PT3898981T - Métodos e composições para tratar doenças de armazenamento de glicogénio - Google Patents
Métodos e composições para tratar doenças de armazenamento de glicogénioInfo
- Publication number
- PT3898981T PT3898981T PT198457897T PT19845789T PT3898981T PT 3898981 T PT3898981 T PT 3898981T PT 198457897 T PT198457897 T PT 198457897T PT 19845789 T PT19845789 T PT 19845789T PT 3898981 T PT3898981 T PT 3898981T
- Authority
- PT
- Portugal
- Prior art keywords
- compositions
- methods
- storage diseases
- glycogen storage
- treating glycogen
- 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
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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
- 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
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/34—Vector systems having a special element relevant for transcription being a transcription initiation element
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862781380P | 2018-12-18 | 2018-12-18 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3898981T true PT3898981T (pt) | 2023-12-28 |
Family
ID=69400618
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT198457897T PT3898981T (pt) | 2018-12-18 | 2019-12-18 | Métodos e composições para tratar doenças de armazenamento de glicogénio |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US12692512B2 (enExample) |
| EP (1) | EP3898981B1 (enExample) |
| JP (1) | JP7486274B2 (enExample) |
| KR (1) | KR102965926B1 (enExample) |
| CN (1) | CN113454226B (enExample) |
| AU (1) | AU2019405758B2 (enExample) |
| BR (1) | BR112021012104A2 (enExample) |
| CA (1) | CA3123841A1 (enExample) |
| DK (1) | DK3898981T5 (enExample) |
| ES (1) | ES2969222T3 (enExample) |
| IL (1) | IL283956A (enExample) |
| MX (1) | MX2021007379A (enExample) |
| PL (1) | PL3898981T3 (enExample) |
| PT (1) | PT3898981T (enExample) |
| WO (1) | WO2020132115A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023069891A1 (en) * | 2021-10-19 | 2023-04-27 | University Of Connecticut | Compositions and methods for the treatment of glycogen storage disorders |
Family Cites Families (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5387484A (en) | 1992-07-07 | 1995-02-07 | International Business Machines Corporation | Two-sided mask for patterning of materials with electromagnetic radiation |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5872005A (en) | 1994-11-03 | 1999-02-16 | Cell Genesys Inc. | Packaging cell lines for adeno-associated viral vectors |
| US6924128B2 (en) | 1994-12-06 | 2005-08-02 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| IL116816A (en) | 1995-01-20 | 2003-05-29 | Rhone Poulenc Rorer Sa | Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof |
| US5688676A (en) | 1995-06-07 | 1997-11-18 | Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| 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 |
| US6027931A (en) | 1995-08-03 | 2000-02-22 | Avigen, Inc. | High-efficiency AA V helper functions |
| US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| CA2270285A1 (en) | 1996-12-18 | 1998-06-25 | Targeted Genetics Corporation | Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999014354A1 (en) | 1997-09-19 | 1999-03-25 | The Trustees Of The University Of The Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| IT1297074B1 (it) | 1997-11-21 | 1999-08-03 | Angeletti P Ist Richerche Bio | Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| DE19905501B4 (de) | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
| JP2002191363A (ja) * | 1999-07-29 | 2002-07-09 | Herikkusu Kenkyusho:Kk | 全長ｃＤＮＡ合成用プライマー、およびその用途 |
| US20040235173A1 (en) | 2000-07-03 | 2004-11-25 | Gala Design, Inc. | Production of host cells containing multiple integrating vectors by serial transduction |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| EP1572893B1 (en) | 2001-11-09 | 2009-01-07 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES | Production of adeno-associated virus in insect cells |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| CA2369985A1 (en) | 2002-01-18 | 2003-07-18 | Duke University | Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| JP4559429B2 (ja) | 2003-05-21 | 2010-10-06 | ジェンザイム・コーポレーション | 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法 |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| US7195394B2 (en) | 2004-07-19 | 2007-03-27 | Vijay Singh | Method for resonant wave mixing in closed containers |
| US8980247B2 (en) | 2004-10-21 | 2015-03-17 | The Penn State Research Foundation | Parvovirus methods and compositions for killing neoplastic cells |
