SG11201808176TA - Gene therapy for treating mucopolysaccharidosis type ii - Google Patents
Gene therapy for treating mucopolysaccharidosis type iiInfo
- Publication number
- SG11201808176TA SG11201808176TA SG11201808176TA SG11201808176TA SG11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- philadelphia
- pct
- street
- treating
- Prior art date
Links
- 201000002273 mucopolysaccharidosis II Diseases 0.000 title abstract 4
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 title abstract 4
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 235000014036 Castanea Nutrition 0.000 abstract 2
- 241001070941 Castanea Species 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 230000001404 mediated effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 239000000725 suspension Substances 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
- 238000012385 systemic delivery Methods 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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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
- A61K48/005—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
- A61K9/10—Dispersions; Emulsions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
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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
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6813—Hybridisation assays
- C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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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
- 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
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Analytical Chemistry (AREA)
- Dispersion Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662323194P | 2016-04-15 | 2016-04-15 | |
US201662330938P | 2016-05-03 | 2016-05-03 | |
US201662337163P | 2016-05-16 | 2016-05-16 | |
US201662367780P | 2016-07-28 | 2016-07-28 | |
US201762452494P | 2017-01-31 | 2017-01-31 | |
PCT/US2017/027770 WO2017181113A1 (en) | 2016-04-15 | 2017-04-14 | Gene therapy for treating mucopolysaccharidosis type ii |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201808176TA true SG11201808176TA (en) | 2018-10-30 |
Family
ID=60042827
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201808176TA SG11201808176TA (en) | 2016-04-15 | 2017-04-14 | Gene therapy for treating mucopolysaccharidosis type ii |
SG10201912761UA SG10201912761UA (en) | 2016-04-15 | 2017-04-14 | Gene therapy for treating mucopolysaccharidosis type ii |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201912761UA SG10201912761UA (en) | 2016-04-15 | 2017-04-14 | Gene therapy for treating mucopolysaccharidosis type ii |
Country Status (11)
Country | Link |
---|---|
US (2) | US11253612B2 (ko) |
EP (1) | EP3452104A1 (ko) |
JP (2) | JP7171439B2 (ko) |
KR (2) | KR20230125339A (ko) |
AU (1) | AU2017250298A1 (ko) |
BR (1) | BR112018071156A2 (ko) |
CA (1) | CA3019427A1 (ko) |
IL (2) | IL305449A (ko) |
MA (1) | MA44874A (ko) |
SG (2) | SG11201808176TA (ko) |
WO (1) | WO2017181113A1 (ko) |
Families Citing this family (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL307936A (en) | 2017-04-14 | 2023-12-01 | Regenxbio Inc | Treatment for mucopolysaccharidosis 2 with the help of recombinant human iduronate-2 suplatase (ids) produced by human neuron or glial cells |
BR112020005249A2 (pt) * | 2017-09-22 | 2020-09-24 | The Trustees Of The University Of Pennsylvania | terapia de gene para tratar mucopolissacaridose tipo ii |
JP2021500857A (ja) | 2017-10-02 | 2021-01-14 | デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. | 酵素補充療法用酵素を含む融合タンパク質 |
CA3082136A1 (en) * | 2017-11-08 | 2019-05-16 | Avexis, Inc. | Means and method for preparing viral vectors and uses of same |
KR20220112283A (ko) | 2019-12-10 | 2022-08-10 | 다케다 야쿠힌 고교 가부시키가이샤 | Hunter 질환 치료용 아데노-연합된 바이러스 벡터 |
US20230088992A1 (en) | 2020-01-29 | 2023-03-23 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells |
AU2021252515A1 (en) * | 2020-04-06 | 2022-10-27 | Homology Medicines, Inc. | Adeno-associated virus compositions for IDS gene transfer and methods of use thereof |
US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2022040530A2 (en) * | 2020-08-21 | 2022-02-24 | Capsida, Inc. | Virus compositions for treating mucopolysaccharidosis ii |
KR20230145386A (ko) | 2021-02-10 | 2023-10-17 | 리젠엑스바이오 인크. | 재조합 인간 이두로네이트-2-설파타아제(ids)를 사용한 점액다당류증 ii의 치료 |
WO2022212616A1 (en) * | 2021-04-01 | 2022-10-06 | The University Of North Carolina At Chapel Hill | Aav-ids vectors for treatment of mucopolysaccharidosis ii |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2023137233A2 (en) * | 2022-01-17 | 2023-07-20 | Danmarks Tekniske Universitet | Compositions and methods for editing genomes |
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- 2017-04-14 IL IL305449A patent/IL305449A/en unknown
- 2017-04-14 BR BR112018071156-0A patent/BR112018071156A2/pt unknown
- 2017-04-14 US US16/093,413 patent/US11253612B2/en active Active
- 2017-04-14 CA CA3019427A patent/CA3019427A1/en active Pending
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SG10201912761UA (en) | 2020-02-27 |
EP3452104A1 (en) | 2019-03-13 |
US11253612B2 (en) | 2022-02-22 |
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IL262211B2 (en) | 2024-01-01 |
BR112018071156A2 (pt) | 2019-03-12 |
JP2023011821A (ja) | 2023-01-24 |
IL262211B1 (en) | 2023-09-01 |
CA3019427A1 (en) | 2017-10-19 |
KR20230125339A (ko) | 2023-08-29 |
JP2019518002A (ja) | 2019-06-27 |
MA44874A (fr) | 2019-03-13 |
AU2017250298A1 (en) | 2018-10-11 |
IL305449A (en) | 2023-10-01 |
US20220125949A1 (en) | 2022-04-28 |
JP7171439B2 (ja) | 2022-11-15 |
KR20190008237A (ko) | 2019-01-23 |
IL262211A (en) | 2018-11-29 |
US20190070311A1 (en) | 2019-03-07 |
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