IL282140A - Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy - Google Patents
Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapyInfo
- Publication number
- IL282140A IL282140A IL282140A IL28214021A IL282140A IL 282140 A IL282140 A IL 282140A IL 282140 A IL282140 A IL 282140A IL 28214021 A IL28214021 A IL 28214021A IL 282140 A IL282140 A IL 282140A
- Authority
- IL
- Israel
- Prior art keywords
- liver
- generation
- treatment
- replacement therapy
- gene replacement
- Prior art date
Links
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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/44—Oxidoreductases (1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/44—Oxidoreductases (1)
- A61K38/443—Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
- 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
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862744944P | 2018-10-12 | 2018-10-12 | |
| PCT/US2019/055917 WO2020077250A1 (en) | 2018-10-12 | 2019-10-11 | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL282140A true IL282140A (en) | 2021-05-31 |
Family
ID=68426839
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL282140A IL282140A (en) | 2018-10-12 | 2021-04-07 | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20210348135A1 (enExample) |
| EP (2) | EP3863662A1 (enExample) |
| JP (2) | JP2022504740A (enExample) |
| KR (1) | KR20210076051A (enExample) |
| CN (2) | CN120349985A (enExample) |
| AR (1) | AR116700A1 (enExample) |
| AU (2) | AU2019357039B2 (enExample) |
| BR (1) | BR112021006796A2 (enExample) |
| CA (1) | CA3116283A1 (enExample) |
| CO (1) | CO2021006060A2 (enExample) |
| IL (1) | IL282140A (enExample) |
| MX (2) | MX2021004213A (enExample) |
| PH (1) | PH12021550794A1 (enExample) |
| SG (1) | SG11202103614RA (enExample) |
| TW (1) | TW202035439A (enExample) |
| WO (1) | WO2020077250A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2020001593A (es) | 2017-08-09 | 2020-07-13 | Bioverativ Therapeutics Inc | Moleculas de acido nucleico y usos de las mismas. |
| US11939601B2 (en) | 2017-11-22 | 2024-03-26 | Modernatx, Inc. | Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria |
| JP7602454B2 (ja) | 2018-08-09 | 2024-12-18 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
| TW202140791A (zh) * | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| JP7721568B2 (ja) * | 2020-04-21 | 2025-08-12 | アレン インスティテュート | 新皮質層５グルタミン酸作動性ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物 |
| CA3177268A1 (en) * | 2020-05-05 | 2021-11-11 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus 5 capsids and uses thereof |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| AU2021285812A1 (en) * | 2020-06-01 | 2023-01-05 | Modernatx, Inc. | Phenylalanine hydroxylase variants and uses thereof |
| MX2023003019A (es) * | 2020-09-16 | 2023-05-08 | Generation Bio Co | Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah). |
| CN116648503A (zh) * | 2020-10-01 | 2023-08-25 | 建新公司 | 通过肝导向基因替代疗法治疗pku的人pah表达盒 |
| KR20230079172A (ko) | 2020-10-01 | 2023-06-05 | 젠자임 코포레이션 | 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 |
| US20230084036A1 (en) * | 2021-08-23 | 2023-03-16 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| CN115572730B (zh) * | 2022-04-24 | 2026-07-17 | 复旦大学附属中山医院 | 一种gtf2h4高表达的内皮细胞系 |
| CN115772503B (zh) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | 一种表达pah的基因修饰细胞药物及其制备方法与应用 |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| CN117899238A (zh) * | 2023-12-07 | 2024-04-19 | 哈尔滨工业大学 | Pglyrp2基因的用途及其相关药物 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| WO2002092134A1 (en) * | 2001-05-14 | 2002-11-21 | Cell Genesys, Inc. | Lentiviral vectors encoding clotting factors for gene therapy |
| 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 |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| 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 |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8440182B2 (en) * | 2007-05-24 | 2013-05-14 | The Board Of Regents Of The University Of Texas System | Methods and compositions for treating phenylketonuria |
