MA43968A - Thérapie génique pour traiter la mucopolysaccharidose de type i - Google Patents
Thérapie génique pour traiter la mucopolysaccharidose de type iInfo
- Publication number
- MA43968A MA43968A MA043968A MA43968A MA43968A MA 43968 A MA43968 A MA 43968A MA 043968 A MA043968 A MA 043968A MA 43968 A MA43968 A MA 43968A MA 43968 A MA43968 A MA 43968A
- Authority
- MA
- Morocco
- Prior art keywords
- gene therapy
- mucopolysaccharidosis type
- treat mucopolysaccharidosis
- treat
- type
- Prior art date
Links
Classifications
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- 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
- 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
- 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)
- C12Y302/01031—Beta-glucuronidase (3.2.1.31)
-
- 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)
-
- 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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Dispersion Chemistry (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662290547P | 2016-02-03 | 2016-02-03 | |
| US201662323271P | 2016-04-15 | 2016-04-15 | |
| US201662337178P | 2016-05-16 | 2016-05-16 | |
| US201662367798P | 2016-07-28 | 2016-07-28 | |
| US201762452560P | 2017-01-31 | 2017-01-31 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA43968A true MA43968A (fr) | 2018-12-12 |
Family
ID=58108727
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA043968A MA43968A (fr) | 2016-02-03 | 2017-02-02 | Thérapie génique pour traiter la mucopolysaccharidose de type i |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US10973929B2 (fr) |
| EP (1) | EP3411488A1 (fr) |
| JP (2) | JP7360241B2 (fr) |
| KR (2) | KR20250171426A (fr) |
| AU (2) | AU2017215211C1 (fr) |
| BR (1) | BR112018015751A2 (fr) |
| CA (1) | CA3012195A1 (fr) |
| IL (1) | IL260959B2 (fr) |
| MA (1) | MA43968A (fr) |
| SG (2) | SG10201912758TA (fr) |
| WO (1) | WO2017136500A1 (fr) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10201707319UA (en) | 2013-03-15 | 2017-10-30 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| CA2912678C (fr) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Transfert genique au systeme nerveux central a mediation par un virus adeno-associe |
| WO2017136500A1 (fr) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| IL316926A (en) | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| WO2019060662A1 (fr) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour le traitement de la mucopolysaccharidose de type ii |
| 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 |
| JP7389744B2 (ja) | 2017-11-30 | 2023-11-30 | 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ｉｉｉａ型のための遺伝子療法 |
| CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| EP3870976B1 (fr) | 2018-10-25 | 2023-11-29 | Regeneron Pharmaceuticals, Inc. | Procédés d'analyse d'une composition de protéines de capside virale |
| MA54450A (fr) * | 2018-12-10 | 2021-10-20 | Denali Therapeutics Inc | Biomarqueurs de maladie lysosomale et leurs méthodes d'utilisation |
| PE20211581A1 (es) * | 2018-12-21 | 2021-08-17 | Univ Pennsylvania | Composiciones para la reduccion de la expresion transgenica especifica de drg |
| EP3826664A4 (fr) * | 2019-02-06 | 2022-10-26 | Sangamo Therapeutics, Inc. | Procédé de traitement des mucopolysaccharidoses de type i |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| US20220389457A1 (en) * | 2019-10-23 | 2022-12-08 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| BR112022014563A2 (pt) * | 2020-01-22 | 2022-09-13 | Regenxbio Inc | Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i) |
| CA3165057A1 (fr) * | 2020-02-02 | 2021-08-05 | James M. Wilson | Compositions utiles pour traiter la gm1 gan[[d]]gliosidose |
| EP4162059A1 (fr) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions pour la réduction spécifique à des drg d'expression transgénique |
| IL300622A (en) * | 2020-08-26 | 2023-04-01 | Univ Pennsylvania | Recombinant adeno-associated virus for the treatment of adult-onset GRN-related neurodegeneration |
| AU2021356684A1 (en) | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| EP4236968A4 (fr) * | 2020-10-30 | 2024-03-27 | Immusoft Corporation | Méthodes d'administration de lymphocytes b génétiquement modifiés pour l'administration in vivo d'agents thérapeutiques |
| EP4284335A1 (fr) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Thérapie génique de céroïdes-lipofuscinoses neuronales |
| EP4405469A4 (fr) * | 2021-09-24 | 2025-09-10 | Univ Pennsylvania | Compositions utiles pour le traitement de la maladie de charcot-marie-tooth |
| AU2022379625A1 (en) * | 2021-10-27 | 2024-05-16 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023102517A1 (fr) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions et méthodes de traitement de la maladie de fabry |
| WO2023172975A1 (fr) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Méthodes d'encapsulation de vaar |
| CN119317645A (zh) | 2022-04-06 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法 |
| WO2023196892A1 (fr) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale |
| CN115838709A (zh) * | 2022-11-29 | 2023-03-24 | 武汉禾元生物科技股份有限公司 | 以基因工程水稻表达和制备重组人艾杜糖苷酸酶的方法 |
