SG11201803353PA - Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy - Google Patents
Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathyInfo
- Publication number
- SG11201803353PA SG11201803353PA SG11201803353PA SG11201803353PA SG11201803353PA SG 11201803353P A SG11201803353P A SG 11201803353PA SG 11201803353P A SG11201803353P A SG 11201803353PA SG 11201803353P A SG11201803353P A SG 11201803353PA SG 11201803353P A SG11201803353P A SG 11201803353PA
- Authority
- SG
- Singapore
- Prior art keywords
- adrenomyeloneuropathy
- treatment
- nucleic acid
- acid sequences
- sequences encoding
- Prior art date
Links
- 201000011452 Adrenoleukodystrophy Diseases 0.000 title 1
- 208000018126 Adrenomyeloneuropathy Diseases 0.000 title 1
- 238000007913 intrathecal administration Methods 0.000 title 1
- 150000007523 nucleic acids Chemical group 0.000 title 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
- C12N7/02—Recovery or purification
-
- 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
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- 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
- 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
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- 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
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- 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
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- 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
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—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
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562251208P | 2015-11-05 | 2015-11-05 | |
US201662300691P | 2016-02-26 | 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 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201803353PA true SG11201803353PA (en) | 2018-05-30 |
Family
ID=58662371
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201803353PA SG11201803353PA (en) | 2015-11-05 | 2016-11-03 | Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy |
Country Status (15)
Country | Link |
---|---|
US (3) | US20180320176A1 (en) |
EP (2) | EP3370705B1 (en) |
JP (2) | JP7037490B2 (en) |
KR (1) | KR20180066252A (en) |
CN (1) | CN108472264A (en) |
AU (1) | AU2016349381B2 (en) |
BR (1) | BR112018008993A2 (en) |
CA (1) | CA3003747A1 (en) |
ES (1) | ES2916325T3 (en) |
IL (1) | IL258904B (en) |
MX (1) | MX2018005689A (en) |
PL (1) | PL3370705T3 (en) |
RU (2) | RU2739384C2 (en) |
SG (1) | SG11201803353PA (en) |
WO (1) | WO2017079467A1 (en) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MX2022008201A (en) | 2019-12-31 | 2022-10-18 | Swanbio Therapeutics Ltd | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn). |
Family Cites Families (31)
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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 |
ES2246004T3 (en) | 1996-02-02 | 2006-02-01 | Alza Corporation | PROLONGED RELEASE 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 |
US6261584B1 (en) | 1996-02-02 | 2001-07-17 | Alza Corporation | Sustained delivery of an active agent using an implantable system |
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 |
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 |
DK1041975T3 (en) | 1997-12-22 | 2002-11-04 | Alza Corp | Speed-controlling membranes for controlled drug delivery devices |
WO1999033446A1 (en) | 1997-12-29 | 1999-07-08 | Alza Corporation | Osmotic delivery system with membrane plug retention mechanism |
CA2316886C (en) | 1997-12-30 | 2007-09-25 | Alza Corporation | Beneficial agent delivery system with membrane plug |
EP1140014B1 (en) | 1998-12-31 | 2003-03-19 | Alza Corporation | 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 |
DK1328256T3 (en) | 1999-12-21 | 2006-02-20 | Alza Corp | Valve for osmotic devices |
US7163688B2 (en) | 2001-06-22 | 2007-01-16 | Alza Corporation | Osmotic implant with membrane and membrane retention means |
