SG10201912758TA - Gene therapy for treating mucopolysaccharidosis type i - Google Patents
Gene therapy for treating mucopolysaccharidosis type iInfo
- Publication number
- SG10201912758TA SG10201912758TA SG10201912758TA SG10201912758TA SG10201912758TA SG 10201912758T A SG10201912758T A SG 10201912758TA SG 10201912758T A SG10201912758T A SG 10201912758TA SG 10201912758T A SG10201912758T A SG 10201912758TA SG 10201912758T A SG10201912758T A SG 10201912758TA
- Authority
- SG
- Singapore
- Prior art keywords
- gene therapy
- mucopolysaccharidosis type
- treating mucopolysaccharidosis
- treating
- type
- Prior art date
Links
- 102100035028 Alpha-L-iduronidase Human genes 0.000 title 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 title 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 title 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
Classifications
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- 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)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (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)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Dispersion Chemistry (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)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (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 |
|---|---|
| SG10201912758TA true SG10201912758TA (en) | 2020-02-27 |
Family
ID=58108727
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201806270XA SG11201806270XA (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
| SG10201912758TA SG10201912758TA (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201806270XA SG11201806270XA (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US10973929B2 (ko) |
| EP (1) | EP3411488A1 (ko) |
| JP (2) | JP7360241B2 (ko) |
| KR (1) | KR20180121899A (ko) |
| AU (2) | AU2017215211C1 (ko) |
| BR (1) | BR112018015751A2 (ko) |
| CA (1) | CA3012195A1 (ko) |
| IL (1) | IL260959B1 (ko) |
| MA (1) | MA43968A (ko) |
| SG (2) | SG11201806270XA (ko) |
| WO (1) | WO2017136500A1 (ko) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2984166T3 (pl) | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| KR20190132639A (ko) | 2017-02-28 | 2019-11-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 아데노-연관 바이러스(aav) 클레이드 f 벡터 및 이의 용도 |
| AU2018265531B2 (en) | 2017-05-11 | 2024-07-11 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
| EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| 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 |
| MX2020005663A (es) | 2017-11-30 | 2020-08-20 | Univ Pennsylvania | Terapia genica para mucopolisacaridosis iii a. |
| 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 |
| AU2019306236A1 (en) * | 2018-07-18 | 2021-03-11 | