PL2984166T3 - Kompozycje do leczenia mpsi - Google Patents
Kompozycje do leczenia mpsiInfo
- Publication number
- PL2984166T3 PL2984166T3 PL14770186T PL14770186T PL2984166T3 PL 2984166 T3 PL2984166 T3 PL 2984166T3 PL 14770186 T PL14770186 T PL 14770186T PL 14770186 T PL14770186 T PL 14770186T PL 2984166 T3 PL2984166 T3 PL 2984166T3
- Authority
- PL
- Poland
- Prior art keywords
- compositions
- mpsi
- treating
- treating mpsi
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
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
-
- 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
- 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
-
- 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
- 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
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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (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)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| 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 |
| EP14770186.6A EP2984166B1 (en) | 2013-03-15 | 2014-03-13 | Compositions for treating mpsi |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2984166T3 true PL2984166T3 (pl) | 2021-01-11 |
Family
ID=51580934
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL14770186T PL2984166T3 (pl) | 2013-03-15 | 2014-03-13 | Kompozycje do leczenia mpsi |
Country Status (22)
| Country | Link |
|---|---|
| US (3) | US10137176B2 (pl) |
| EP (2) | EP3747998B1 (pl) |
| JP (4) | JP6591956B2 (pl) |
| KR (2) | KR102581652B1 (pl) |
| CN (1) | CN105026554B (pl) |
| AU (2) | AU2014235096B2 (pl) |
| BR (1) | BR112015021036A2 (pl) |
| CA (1) | CA2901328C (pl) |
| CY (1) | CY1123169T1 (pl) |
| DK (1) | DK2984166T3 (pl) |
| ES (1) | ES2805355T3 (pl) |
| HK (1) | HK1221735A1 (pl) |
| HR (1) | HRP20201063T1 (pl) |
| HU (1) | HUE051373T2 (pl) |
| LT (1) | LT2984166T (pl) |
| MX (2) | MX2015012739A (pl) |
| PL (1) | PL2984166T3 (pl) |
| PT (1) | PT2984166T (pl) |
| RU (1) | RU2708318C2 (pl) |
| SG (2) | SG10201707319UA (pl) |
| SI (1) | SI2984166T1 (pl) |
| WO (1) | WO2014151341A1 (pl) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2015012739A (es) | 2013-03-15 | 2016-02-19 | Univ Pennsylvania | Composiciones y metodos para tratar la mucopolisacaridosis tipo 1. |
| BR112016024379A2 (pt) * | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| US11241506B2 (en) | 2015-12-14 | 2022-02-08 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| JP7360241B2 (ja) | 2016-02-03 | 2023-10-12 | 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 |
| WO2017136533A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| RU2018132517A (ru) * | 2016-02-22 | 2020-03-24 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Aav-idua вектор для лечения ассоциированной с mps i слепоты |
| EP3452104A1 (en) | 2016-04-15 | 2019-03-13 | The Trustees of The University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| JP7046828B2 (ja) | 2016-04-15 | 2022-04-04 | 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 | 滲出型加齢性黄斑変性の治療のための組成物 |
| 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 |
| SG11201811603WA (en) | 2016-07-26 | 2019-02-27 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2017313917B2 (en) | 2016-08-18 | 2023-12-21 | The Regents Of The University Of California | CRISPR-Cas genome engineering via a modular AAV delivery system |
| EP3562494A4 (en) * | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR TREATMENT OF PHENYLKETONURIA |
| KR20240063170A (ko) | 2017-07-06 | 2024-05-09 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis 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 |
| MX2020005673A (es) | 2017-11-30 | 2020-12-03 | Univ Pennsylvania | Terapia génica para mucopolisacaridosis 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 |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| EP3790627A2 (en) | 2018-05-09 | 2021-03-17 | 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 |
| JP7413629B2 (ja) | 2018-07-17 | 2024-01-16 | ヘリックスミス カンパニー， リミテッド | Ｉｇｆ－１異型体を発現させるｄｎａコンストラクトを用いた神経病症の治療 |
| 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 |
| KR20230010670A (ko) | 2020-05-12 | 2023-01-19 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이식유전자 발현의 drg-특이적 감소를 위한 조성물 |
| 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 |
| CN115968302A (zh) | 2020-06-17 | 2023-04-14 | 宾夕法尼亚州大学信托人 | 用于治疗基因疗法患者的组合物和方法 |
| TW202229560A (zh) | 2020-10-09 | 2022-08-01 | 賓州大學委員會 | 治療法布瑞氏症之組成物及方法 |
| KR102590276B1 (ko) * | 2021-01-04 | 2023-10-16 | 부산대학교 산학협력단 | 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도 |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4148078A1 (de) | 2021-09-08 | 2023-03-15 | 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 |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | 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 |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| WO2023196893A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
Family Cites Families (43)
| 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 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| IL139616A0 (en) | 1998-05-13 | 2002-02-10 | Harbor Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating, diseases caused by deficiencies thereof |
