CY1123169T1 - Συνθεσεις για αντιμετωπιση της mpsi - Google Patents
Συνθεσεις για αντιμετωπιση της mpsiInfo
- Publication number
- CY1123169T1 CY1123169T1 CY20201100628T CY201100628T CY1123169T1 CY 1123169 T1 CY1123169 T1 CY 1123169T1 CY 20201100628 T CY20201100628 T CY 20201100628T CY 201100628 T CY201100628 T CY 201100628T CY 1123169 T1 CY1123169 T1 CY 1123169T1
- Authority
- CY
- Cyprus
- Prior art keywords
- mpsi
- vector
- compositions
- treat
- hurler
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 2
- 239000013598 vector Substances 0.000 abstract 4
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 abstract 3
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 abstract 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 abstract 1
- 208000015178 Hurler syndrome Diseases 0.000 abstract 1
- 208000015204 Hurler-Scheie syndrome Diseases 0.000 abstract 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 abstract 1
- 201000002883 Scheie syndrome Diseases 0.000 abstract 1
- 102000056929 human IDUA Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 239000013647 rAAV8 vector Substances 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
Classifications
-
- 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
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- 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
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Dermatology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361788724P | 2013-03-15 | 2013-03-15 | |
| PCT/US2014/025509 WO2014151341A1 (en) | 2013-03-15 | 2014-03-13 | Compositions and methods for treating mpsi |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1123169T1 true CY1123169T1 (el) | 2021-10-29 |
Family
ID=51580934
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20201100628T CY1123169T1 (el) | 2013-03-15 | 2020-07-07 | Συνθεσεις για αντιμετωπιση της mpsi |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US10137176B2 (enExample) |
| EP (2) | EP3747998B1 (enExample) |
| JP (4) | JP6591956B2 (enExample) |
| KR (2) | KR102346455B1 (enExample) |
| CN (1) | CN105026554B (enExample) |
| AU (2) | AU2014235096B2 (enExample) |
| BR (1) | BR112015021036A2 (enExample) |
| CA (1) | CA2901328C (enExample) |
| CY (1) | CY1123169T1 (enExample) |
| DK (1) | DK2984166T3 (enExample) |
| ES (2) | ES2805355T3 (enExample) |
| HR (1) | HRP20201063T1 (enExample) |
| HU (1) | HUE051373T2 (enExample) |
| LT (1) | LT2984166T (enExample) |
| MX (2) | MX386059B (enExample) |
| PL (1) | PL2984166T3 (enExample) |
| PT (1) | PT2984166T (enExample) |
| RU (1) | RU2708318C2 (enExample) |
| SG (2) | SG10201707319UA (enExample) |
| SI (1) | SI2984166T1 (enExample) |
| WO (1) | WO2014151341A1 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3747998B1 (en) | 2013-03-15 | 2024-07-24 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
| WO2015164778A1 (en) * | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennysylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| EP3400304B1 (en) * | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11241506B2 (en) | 2015-12-14 | 2022-02-08 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| US10973929B2 (en) * | 2016-02-03 | 2021-04-13 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type I |
| WO2017136533A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| MX2018009426A (es) * | 2016-02-22 | 2018-12-19 | Univ North Carolina Chapel Hill | Vector aav-idua para el tratamiento de ceguera asociada con mucopolisacaridosis i (mps i). |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| MA44873A (fr) * | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative |
| US11883470B2 (en) | 2016-07-25 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| IL264872B2 (en) | 2016-08-18 | 2025-02-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| JP2020526514A (ja) | 2017-07-06 | 2020-08-31 | 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ｉ型を治療するための遺伝子治療 |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| CA3083761A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| EP3780952A4 (en) | 2018-04-16 | 2022-03-09 | The Trustees of the University of Pennsylvania | COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| WO2020016655A2 (en) | 2018-07-17 | 2020-01-23 | Helixmith Co., Ltd. | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| US20230193315A1 (en) | 2019-01-31 | 2023-06-22 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| 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 |
| AU2021270447A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| 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 |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| KR20230118075A (ko) | 2020-10-09 | 2023-08-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 파브리병 치료를 위한 조성물 및 방법 |
| KR102590276B1 (ko) * | 2021-01-04 | 2023-10-16 | 부산대학교 산학협력단 | 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4148078B1 (de) | 2021-09-08 | 2025-11-05 | Covestro Deutschland AG | Auf bernsteinsäure basierende polyesterpolyole |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| CA3247109A1 (en) | 2022-04-06 | 2023-10-12 | Univ Pennsylvania | Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers |
| EP4504258A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of the University of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
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| US10604747B2 (en) | 2015-08-06 | 2020-03-31 | Academia Sinica | Engineered enzyme for enzyme replacement therapy |
| WO2017070678A1 (en) | 2015-10-23 | 2017-04-27 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| WO2017100704A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| US10973929B2 (en) | 2016-02-03 | 2021-04-13 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type I |
| JP2020526514A (ja) | 2017-07-06 | 2020-08-31 | 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ｉ型を治療するための遺伝子治療 |
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