SG11201910015SA - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents
Gene therapy for neuronal ceroid lipofuscinosesInfo
- Publication number
- SG11201910015SA SG11201910015SA SG11201910015SA SG11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- street
- philadelphia
- pct
- aav
- Prior art date
Links
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 4
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 abstract 2
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 208000031277 Amaurotic familial idiocy Diseases 0.000 abstract 1
- 235000014036 Castanea Nutrition 0.000 abstract 1
- 241001070941 Castanea Species 0.000 abstract 1
- 108091026890 Coding region Proteins 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 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
- 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
- A61K48/005—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
- A61K9/0012—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
-
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Dispersion Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (4)
| 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 | |
| PCT/US2018/032278 WO2018209205A1 (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201910015SA true SG11201910015SA (en) | 2019-11-28 |
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 Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201912401QA SG10201912401QA (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11591614B2 (enExample) |
| EP (1) | EP3621612A4 (enExample) |
| JP (2) | JP7273730B2 (enExample) |
| KR (1) | KR102719222B1 (enExample) |
| AU (1) | AU2018265531B2 (enExample) |
| BR (1) | BR112019023303A2 (enExample) |
| CA (1) | CA3061655A1 (enExample) |
| IL (2) | IL316926A (enExample) |
| RU (1) | RU2019139555A (enExample) |
| SG (2) | SG10201912401QA (enExample) |
| WO (1) | WO2018209205A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CN110198712A (zh) * | 2016-11-04 | 2019-09-03 | 费城儿童医院 | 用于治疗神经退行性疾病的基因转移组合物、方法和用途 |
| JP7449223B2 (ja) | 2017-09-22 | 2024-03-13 | 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 | ムコ多糖症ii型を治療するための遺伝子療法 |
| KR20210092755A (ko) * | 2018-11-14 | 2021-07-26 | 리젠엑스바이오 인크. | 신경원성 세로이드 리포푸신증에 대한 유전자 요법 |
| MX2021006253A (es) * | 2018-11-28 | 2021-09-21 | Prevail Therapeutics Inc | Terapias génicas para enfermedad neurodegenerativa. |
| WO2020210324A1 (en) * | 2019-04-08 | 2020-10-15 | The Children's Hospital Of Philadelphia | Treatment of lysosomal storage disease in the eye through administration of aavs expressing tpp1 |
| WO2020210633A1 (en) * | 2019-04-12 | 2020-10-15 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
| US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| IL300409A (en) * | 2020-08-10 | 2023-04-01 | Prevail Therapeutics Inc | Gene therapies for neurodegenerative disorders |
| MX2023008826A (es) * | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1998044151A1 (en) | 1997-04-01 | 1998-10-08 | Glaxo Group Limited | Method of nucleic acid amplification |
| NZ500546A (en) | 1997-04-14 | 2001-09-28 | Cell Genesys Inc | Methods for increasing the production of high titre stocks of recombinant adeno-associated virus (AAV) |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| JP2002538770A (ja) | 1998-11-10 | 2002-11-19 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ウイルスベクターとその製造及び投与の方法 |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand 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 |
| ATE403013T1 (de) | 2001-05-18 | 2008-08-15 | Wisconsin Alumni Res Found | Verfahren zur synthese von dna-sequenzen die photolabile linker verwenden |
| ES2258601T3 (es) | 2001-11-13 | 2006-09-01 | The Trustees Of The University Of Pennsylvania | Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo. |
| 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 |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | 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 |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| US20080050357A1 (en) | 2003-08-01 | 2008-02-28 | Claes Gustafsson | Systems and Methods for Antibody Engineering |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP4234687A2 (en) | 2005-04-07 | 2023-08-30 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| CA3006395C (en) | 2008-11-07 | 2022-05-31 | 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 |
| US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| WO2012135857A1 (en) | 2011-03-31 | 2012-10-04 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| LT2717893T (lt) | 2011-06-08 | 2019-08-12 | Translate Bio, Inc. | Lipidų nanodalelių kompozicijos ir mrnr pristatymo būdai |
| EP2834260A4 (en) | 2012-04-02 | 2016-08-10 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | 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 |
