Способ доставки представителя 1 надсемейства D АТФ-связывающей кассеты (ABCD1) субъекту с X-сцепленной адренолейкодистрофией (X-ALD), предусматривающий введение субъекту вектора, кодирующего ABCD1, где указанный вектор вводят субъекту посредством интратекального введения, и где экспрессия ABCD1 из указанного вектора в центральной нервной системе является меньшей, чем экспрессия ABCD1 из указанного вектора в периферических органах.
13. Способ по п. 12, где экспрессия ABCD1 из указанного вектора в центральной нервной системе превышает в приблизительно 3 раза экспрессию ABCD1 в центральной нервной системе необработанного субъекта, у которого X-ALD отсутствует.
14. Способ по п. 13, где экспрессия ABCD1 из указанного вектора в периферических органах является на приблизительно 90% меньшей, чем экспрессия ABCD1 в периферических органах необработанного субъекта, у которого X-ALD отсутствует.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251208P | 2015-11-05 | 2015-11-05 | |
| US62/251,208 | 2015-11-05 | ||
| US201662300691P | 2016-02-26 | 2016-02-26 | |
| US62/300,691 | 2016-02-26 | ||
| PCT/US2016/060375 WO2017079467A1 (en) | 2015-11-05 | 2016-11-03 | Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020141860A Division RU2020141860A (ru) | 2015-11-05 | 2016-11-03 | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2018120484A true RU2018120484A (ru) | 2019-12-05 |
| RU2018120484A3 RU2018120484A3 (ru) | 2020-03-11 |
| RU2739384C2 RU2739384C2 (ru) | 2020-12-23 |
Family
ID=58662371
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018120484A RU2739384C2 (ru) | 2015-11-05 | 2016-11-03 | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
| RU2020141860A RU2020141860A (ru) | 2015-11-05 | 2016-11-03 | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020141860A RU2020141860A (ru) | 2015-11-05 | 2016-11-03 | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US20180320176A1 (ru) |
| EP (2) | EP4066862A1 (ru) |
| JP (2) | JP7037490B2 (ru) |
| KR (1) | KR20180066252A (ru) |
| CN (1) | CN108472264A (ru) |
| AU (1) | AU2016349381B2 (ru) |
| BR (1) | BR112018008993A2 (ru) |
| CA (1) | CA3003747A1 (ru) |
| ES (1) | ES2916325T3 (ru) |
| IL (1) | IL258904B (ru) |
| MX (1) | MX2018005689A (ru) |
| PL (1) | PL3370705T3 (ru) |
| RU (2) | RU2739384C2 (ru) |
| SG (1) | SG11201803353PA (ru) |
| WO (1) | WO2017079467A1 (ru) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112022013027A2 (pt) * | 2019-12-31 | 2022-09-06 | Swanbio Therapeutics Ltd | Construtos aav-abcd1 melhorados e uso para tratamento ou prevenção de adrenoleucodistrofia (ald) e/ou adrenomieloeuropatia (amn) |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1975A (en) | 1841-02-12 | Manner of constructing corn-shellers | ||
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5609885A (en) | 1992-09-15 | 1997-03-11 | Alza Corporation | Osmotic membrane and delivery device |
| US5869039A (en) * | 1993-10-15 | 1999-02-09 | Institut National De La Sante Et De La Recherche Medicale | X-linked adrenoleukodystrophy gene and corresponding protein |
| US6132420A (en) | 1996-02-02 | 2000-10-17 | Alza Corporation | Osmotic delivery system and method for enhancing start-up and performance of osmotic delivery systems |
| US6156331A (en) | 1996-02-02 | 2000-12-05 | Alza Corporation | Sustained delivery of an active agent using an implantable system |
| US6261584B1 (en) | 1996-02-02 | 2001-07-17 | Alza Corporation | Sustained delivery of an active agent using an implantable system |
| US6395292B2 (en) | 1996-02-02 | 2002-05-28 | Alza Corporation | Sustained delivery of an active agent using an implantable system |
