HUE051373T2 - Készítmények MPSI kezelésére - Google Patents
Készítmények MPSI kezeléséreInfo
- Publication number
- HUE051373T2 HUE051373T2 HUE14770186A HUE14770186A HUE051373T2 HU E051373 T2 HUE051373 T2 HU E051373T2 HU E14770186 A HUE14770186 A HU E14770186A HU E14770186 A HUE14770186 A HU E14770186A HU E051373 T2 HUE051373 T2 HU E051373T2
- Authority
- HU
- Hungary
- 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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361788724P | 2013-03-15 | 2013-03-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE051373T2 true HUE051373T2 (hu) | 2021-03-01 |
Family
ID=51580934
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE14770186A HUE051373T2 (hu) | 2013-03-15 | 2014-03-13 | Készítmények MPSI kezelésére |
Country Status (21)
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6591956B2 (ja) * | 2013-03-15 | 2019-10-16 | 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 | Ｍｐｓ１を治療するための組成物および方法 |
| US11555059B2 (en) * | 2014-04-25 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | LDLR variants and their use in compositions for reducing cholesterol levels |
| IL259850B2 (en) * | 2015-12-11 | 2023-11-01 | Univ 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 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de 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 |
| US11116850B2 (en) * | 2016-02-22 | 2021-09-14 | The University Of North Carolina At Chapel Hill | AAV-IDUA vector for treatment of MPS I-associated blindness |
| MA44874A (fr) | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type ii |
| IL322403A (en) * | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| WO2018022511A1 (en) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| KR20230161535A (ko) | 2016-07-26 | 2023-11-27 | 바이오마린 파머수티컬 인크. | 신규 아데노-관련 바이러스 캡시드 단백질 |
| IL315358A (en) | 2016-08-18 | 2024-11-01 | 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 |
| WO2019010335A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I |
| IL273427B2 (en) | 2017-09-22 | 2025-04-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous polycythemia vera 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 |
| KR20200104864A (ko) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiib형에 대한 유전자 요법 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019152843A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| JP7575273B2 (ja) | 2018-04-16 | 2024-10-29 | 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 | デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 |
| AU2019265560A1 (en) | 2018-05-09 | 2020-11-26 | 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 |
| SG11202100139VA (en) | 2018-07-17 | 2021-02-25 | Neuromyon Inc | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| AU2020216480A1 (en) | 2019-01-31 | 2021-09-09 | 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 |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| 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 |
| JP2023545433A (ja) | 2020-10-09 | 2023-10-30 | 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 | ファブリー病の治療のための組成物及び方法 |
| KR102590276B1 (ko) * | 2021-01-04 | 2023-10-16 | 부산대학교 산학협력단 | 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도 |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| 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 | The Trustees Of The University Of 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 |
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 |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| BR9910323A (pt) | 1998-05-13 | 2001-01-30 | Harbor Ucla | Alfa-l-iduronidase recombinante, métodos para produção e purificação da mesma e métodos para tratamento de doenças ocasionadas deficiência da mesma |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | 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 instrastrand 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 |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | 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 | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| WO2004055157A2 (en) | 2002-08-13 | 2004-07-01 | Whitley Chester B | Methods of using vectors to treat metabolic disorders |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| EP2383346B1 (en) | 2005-04-07 | 2014-10-08 | The Trustees of the University of Pennsylvania | Modified AAVrh.64 capsids, compositions containing same and uses thereof |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| 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 |
| JP2014503173A (ja) * | 2009-11-05 | 2014-02-13 | プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ | 制御された発現系 |
| EP2333074A1 (en) * | 2009-12-14 | 2011-06-15 | Robert Steinfeld | Substances and methods for the treatment of lysosmal storage diseases |
| EP2553106A2 (en) * | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| CA2833908C (en) | 2010-04-23 | 2021-02-09 | 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 |
| WO2012054723A2 (en) * | 2010-10-22 | 2012-04-26 | Opko Curna Llc | Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| 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 |
| AU2013289206B2 (en) | 2012-07-11 | 2018-08-09 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| JP6437443B2 (ja) | 2012-11-21 | 2018-12-12 | アールキューエックス ファーマシューティカルズ，インク． | 大環状広域抗生物質 |
| 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 |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | 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 | Ｍｐｓ１を治療するための組成物および方法 |
| EP3622821A1 (en) | 2013-05-15 | 2020-03-18 | Regents of the University of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| SG11201702073XA (en) | 2014-09-24 | 2017-04-27 | Hope City | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3204503A1 (en) | 2014-10-06 | 2017-08-16 | Arthrogen BV | Aav-based gene therapy |
| RU2751952C2 (ru) | 2015-05-15 | 2021-07-21 | Реджентс Оф Зэ Юниверсити Оф Миннесота | Вектор на основе аденоассоциированного вируса для терапевтической доставки в центральную нервную систему |
| US10604747B2 (en) | 2015-08-06 | 2020-03-31 | Academia Sinica | Engineered enzyme for enzyme replacement therapy |
| SG11201803218PA (en) | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| CA3010738A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| WO2019010335A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I |
-
2014
- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 SG SG10201707319UA patent/SG10201707319UA/en unknown
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 ES ES20170476T patent/ES2991471T3/es active Active
- 2014-03-13 PT PT147701866T patent/PT2984166T/pt unknown
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 EP EP20170476.4A patent/EP3747998B1/en active Active
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
- 2014-03-13 BR BR112015021036A patent/BR112015021036A2/pt not_active Application Discontinuation
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active Active
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/ko active Active
- 2014-03-13 PL PL14770186T patent/PL2984166T3/pl unknown
- 2014-03-13 MX MX2015012739A patent/MX386059B/es unknown
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 LT LTEP14770186.6T patent/LT2984166T/lt unknown
- 2014-03-13 SG SG11201507507PA patent/SG11201507507PA/en unknown
- 2014-03-13 SI SI201431605T patent/SI2984166T1/sl unknown
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en not_active Ceased
-
2015
- 2015-09-14 MX MX2021010918A patent/MX2021010918A/es 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
- 2020-09-03 JP JP2020148103A patent/JP7168621B2/ja active Active
- 2020-09-10 US US17/017,150 patent/US20200397872A1/en not_active Abandoned
-
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 | |
| HUE051373T2 (hu) | Készítmények MPSI kezelésére | |
| GB201304662D0 (en) | Compositions | |
| PL3065569T3 (pl) | Kompozycja | |
| HUE044039T2 (hu) | Biokatalitikus készítmény | |
| GB201608461D0 (en) | Compositions | |
| IL244118B (en) | A preparation containing dexpermipexole for the treatment of various types of urticaria | |
| EP3006525A4 (en) | ASPHALT-URETHANE COMPOSITION | |
| GB201318489D0 (en) | Composition | |
| GB201319540D0 (en) | Composition | |
| GB201320919D0 (en) | Composition | |
| GB201317388D0 (en) | Composition | |
| GB201320303D0 (en) | Composition | |
| GB201319538D0 (en) | Composition | |
| GB201306984D0 (en) | Composition | |
| GB201320409D0 (en) | Prebiotic composition | |
| GB201322772D0 (en) | Composition | |
| GB201317196D0 (en) | Composition | |
| GB201307622D0 (en) | Composition | |
| GB201320962D0 (en) | Compositions | |
| GB201420672D0 (en) | Composition | |
| GB201308502D0 (en) | Composition | |
| GB201301774D0 (en) | Composition | |
| ZA201507015B (en) | Composition for treating hemorrhoids | |
| GB201318394D0 (en) | Composition |