| WO2024258961A1 (fr) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Thérapie génique à base d'aav pour la mucopolysaccharidose iiib |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025035143A1 (fr) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions et méthodes de traitement de l'amyotrophie spinale |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6149909A (en) | 1995-06-23 | 2000-11-21 | Women's And Children's Hospital | Synthetic α-L-iduronidase and genetic sequences encoding same |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| EP0931158A1 (fr) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | Procede inductible de production de virus adeno-associes recombines au moyen de la polymerase t7 |
| AU4072499A (en) | 1998-05-13 | 1999-11-29 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| US6426208B1 (en) | 1999-11-12 | 2002-07-30 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| RU2196988C2 (ru) | 2000-04-19 | 2003-01-20 | Государственный новосибирский областной клинический диагностический центр | Способ диагностики мукополисахаридозов |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 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 |
| WO2003052051A2 (fr) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Sequences du serotype 8 du virus associe a l'adenovirus (aav), vecteurs les contenant et utilisations correspondantes |
| WO2004055157A2 (fr) | 2002-08-13 | 2004-07-01 | Whitley Chester B | Procedes d'utilisation de vecteurs pour traiter des troubles metaboliques |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20090062144A1 (en) | 2007-04-03 | 2009-03-05 | Nancy Lan Guo | Gene signature for prognosis and diagnosis of lung cancer |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2394667A1 (fr) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vecteurs et séquences pour le traitement de maladies |
| ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
| DK4005603T3 (da) | 2011-04-22 | 2024-11-25 | Univ California | Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| WO2014125647A1 (fr) | 2013-02-18 | 2014-08-21 | 富士通オプティカルコンポーネンツ株式会社 | Dispositif photorécepteur |
| SG10201707319UA (en) * | 2013-03-15 | 2017-10-30 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| CA2912678C (fr) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Transfert genique au systeme nerveux central a mediation par un virus adeno-associe |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| CA2963168A1 (fr) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Therapie genique a base de virus adeno-associes |
| HRP20220050T1 (hr) | 2015-05-15 | 2022-10-14 | Regents Of The University Of Minnesota | Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav |
| CN107922932A (zh) | 2015-08-06 | 2018-04-17 | 中央研究院 | 用于酶置换疗法的改造酶 |
| CN108601771B (zh) | 2015-10-23 | 2023-02-21 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| WO2017100676A1 (fr) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Procédé de purification évolutif d'aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| EP3387118B1 (fr) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Procédé de purification évolutif d'aavrh10 |
| ES2895430T3 (es) | 2016-01-15 | 2022-02-21 | Univ Minnesota | Métodos y composiciones para el tratamiento de enfermedad neurológica |
| WO2017136500A1 (fr) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
-
2017
- 2017-02-02 WO PCT/US2017/016133 patent/WO2017136500A1/fr not_active Ceased
- 2017-02-02 CA CA3012195A patent/CA3012195A1/fr active Pending
- 2017-02-02 MA MA043968A patent/MA43968A/fr unknown
- 2017-02-02 EP EP17706915.0A patent/EP3411488A1/fr active Pending
- 2017-02-02 SG SG10201912758TA patent/SG10201912758TA/en unknown
- 2017-02-02 SG SG11201806270XA patent/SG11201806270XA/en unknown
- 2017-02-02 AU AU2017215211A patent/AU2017215211C1/en active Active
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/ja active Active
- 2017-02-02 IL IL260959A patent/IL260959B2/en unknown
- 2017-02-02 US US16/075,056 patent/US10973929B2/en active Active
- 2017-02-02 BR BR112018015751-1A patent/BR112018015751A2/pt not_active Application Discontinuation
- 2017-02-02 KR KR1020257038989A patent/KR20250171426A/ko active Pending
- 2017-02-02 KR KR1020187025463A patent/KR20180121899A/ko not_active Ceased
-
2021
- 2021-03-15 US US17/201,301 patent/US20210228740A1/en not_active Abandoned
-
2023
- 2023-02-17 JP JP2023023041A patent/JP2023062088A/ja active Pending
- 2023-05-02 AU AU2023202680A patent/AU2023202680A1/en not_active Abandoned
- 2023-09-27 US US18/475,999 patent/US20240024510A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL260959B2 (en) | 2024-09-01 |
| SG11201806270XA (en) | 2018-08-30 |
| CA3012195A1 (fr) | 2017-08-10 |
| AU2017215211A1 (en) | 2018-08-09 |
| JP7360241B2 (ja) | 2023-10-12 |
| AU2017215211B2 (en) | 2023-05-18 |
| EP3411488A1 (fr) | 2018-12-12 |
| WO2017136500A1 (fr) | 2017-08-10 |
| IL260959A (en) | 2018-09-20 |
| AU2017215211C1 (en) | 2023-11-16 |
| JP2023062088A (ja) | 2023-05-02 |
| SG10201912758TA (en) | 2020-02-27 |
| AU2023202680A1 (en) | 2023-07-13 |
| IL260959B1 (en) | 2024-05-01 |
| US20210228740A1 (en) | 2021-07-29 |
| JP2019512005A (ja) | 2019-05-09 |
| BR112018015751A2 (pt) | 2019-02-05 |
| KR20250171426A (ko) | 2025-12-08 |
| US10973929B2 (en) | 2021-04-13 |
| KR20180121899A (ko) | 2018-11-09 |
| US20190038772A1 (en) | 2019-02-07 |
| US20240024510A1 (en) | 2024-01-25 |
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