| WO1997027840A1 (en) | 1996-02-02 | 1997-08-07 | Alza Corporation | Sustained delivery of an active agent using an implantable system |
| US6713300B1 (en) * | 1997-02-27 | 2004-03-30 | University Of Utah Research Foundation | Nucleic acid and amino acid sequences for ATP-binding cassette transporter and methods of screening for agents that modify ATP-binding cassette transporter |
| ZA981610B (en) | 1997-03-24 | 1999-08-26 | Alza Corp | Self adjustable exit port. |
| MY125849A (en) | 1997-07-25 | 2006-08-30 | Alza Corp | Osmotic delivery system, osmotic delivery system semipermeable body assembly, and method for controlling delivery rate of beneficial agents from osmotic delivery systems |
| AU1623099A (en) | 1997-12-22 | 1999-07-12 | Alza Corporation | Rate controlling membranes for controlled drug delivery devices |
| DE69822208T2 (de) | 1997-12-29 | 2005-04-28 | Alza Corp., Mountain View | Osmotisches verabreichungssystem mit stöpselrückhaltemechanismus |
| ES2378675T3 (es) | 1997-12-30 | 2012-04-16 | Intarcia Therapeutics, Inc | Sistema de suministro de un agente beneficioso con una membrana obturadora |
| NZ512671A (en) | 1998-12-31 | 2003-12-19 | Alza Corp | Osmotic delivery system having space efficient piston |
| US6225525B1 (en) * | 1999-10-13 | 2001-05-01 | Ortho-Mcneil Pharmaceutical, Inc. | ATP-binding cassette transporter (ABC1) modified transgenic mice |
| EP1328256B1 (en) | 1999-12-21 | 2005-10-19 | Alza Corporation | Valve for osmotic devices |
| US7163688B2 (en) | 2001-06-22 | 2007-01-16 | Alza Corporation | Osmotic implant with membrane and membrane retention means |
| US20050032219A1 (en) * | 2001-07-03 | 2005-02-10 | Patrick Aubourg | Methods of administering vectors to synaptically connected neurons |
| EP1551493B1 (en) | 2002-06-26 | 2007-10-31 | Alza Corporation | Minimally compliant, volume efficient piston for osmotic drug delivery systems |
| US7014636B2 (en) | 2002-11-21 | 2006-03-21 | Alza Corporation | Osmotic delivery device having a two-way valve and a dynamically self-adjusting flow channel |
| JP2006521897A (ja) | 2003-03-31 | 2006-09-28 | アルザ・コーポレーション | 内部圧力を放散する手段を備える浸透ポンプ |
| US20050175701A1 (en) | 2004-02-10 | 2005-08-11 | Alza Corporation | Capillary moderator for osmotic delivery system |
| ES2351527T3 (es) | 2006-05-30 | 2011-02-07 | Intarcia Therapeutics, Inc | Modulador de flujo en dos piezas con conducto interno para un sistema osmótico de administración. |
| NZ574524A (en) | 2006-08-09 | 2011-07-29 | Intarcia Therapeutics Inc | Piston assembly for positioning lumen of a reservoir for an osmotic delivery having a columnar body and a spring |
| WO2011106376A2 (en) * | 2010-02-23 | 2011-09-01 | The General Hospital Corporation | Use of microvesicles in the treatment of medical conditions |
| CA2838749A1 (en) * | 2011-06-10 | 2012-12-13 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| GB201401707D0 (en) * | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
-
2016
- 2016-11-03 EP EP22159649.7A patent/EP4066862A1/en not_active Withdrawn
- 2016-11-03 WO PCT/US2016/060375 patent/WO2017079467A1/en active Application Filing
- 2016-11-03 KR KR1020187015254A patent/KR20180066252A/ko not_active Application Discontinuation
- 2016-11-03 RU RU2018120484A patent/RU2739384C2/ru active
- 2016-11-03 SG SG11201803353PA patent/SG11201803353PA/en unknown
- 2016-11-03 MX MX2018005689A patent/MX2018005689A/es unknown
- 2016-11-03 JP JP2018543041A patent/JP7037490B2/ja active Active
- 2016-11-03 CA CA3003747A patent/CA3003747A1/en active Pending
- 2016-11-03 EP EP16862990.5A patent/EP3370705B1/en active Active
- 2016-11-03 IL IL258904A patent/IL258904B/en unknown
- 2016-11-03 AU AU2016349381A patent/AU2016349381B2/en active Active