| US20100316623A1 (en) * | 2009-04-23 | 2010-12-16 | Andrew Turner | Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria |
| 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 |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CN103502458B (zh) | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| NO2718319T3 (enExample) * | 2011-06-07 | 2018-05-05 | ||
| FR2978457A1 (fr) * | 2011-07-27 | 2013-02-01 | Univ Paris Diderot Paris 7 | Composes contenant un vecteur viral a specificite hepatique et presentant une activite anti-inflammatoire |
| ES2813698T3 (es) * | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| WO2016037931A1 (en) * | 2014-09-11 | 2016-03-17 | Fundación Para La Investigación Médica Aplicada | Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases |
| CA3255657A1 (en) * | 2015-02-10 | 2025-12-01 | Genzyme Corporation | VARIANT RNAi |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CN115094062A (zh) * | 2015-04-16 | 2022-09-23 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| PE20181922A1 (es) * | 2016-04-15 | 2018-12-11 | Univ Pennsylvania | Terapia genica para tratar la hemofilia a |
| EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| JP6811338B2 (ja) * | 2016-12-13 | 2021-01-13 | エーエム サイエンシーズ インコーポレイテッド | Ｂ型肝炎の予防または治療用医薬組成物 |
| EP3562494A4 (en) * | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS |
| WO2019028273A1 (en) * | 2017-08-02 | 2019-02-07 | Dna2.0, Inc. Dba Atum | DNA VECTORS AND ELEMENTS FOR SUSTAINED GENE EXPRESSION IN EUKARYOTIC CELLS |
| KR20230079172A (ko) * | 2020-10-01 | 2023-06-05 | 젠자임 코포레이션 | 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 |
-
2019
- 2019-10-11 CN CN202510330069.9A patent/CN120349985A/zh active Pending
- 2019-10-11 CA CA3116283A patent/CA3116283A1/en active Pending
- 2019-10-11 US US17/283,433 patent/US20210348135A1/en active Pending
- 2019-10-11 KR KR1020217013976A patent/KR20210076051A/ko active Pending
- 2019-10-11 SG SG11202103614RA patent/SG11202103614RA/en unknown
- 2019-10-11 CN CN201980082127.3A patent/CN113194982B/zh active Active
- 2019-10-11 BR BR112021006796-5A patent/BR112021006796A2/pt unknown
- 2019-10-11 EP EP19797895.0A patent/EP3863662A1/en not_active Withdrawn
- 2019-10-11 WO PCT/US2019/055917 patent/WO2020077250A1/en not_active Ceased
- 2019-10-11 MX MX2021004213A patent/MX2021004213A/es unknown
- 2019-10-11 EP EP24200416.6A patent/EP4461814A3/en active Pending
- 2019-10-11 JP JP2021519879A patent/JP2022504740A/ja active Pending
- 2019-10-11 AU AU2019357039A patent/AU2019357039B2/en active Active
- 2019-10-14 TW TW108136919A patent/TW202035439A/zh unknown
- 2019-10-15 AR ARP190102921A patent/AR116700A1/es unknown
-
2021
- 2021-04-07 IL IL282140A patent/IL282140A/en unknown
- 2021-04-11 PH PH12021550794A patent/PH12021550794A1/en unknown
- 2021-04-12 MX MX2025013445A patent/MX2025013445A/es unknown
- 2021-05-10 CO CONC2021/0006060A patent/CO2021006060A2/es unknown
-
2024
- 2024-06-19 JP JP2024098655A patent/JP2024123125A/ja active Pending
-
2025
- 2025-09-25 AU AU2025237999A patent/AU2025237999A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024123125A (ja) | 2024-09-10 |
| MX2025013445A (es) | 2025-12-01 |
| EP3863662A1 (en) | 2021-08-18 |
| CO2021006060A2 (es) | 2021-05-20 |
| EP4461814A3 (en) | 2025-02-26 |
| AU2025237999A1 (en) | 2025-10-23 |
| CN120349985A (zh) | 2025-07-22 |
| WO2020077250A1 (en) | 2020-04-16 |
| AU2019357039A1 (en) | 2021-06-03 |
| BR112021006796A2 (pt) | 2021-07-20 |
| AR116700A1 (es) | 2021-06-02 |
| TW202035439A (zh) | 2020-10-01 |
| JP2022504740A (ja) | 2022-01-13 |
| PH12021550794A1 (en) | 2021-12-13 |
| KR20210076051A (ko) | 2021-06-23 |
| EP4461814A2 (en) | 2024-11-13 |
| CN113194982B (zh) | 2025-04-01 |
| MX2021004213A (es) | 2021-07-16 |
| SG11202103614RA (en) | 2021-05-28 |
| US20210348135A1 (en) | 2021-11-11 |
| CA3116283A1 (en) | 2020-04-16 |
| CN113194982A (zh) | 2021-07-30 |
| AU2019357039B2 (en) | 2025-10-09 |
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