| PT3272872T (pt) | 2005-10-20 | 2020-06-26 | Uniqure Ip Bv | Vetores aav melhorados produzidos em células de insetos |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| GB0706638D0 (en) | 2007-04-04 | 2007-05-16 | Mbda Uk Ltd | A high-dielectric material |
| EP2242840B1 (en) | 2008-01-29 | 2019-07-24 | Applied Genetic Technologies Corporation | Recombinant adeno-associated virus production using bhk cells in suspension |
| US8580755B2 (en) | 2008-02-19 | 2013-11-12 | University Of Rochester | Methods and compositions for treating inflammatory conditions |
| US20110251547A1 (en) | 2008-11-07 | 2011-10-13 | Intelligentnano Inc. | Transfection with magnetic nanoparticles and ultrasound |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| JP5956331B2 (ja) | 2009-06-16 | 2016-07-27 | ジェンザイム・コーポレーション | 組換えａａｖベクターの改良された精製方法 |
| EP2529020B1 (en) | 2010-01-28 | 2018-04-25 | The Children's Hospital of Philadelphia | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| US20140359799A1 (en) | 2011-12-23 | 2014-12-04 | Case Western Reserve University | Targeted gene modification using hybrid recombinant adeno-associated virus |
| DK2943567T3 (en) | 2013-01-08 | 2018-01-02 | Genzyme Corp | USE OF INOS INHIBITORS TO INCREASE VIRAL YIELD IN CULTURE |
| AU2014216160B2 (en) * | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
| JP6649265B2 (ja) | 2013-11-26 | 2020-02-19 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 糖原病の処置のためのアデノ随伴ウイルスベクター |
| EP3236984B1 (en) | 2014-12-23 | 2020-07-22 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Adeno-associated virus vectors encoding modified g6pc and uses thereof |
| ES2938833T3 (es) | 2016-03-28 | 2023-04-17 | Ultragenyx Pharmaceutical Inc | Métodos de inactivación térmica de adenovirus |
| EP3484493A4 (en) | 2016-07-12 | 2019-12-25 | Ultragenyx Pharmaceutical Inc. | USE OF GLUCOCORTICOID ANALOGS TO INCREASE THE PERFORMANCE OF RECOMBINANT ADENO-ASSOCIATED VIRUSES |
| ES3012649T3 (en) | 2016-10-14 | 2025-04-09 | Ultragenyx Pharmaceutical Inc | Use of tonicifying agents to enhance recombinant adeno-associated virus yield |
| AU2018212002A1 (en) * | 2017-01-30 | 2019-08-29 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
| US20200048641A1 (en) | 2017-03-22 | 2020-02-13 | Ultragenyx Pharmaceutical Inc. | Methods for enhancing yield of recombinant adeno-associated virus |
| EP3601581B1 (en) | 2017-03-22 | 2025-04-30 | Ultragenyx Pharmaceutical Inc. | CELL CULTURE METHODS INVOLVING HDAC INHIBITORS OR REP PROTEINS |
| EP3621734A4 (en) | 2017-05-09 | 2020-12-30 | Ultragenyx Pharmaceutical Inc. | MODULAR PROCESS FOR THE PRODUCTION OF TRANSFECTION REAGENTS |
-
2019
- 2019-12-18 CA CA3123841A patent/CA3123841A1/en active Pending
- 2019-12-18 WO PCT/US2019/067247 patent/WO2020132115A1/en not_active Ceased
- 2019-12-18 KR KR1020217022096A patent/KR102965926B1/ko active Active
- 2019-12-18 JP JP2021534702A patent/JP7486274B2/ja active Active
- 2019-12-18 PL PL19845789.7T patent/PL3898981T3/pl unknown
- 2019-12-18 ES ES19845789T patent/ES2969222T3/es active Active
- 2019-12-18 EP EP19845789.7A patent/EP3898981B1/en active Active
- 2019-12-18 DK DK19845789.7T patent/DK3898981T5/da active
- 2019-12-18 PT PT198457897T patent/PT3898981T/pt unknown
- 2019-12-18 BR BR112021012104-8A patent/BR112021012104A2/pt unknown
- 2019-12-18 MX MX2021007379A patent/MX2021007379A/es unknown
- 2019-12-18 US US17/311,869 patent/US12692512B2/en active Active
- 2019-12-18 AU AU2019405758A patent/AU2019405758B2/en active Active
- 2019-12-18 CN CN201980083410.8A patent/CN113454226B/zh active Active
-
2021
- 2021-06-13 IL IL283956A patent/IL283956A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2019405758B2 (en) | 2026-02-05 |
| AU2019405758A2 (en) | 2022-05-12 |
| CN113454226B (zh) | 2025-10-31 |
| WO2020132115A1 (en) | 2020-06-25 |
| MX2021007379A (es) | 2021-07-15 |
| KR102965926B1 (ko) | 2026-05-18 |
| CA3123841A1 (en) | 2020-06-25 |
| AU2019405758A1 (en) | 2021-07-01 |
| KR20210105390A (ko) | 2021-08-26 |
| BR112021012104A2 (pt) | 2021-09-08 |
| US20220017922A1 (en) | 2022-01-20 |
| IL283956A (en) | 2021-07-29 |
| JP2022514271A (ja) | 2022-02-10 |
| DK3898981T5 (da) | 2024-08-05 |
| PL3898981T3 (pl) | 2024-04-02 |
| ES2969222T3 (es) | 2024-05-17 |
| DK3898981T3 (da) | 2024-01-22 |
| EP3898981A1 (en) | 2021-10-27 |
| JP7486274B2 (ja) | 2024-05-17 |
| WO2020132115A8 (en) | 2021-05-14 |
| EP3898981B1 (en) | 2023-10-18 |
| US12692512B2 (en) | 2026-07-28 |
| CN113454226A (zh) | 2021-09-28 |
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