| EP1204739B1 (en) | 1999-08-09 | 2008-08-06 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003302724A1 (en) | 2002-08-13 | 2004-07-09 | Mcivor, Scott, R. | Methods of using vectors to treat metabolic disorders |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| DK2292780T3 (en) | 2003-09-30 | 2017-12-04 | Univ Pennsylvania | Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | 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 |
| CA2780671A1 (en) * | 2009-11-05 | 2011-05-12 | Proyecto De Biomedicina Cima, S.L. | Regulated expression system |
| EP2333074A1 (en) * | 2009-12-14 | 2011-06-15 | Robert Steinfeld | Substances and methods for the treatment of lysosmal storage diseases |
| SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CA2815212A1 (en) * | 2010-10-22 | 2012-04-26 | Curna, Inc. | Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua |
| 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 |
| JP6072772B2 (ja) | 2011-04-22 | 2017-02-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| MX2015006340A (es) | 2012-11-21 | 2015-09-07 | Rqx Pharmaceuticals Inc | Antibioticos macrociclicos de amplio espectro. |
| WO2014125647A1 (ja) | 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 | 光受信装置 |
| MX2015012739A (es) * | 2013-03-15 | 2016-02-19 | Univ Pennsylvania | Composiciones y metodos para tratar la mucopolisacaridosis tipo 1. |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| WO2016055437A1 (en) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Aav-based gene therapy |
| US20180289839A1 (en) | 2015-05-15 | 2018-10-11 | Regents Of The University Of Minnesota | Intranasal therapeutic delivery of adeno-associated virus to central nervous system |
| TWI617573B (zh) | 2015-08-06 | 2018-03-11 | 中央研究院 | 用於酵素置換療法的改造酵素 |
| AU2016341428B2 (en) | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| JP6930052B2 (ja) | 2016-01-15 | 2021-09-01 | サンガモ セラピューティクス， インコーポレイテッド | 神経性疾患の処置のための方法および組成物 |
| JP7360241B2 (ja) | 2016-02-03 | 2023-10-12 | 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 |
| KR20240063170A (ko) | 2017-07-06 | 2024-05-09 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
-
2014
- 2014-03-13 MX MX2015012739A patent/MX2015012739A/es unknown
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en active Application Filing
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 PL PL14770186T patent/PL2984166T3/pl unknown
- 2014-03-13 PT PT147701866T patent/PT2984166T/pt unknown
- 2014-03-13 EP EP20170476.4A patent/EP3747998B1/en active Active
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 SI SI201431605T patent/SI2984166T1/sl unknown
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
- 2014-03-13 LT LTEP14770186.6T patent/LT2984166T/lt unknown
- 2014-03-13 SG SG10201707319UA patent/SG10201707319UA/en unknown
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/ko active IP Right Grant
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active IP Right Grant
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
- 2014-03-13 SG SG11201507507PA patent/SG11201507507PA/en unknown
- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 BR BR112015021036A patent/BR112015021036A2/pt not_active Application Discontinuation
-
2015
- 2015-09-14 MX MX2021010918A patent/MX2021010918A/es unknown
-
2016
- 2016-08-16 HK HK16109777.2A patent/HK1221735A1/zh unknown
-
2018
- 2018-10-22 US US16/166,346 patent/US10792343B2/en active Active
-
2019
- 2019-04-19 JP JP2019079881A patent/JP2019134717A/ja active Pending
-
2020
- 2020-06-10 AU AU2020203826A patent/AU2020203826B2/en active Active
- 2020-07-07 CY CY20201100628T patent/CY1123169T1/el unknown
- 2020-07-07 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2020-09-03 JP JP2020148103A patent/JP7168621B2/ja active Active
- 2020-09-10 US US17/017,150 patent/US20200397872A1/en active Pending
-
2022
- 2022-10-27 JP JP2022172314A patent/JP7553527B2/ja active Active
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL278912A (en) | Improved preparations for the treatment of muscular dystrophy | |
| LT2984166T (lt) | Kompozicijos, skirtos mpsi gydymui | |
| GB201304662D0 (en) | Compositions | |
| GB201319525D0 (en) | Composition | |
| EP2889350A4 (en) | ADHESIVE COMPOSITION | |
| GB201313615D0 (en) | Compositions | |
| IL244118B (en) | A preparation containing dexpermipexole for the treatment of various types of urticaria | |
| EP3006525A4 (en) | ASPHALT-URETHANE COMPOSITION | |
| GB201319540D0 (en) | Composition | |
| GB201318489D0 (en) | Composition | |
| GB201317388D0 (en) | Composition | |
| GB201320409D0 (en) | Prebiotic composition | |
| HK1212228A1 (en) | Emulsified composition for external application | |
| GB201320919D0 (en) | Composition | |
| GB201306984D0 (en) | Composition | |
| EP3066178A4 (en) | GREASE COMPOSITION | |
| GB201320303D0 (en) | Composition | |
| GB201319538D0 (en) | Composition | |
| GB201317196D0 (en) | Composition | |
| GB201307622D0 (en) | Composition | |
| GB201320962D0 (en) | Compositions | |
| GB201320504D0 (en) | Composition | |
| GB201308502D0 (en) | Composition | |
| GB201301774D0 (en) | Composition | |
| ZA201507015B (en) | Composition for treating hemorrhoids |