- 2016-11-03 BR BR112018008993A patent/BR112018008993A2/pt active Search and Examination
- 2016-11-03 ES ES16862990T patent/ES2916325T3/es active Active
- 2016-11-03 US US15/773,337 patent/US20180320176A1/en not_active Abandoned
- 2016-11-03 CN CN201680077741.7A patent/CN108472264A/zh active Pending
- 2016-11-03 PL PL16862990.5T patent/PL3370705T3/pl unknown
- 2016-11-03 RU RU2020141860A patent/RU2020141860A/ru unknown
-
2019
- 2019-04-02 US US16/373,081 patent/US10519445B2/en active Active
-
2022
- 2022-01-17 JP JP2022005016A patent/JP2022064910A/ja active Pending
-
2023
- 2023-04-04 US US18/295,566 patent/US20230365967A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20180066252A (ko) | 2018-06-18 |
| AU2016349381A1 (en) | 2018-06-21 |
| US20230365967A1 (en) | 2023-11-16 |
| WO2017079467A1 (en) | 2017-05-11 |
| EP3370705A4 (en) | 2019-05-15 |
| CN108472264A (zh) | 2018-08-31 |
| MX2018005689A (es) | 2018-08-01 |
| RU2018120484A3 (ru) | 2020-03-11 |
| EP4066862A1 (en) | 2022-10-05 |
| JP2022064910A (ja) | 2022-04-26 |
| IL258904B (en) | 2022-09-01 |
| AU2016349381B2 (en) | 2023-02-23 |
| EP3370705B1 (en) | 2022-03-09 |
| ES2916325T3 (es) | 2022-06-30 |
| US20190225967A1 (en) | 2019-07-25 |
| US10519445B2 (en) | 2019-12-31 |
| SG11201803353PA (en) | 2018-05-30 |
| IL258904A (en) | 2018-06-28 |
| RU2020141860A (ru) | 2021-04-01 |
| BR112018008993A2 (pt) | 2018-10-30 |
| EP3370705A1 (en) | 2018-09-12 |
| CA3003747A1 (en) | 2017-05-11 |
| US20180320176A1 (en) | 2018-11-08 |
| PL3370705T3 (pl) | 2022-08-01 |
| RU2739384C2 (ru) | 2020-12-23 |
| JP2018537124A (ja) | 2018-12-20 |
| JP7037490B2 (ja) | 2022-03-16 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20210254061A1 (en) | Systems and methods for the treatment of hemoglobinopathies | |
| Stanek et al. | Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease | |
| US9090894B2 (en) | Modulating immune system development and function through microRNA MIR-146 | |
| JP2016500519A5 (ru) | ||
| EP4186975A1 (en) | Non-integrating dna vectors for the genetic modification of cells | |
| WO2019034784A1 (en) | USE OF ANTI-CRISPRANT POLYPEPTIDES FOR THE SPECIFIC ACTIVATION OF NUCLEASES CASES | |
| Zeng et al. | Lentivirus-mediated RNA interference of DC-STAMP expression inhibits the fusion and resorptive activity of human osteoclasts | |
| RU2018120484A (ru) | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии | |
| JP2022501044A (ja) | ヒトアンジェルマン症候群において父方ｕｂｅ３ａ遺伝子発現を復活させるための組成物および方法 | |
| US20120264805A1 (en) | Medicament for the treatment and prevention of liver failure | |
| CA3176553A1 (en) | Improved scaffolds for multiplexed inhibitory rna | |
| US20200263206A1 (en) | Targeted integration systems and methods for the treatment of hemoglobinopathies | |
| US20220290157A1 (en) | Compositions and methods for treating amyotrophic lateral sclerosis | |
| AU2020242848A1 (en) | Expression constructs for the genetic modification of cells | |
| US20090060889A1 (en) | Ii-RNAi involved Ii suppression in cancer immunotherapy | |
| EP2708601A1 (en) | Treatment of heart disease through modulation of hypoxia induced eRNA activity | |
| JP2020517580A (ja) | 電離放射線耐性腫瘍を治療するための方法及び組成物 | |
| US20230098714A1 (en) | Microrna compositions and methods of use | |
| US20150118257A1 (en) | Methods and Compositions for Manipulating the Immune System | |
| WO2023198663A1 (en) | Nucleic acid regulation of snca | |
| Geng et al. | Construction and application of a novel hepatocyte‐directed vector to simultaneous knockdown and overexpression of multiple genes | |
| WO2018130518A1 (en) | Methods and pharmaceutical composition for inducing senescence in cancer cells |