| WO2015013148A2 (en) * | 2013-07-26 | 2015-01-29 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| EP3800260A1 (en) | 2014-09-24 | 2021-04-07 | City of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| BR112018008069A2 (pt) * | 2015-10-23 | 2018-11-13 | Univ Iowa Res Found | métodos de tratamento de doenças neurodegenerati-vas usando terapia gênica para retardar o início e progressão da doença ao mesmo tempo em que fornece proteção cognitiva |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| FI3589730T3 (fi) | 2017-02-28 | 2024-02-22 | Univ Pennsylvania | Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja |
-
2018
- 2018-05-11 KR KR1020197036041A patent/KR102719222B1/ko active Active
- 2018-05-11 JP JP2019562418A patent/JP7273730B2/ja active Active
- 2018-05-11 SG SG10201912401QA patent/SG10201912401QA/en unknown
- 2018-05-11 RU RU2019139555A patent/RU2019139555A/ru unknown
- 2018-05-11 SG SG11201910015S patent/SG11201910015SA/en unknown
- 2018-05-11 EP EP18798476.0A patent/EP3621612A4/en active Pending
- 2018-05-11 WO PCT/US2018/032278 patent/WO2018209205A1/en not_active Ceased
- 2018-05-11 IL IL316926A patent/IL316926A/en unknown
- 2018-05-11 CA CA3061655A patent/CA3061655A1/en active Pending
- 2018-05-11 BR BR112019023303-2A patent/BR112019023303A2/pt active Search and Examination
- 2018-05-11 IL IL270422A patent/IL270422B2/en unknown
- 2018-05-11 US US16/611,512 patent/US11591614B2/en active Active
- 2018-05-11 AU AU2018265531A patent/AU2018265531B2/en active Active
-
2022
- 2022-12-29 US US18/148,077 patent/US12428653B2/en active Active
-
2023
- 2023-04-28 JP JP2023073941A patent/JP2023099113A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| RU2019139555A3 (enExample) | 2021-10-12 |
| IL316926A (en) | 2025-01-01 |
| AU2018265531A1 (en) | 2019-11-14 |
| EP3621612A4 (en) | 2021-06-16 |
| US20230383312A1 (en) | 2023-11-30 |
| AU2018265531B2 (en) | 2024-07-11 |
| CA3061655A1 (en) | 2018-11-15 |
| KR20200023280A (ko) | 2020-03-04 |
| IL270422B1 (en) | 2024-12-01 |
| SG10201912401QA (en) | 2020-02-27 |
| BR112019023303A2 (pt) | 2020-06-16 |
| KR102719222B1 (ko) | 2024-10-21 |
| IL270422A (en) | 2020-01-30 |
| EP3621612A1 (en) | 2020-03-18 |
| WO2018209205A9 (en) | 2019-11-21 |
| JP2020519292A (ja) | 2020-07-02 |
| JP7273730B2 (ja) | 2023-05-15 |
| US20200239908A1 (en) | 2020-07-30 |
| WO2018209205A1 (en) | 2018-11-15 |
| IL270422B2 (en) | 2025-04-01 |
| RU2019139555A (ru) | 2021-06-11 |
| JP2023099113A (ja) | 2023-07-11 |
| US12428653B2 (en) | 2025-09-30 |
| US11591614B2 (en) | 2023-02-28 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| SG11201910015SA (en) | Gene therapy for neuronal ceroid lipofuscinoses | |
| SG11201907714UA (en) | Adeno-associated virus (aav) clade f vector and uses therefor | |
| SG11201811432WA (en) | Rna for cancer therapy | |
| SG11201907474UA (en) | Compositions useful in treatment of spinal muscular atrophy | |
| SG11201809029RA (en) | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy | |
| SG11201908575SA (en) | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy | |
| SG11201909949XA (en) | Targeted immunotolerance | |
| SG11201811603WA (en) | Novel adeno-associated virus capsid proteins | |
| SG11201908023YA (en) | T cell receptors and immune therapy using the same against prame positive cancers | |
| SG11201809684YA (en) | Adeno-associated virus variant capsids and methods of use thereof | |
| SG11201811600PA (en) | Pseudotyped oncolytic viral delivery of therapeutic polypeptides | |
| SG11201906297QA (en) | Nucleic acids encoding crispr-associated proteins and uses thereof | |
| SG11201811338VA (en) | A novel botulinum neurotoxin and its derivatives | |
| SG11201809344QA (en) | Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde | |
| SG11201909777YA (en) | Modulatory polynucleotides | |
| SG11201906319XA (en) | Compositions and methods for the depletion of cd137+ cells | |
| SG11201810210UA (en) | Engineered botulinum neurotoxins | |
| SG11201804312RA (en) | Microbubble-chemotherapeutic agent complex for sonodynamic therapy | |
| SG11201804729RA (en) | Methods and compositions for treating a serpinc1-associated disorder | |
| SG11201901996UA (en) | Formulations of ( r)-2-amino-3-phenylpropyl carbamate | |
| SG11202000312UA (en) | Encapsulated polynucleotides and methods of use | |
| SG11201804673WA (en) | Novel anti-claudin antibodies and methods of use | |
| SG11201408679XA (en) | Methods for treating tyrosine-kinase-inhibitor-resistant malignancies in patients with genetic polymorphisms or ahi1 dysregulations or mutations employing dianhydrogalactitol, diacetyldianhydrogalactitol, dibromodulcitol, or analogs or derivatives thereof | |
| SG11201811244SA (en) | Dual overlapping adeno-associated viral vector system for expressing abc4a | |
| SG11201804070XA (en) | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |