CL2021002049A1 - Administración de virus adenoasociado de polinucleótido de cln3. - Google Patents
Administración de virus adenoasociado de polinucleótido de cln3.Info
- Publication number
- CL2021002049A1 CL2021002049A1 CL2021002049A CL2021002049A CL2021002049A1 CL 2021002049 A1 CL2021002049 A1 CL 2021002049A1 CL 2021002049 A CL2021002049 A CL 2021002049A CL 2021002049 A CL2021002049 A CL 2021002049A CL 2021002049 A1 CL2021002049 A1 CL 2021002049A1
- Authority
- CL
- Chile
- Prior art keywords
- cln3
- administration
- associated virus
- adeno
- polynucleotide
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 102000040430 polynucleotide Human genes 0.000 title abstract 2
- 108091033319 polynucleotide Proteins 0.000 title abstract 2
- 239000002157 polynucleotide Substances 0.000 title abstract 2
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 abstract 2
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 abstract 2
- 208000031277 Amaurotic familial idiocy Diseases 0.000 abstract 1
- 101150038645 CLN3 gene Proteins 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 abstract 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
-
- 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
- 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
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962800911P | 2019-02-04 | 2019-02-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021002049A1 true CL2021002049A1 (es) | 2022-04-01 |
Family
ID=69771089
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021002049A CL2021002049A1 (es) | 2019-02-04 | 2021-08-03 | Administración de virus adenoasociado de polinucleótido de cln3. |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US12404523B2 (https=) |
| EP (1) | EP3921430A1 (https=) |
| JP (2) | JP7691367B2 (https=) |
| KR (1) | KR20210124300A (https=) |
| CN (1) | CN113646436A (https=) |
| AR (1) | AR118005A1 (https=) |
| AU (1) | AU2020217708B2 (https=) |
| BR (1) | BR112021015285A2 (https=) |
| CA (1) | CA3129077A1 (https=) |
| CL (1) | CL2021002049A1 (https=) |
| EA (1) | EA202192160A1 (https=) |
| IL (1) | IL285328A (https=) |
| MX (1) | MX2021009401A (https=) |
| SG (1) | SG11202107986YA (https=) |
| TW (1) | TW202045530A (https=) |
| WO (1) | WO2020163300A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| WO2022170038A1 (en) * | 2021-02-05 | 2022-08-11 | Amicus Therapeutics, Inc. | Adeno-associated virus delivery of cln3 polynucleotide |
| JP2025524569A (ja) * | 2022-07-06 | 2025-07-30 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | 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 | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| WO2016100575A1 (en) * | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| JP7106534B2 (ja) * | 2016-11-17 | 2022-07-26 | ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド | メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 |
-
2020
- 2020-02-04 CA CA3129077A patent/CA3129077A1/en active Pending
- 2020-02-04 BR BR112021015285-7A patent/BR112021015285A2/pt unknown
- 2020-02-04 EA EA202192160A patent/EA202192160A1/ru unknown
- 2020-02-04 KR KR1020217027483A patent/KR20210124300A/ko active Pending
- 2020-02-04 EP EP20709850.0A patent/EP3921430A1/en active Pending
- 2020-02-04 US US17/427,010 patent/US12404523B2/en active Active
- 2020-02-04 WO PCT/US2020/016542 patent/WO2020163300A1/en not_active Ceased
- 2020-02-04 MX MX2021009401A patent/MX2021009401A/es unknown
- 2020-02-04 TW TW109103419A patent/TW202045530A/zh unknown
- 2020-02-04 JP JP2021545401A patent/JP7691367B2/ja active Active
- 2020-02-04 CN CN202080014802.1A patent/CN113646436A/zh active Pending
- 2020-02-04 AU AU2020217708A patent/AU2020217708B2/en active Active
- 2020-02-04 AR ARP200100305A patent/AR118005A1/es unknown
- 2020-02-04 SG SG11202107986YA patent/SG11202107986YA/en unknown
-
2021
- 2021-08-03 IL IL285328A patent/IL285328A/en unknown
- 2021-08-03 CL CL2021002049A patent/CL2021002049A1/es unknown
-
2025
- 2025-05-30 JP JP2025090921A patent/JP2025138648A/ja active Pending
- 2025-08-04 US US19/289,974 patent/US20260062713A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| TW202045530A (zh) | 2020-12-16 |
| CN113646436A (zh) | 2021-11-12 |
| EA202192160A1 (ru) | 2021-11-17 |
| US12404523B2 (en) | 2025-09-02 |
| KR20210124300A (ko) | 2021-10-14 |
| AR118005A1 (es) | 2021-09-08 |
| JP2025138648A (ja) | 2025-09-25 |
| WO2020163300A1 (en) | 2020-08-13 |
| AU2020217708B2 (en) | 2026-01-22 |
| SG11202107986YA (en) | 2021-08-30 |
| US20260062713A1 (en) | 2026-03-05 |
| EP3921430A1 (en) | 2021-12-15 |
| MX2021009401A (es) | 2021-11-12 |
| CA3129077A1 (en) | 2020-08-13 |
| BR112021015285A2 (pt) | 2021-10-05 |
| JP7691367B2 (ja) | 2025-06-11 |
| US20220127641A1 (en) | 2022-04-28 |
| IL285328A (en) | 2021-09-30 |
| AU2020217708A1 (en) | 2021-08-19 |
| WO2020163300A9 (en) | 2021-04-15 |
| JP2022519596A (ja) | 2022-03-24 |
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