SG10201912401QA - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents
Gene therapy for neuronal ceroid lipofuscinosesInfo
- Publication number
- SG10201912401QA SG10201912401QA SG10201912401QA SG10201912401QA SG10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA
- Authority
- SG
- Singapore
- Prior art keywords
- gene therapy
- neuronal ceroid
- ceroid lipofuscinoses
- lipofuscinoses
- neuronal
- Prior art date
Links
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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
-
- 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/485—Exopeptidases (3.4.11-3.4.19)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16141—Use of virus, viral particle or viral elements as a vector
- C12N2710/16143—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
- C12N2750/14141—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
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dispersion Chemistry (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762504817P | 2017-05-11 | 2017-05-11 | |
US201762599816P | 2017-12-18 | 2017-12-18 | |
US201862652006P | 2018-04-03 | 2018-04-03 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG10201912401QA true SG10201912401QA (en) | 2020-02-27 |
Family
ID=64105028
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201912401QA SG10201912401QA (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
SG11201910015S SG11201910015SA (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201910015S SG11201910015SA (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Country Status (10)
Country | Link |
---|---|
US (2) | US11591614B2 (en) |
EP (1) | EP3621612A4 (en) |
JP (2) | JP7273730B2 (en) |
KR (1) | KR20200023280A (en) |
AU (1) | AU2018265531A1 (en) |
BR (1) | BR112019023303A2 (en) |
CA (1) | CA3061655A1 (en) |
RU (1) | RU2019139555A (en) |
SG (2) | SG10201912401QA (en) |
WO (1) | WO2018209205A1 (en) |
Families Citing this family (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2017355502B2 (en) * | 2016-11-04 | 2023-08-31 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
KR20200104852A (en) | 2017-09-22 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Gene therapy for the treatment of type II mucopolysaccharides |
SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
KR20210148333A (en) * | 2019-04-08 | 2021-12-07 | 더 칠드런스 호스피탈 오브 필라델피아 | Treatment of ocular lysosomal storage diseases through administration of AAV expressing TPP1 |
US20220168449A1 (en) * | 2019-04-12 | 2022-06-02 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
MX2022014258A (en) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression. |
MX2023008826A (en) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses. |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
Family Cites Families (43)
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US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
ES2563643T3 (en) | 1997-04-01 | 2016-03-15 | Illumina Cambridge Limited | Nucleic acid sequencing method |
DE69824859T2 (en) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | METHODS OF INCREASING THE EFFICIENCY OF RECOMBINANT AAV PRODUCTS |
US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
ATE454445T1 (en) | 1998-11-10 | 2010-01-15 | Univ North Carolina | VIRUS VECTORS AND METHODS FOR THEIR PRODUCTION AND ADMINISTRATION. |
ES2308989T3 (en) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | INCREASE IN THE EXPRESSION OF A NUCLEOTIDIC SEQUENCE HETEROLOGY FROM RECOMBINANT VIRECT VECTORS CONTAINING A SEQUENCE FORMING INTRACATENARY BASE PAIRS. |
AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
CA2447240C (en) | 2001-05-18 | 2013-02-19 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
CA2945734C (en) | 2001-11-13 | 2020-02-25 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
WO2005013090A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
CA2939103C (en) | 2003-09-30 | 2020-03-24 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
ES2428218T3 (en) | 2005-04-07 | 2013-11-06 | The Trustees Of The University Of Pennsylvania | AAV rh48 modified capsules, compositions containing them and uses thereof |
JP4495210B2 (en) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | Amplitude error compensator and orthogonality error compensator |
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 |
AU2009311667B2 (en) | 2008-11-07 | 2016-04-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
EP4043025A1 (en) | 2011-06-08 | 2022-08-17 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
EP2833923A4 (en) | 2012-04-02 | 2016-02-24 | Moderna Therapeutics Inc | Modified polynucleotides for the production of proteins |
KR20150020288A (en) | 2012-06-08 | 2015-02-25 | 에트리스 게엠베하 | Pulmonary delivery of messenger rna |
WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
BR112016001592A2 (en) | 2013-07-26 | 2017-11-28 | Univ Iowa Res Found | methods and compositions for the treatment of brain diseases |
US10780182B2 (en) | 2014-04-25 | 2020-09-22 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
KR20230067694A (en) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
EP3364970B1 (en) | 2015-10-23 | 2024-02-07 | University of Iowa Research Foundation | Gene therapy for use in treating lysosomal storage disease |
WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
JP7360241B2 (en) | 2016-02-03 | 2023-10-12 | 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 | Gene therapy to treat mucopolysaccharidosis type I |
CN110741082A (en) | 2017-02-28 | 2020-01-31 | 宾夕法尼亚州大学信托人 | Adeno-associated virus (AAV) clade F vectors and uses thereof |
-
2018
- 2018-05-11 SG SG10201912401QA patent/SG10201912401QA/en unknown
- 2018-05-11 BR BR112019023303-2A patent/BR112019023303A2/en active Search and Examination
- 2018-05-11 CA CA3061655A patent/CA3061655A1/en active Pending
- 2018-05-11 WO PCT/US2018/032278 patent/WO2018209205A1/en active Application Filing
- 2018-05-11 RU RU2019139555A patent/RU2019139555A/en unknown
- 2018-05-11 JP JP2019562418A patent/JP7273730B2/en active Active
- 2018-05-11 US US16/611,512 patent/US11591614B2/en active Active
- 2018-05-11 EP EP18798476.0A patent/EP3621612A4/en active Pending
- 2018-05-11 KR KR1020197036041A patent/KR20200023280A/en not_active Application Discontinuation
- 2018-05-11 AU AU2018265531A patent/AU2018265531A1/en active Pending
- 2018-05-11 SG SG11201910015S patent/SG11201910015SA/en unknown
-
2022
- 2022-12-29 US US18/148,077 patent/US20230383312A1/en active Pending
-
2023
- 2023-04-28 JP JP2023073941A patent/JP2023099113A/en active Pending
Also Published As
Publication number | Publication date |
---|---|
US11591614B2 (en) | 2023-02-28 |
RU2019139555A3 (en) | 2021-10-12 |
AU2018265531A1 (en) | 2019-11-14 |
EP3621612A4 (en) | 2021-06-16 |
KR20200023280A (en) | 2020-03-04 |
RU2019139555A (en) | 2021-06-11 |
EP3621612A1 (en) | 2020-03-18 |
BR112019023303A2 (en) | 2020-06-16 |
US20230383312A1 (en) | 2023-11-30 |
WO2018209205A9 (en) | 2019-11-21 |
US20200239908A1 (en) | 2020-07-30 |
SG11201910015SA (en) | 2019-11-28 |
JP7273730B2 (en) | 2023-05-15 |
JP2020519292A (en) | 2020-07-02 |
JP2023099113A (en) | 2023-07-11 |
WO2018209205A1 (en) | 2018-11-15 |
CA3061655A1 (en) | 2018-11-15 |
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