EP1402043A1 (en) * | 2001-07-03 | 2004-03-31 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
NZ537628A (en) | 2002-06-26 | 2005-12-23 | Alza Corp | 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 |
NZ542779A (en) | 2003-03-31 | 2009-02-28 | Intarcia Therapeutics Inc | Osmotic pump with means for dissipating internal pressure by allowing osmotic material to be ejected from the container above a set internal pressure |
US20050175701A1 (en) | 2004-02-10 | 2005-08-11 | Alza Corporation | Capillary moderator for osmotic delivery system |
KR101106510B1 (en) | 2006-05-30 | 2012-01-20 | 인타르시아 세라퓨틱스 인코포레이티드 | Two-piece, internal-channel osmotic delivery system flow modulator |
KR101200728B1 (en) | 2006-08-09 | 2012-11-13 | 인타르시아 세라퓨틱스 인코포레이티드 | Osmotic delivery system and piston assemblies |
WO2011106376A2 (en) * | 2010-02-23 | 2011-09-01 | The General Hospital Corporation | Use of microvesicles in the treatment of medical conditions |
PT2717922T (en) * | 2011-06-10 | 2018-12-18 | Bluebird Bio Inc | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
JP2015516143A (en) * | 2012-04-02 | 2015-06-08 | モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. | Modified polynucleotides for the production of proteins associated with human disease |
CA3209821A1 (en) * | 2013-05-15 | 2014-11-20 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
GB201401707D0 (en) * | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
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2016
- 2016-11-03 MX MX2018005689A patent/MX2018005689A/en unknown
- 2016-11-03 RU RU2018120484A patent/RU2739384C2/en active
- 2016-11-03 KR KR1020187015254A patent/KR20180066252A/en active Search and Examination
- 2016-11-03 PL PL16862990.5T patent/PL3370705T3/en unknown
- 2016-11-03 CA CA3003747A patent/CA3003747A1/en active Pending
- 2016-11-03 IL IL258904A patent/IL258904B/en unknown
- 2016-11-03 RU RU2020141860A patent/RU2020141860A/en unknown
- 2016-11-03 JP JP2018543041A patent/JP7037490B2/en active Active
- 2016-11-03 AU AU2016349381A patent/AU2016349381B2/en active Active
- 2016-11-03 CN CN201680077741.7A patent/CN108472264A/en active Pending
- 2016-11-03 ES ES16862990T patent/ES2916325T3/en active Active
- 2016-11-03 WO PCT/US2016/060375 patent/WO2017079467A1/en active Application Filing
- 2016-11-03 SG SG11201803353PA patent/SG11201803353PA/en unknown
- 2016-11-03 US US15/773,337 patent/US20180320176A1/en not_active Abandoned
- 2016-11-03 EP EP16862990.5A patent/EP3370705B1/en active Active
- 2016-11-03 BR BR112018008993A patent/BR112018008993A2/en active Search and Examination
- 2016-11-03 EP EP22159649.7A patent/EP4066862A1/en not_active Withdrawn
-
2019
- 2019-04-02 US US16/373,081 patent/US10519445B2/en active Active
-
2022
- 2022-01-17 JP JP2022005016A patent/JP2022064910A/en active Pending
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2023
- 2023-04-04 US US18/295,566 patent/US20230365967A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
RU2739384C2 (en) | 2020-12-23 |
IL258904B (en) | 2022-09-01 |
ES2916325T3 (en) | 2022-06-30 |
EP3370705B1 (en) | 2022-03-09 |
JP7037490B2 (en) | 2022-03-16 |
US20190225967A1 (en) | 2019-07-25 |
MX2018005689A (en) | 2018-08-01 |
AU2016349381B2 (en) | 2023-02-23 |
WO2017079467A1 (en) | 2017-05-11 |
KR20180066252A (en) | 2018-06-18 |
EP4066862A1 (en) | 2022-10-05 |
CN108472264A (en) | 2018-08-31 |
CA3003747A1 (en) | 2017-05-11 |
US20230365967A1 (en) | 2023-11-16 |
JP2022064910A (en) | 2022-04-26 |
US20180320176A1 (en) | 2018-11-08 |
RU2020141860A (en) | 2021-04-01 |
EP3370705A4 (en) | 2019-05-15 |
IL258904A (en) | 2018-06-28 |
JP2018537124A (en) | 2018-12-20 |
BR112018008993A2 (en) | 2018-10-30 |
US10519445B2 (en) | 2019-12-31 |
EP3370705A1 (en) | 2018-09-12 |
RU2018120484A (en) | 2019-12-05 |
PL3370705T3 (en) | 2022-08-01 |
AU2016349381A1 (en) | 2018-06-21 |
RU2018120484A3 (en) | 2020-03-11 |
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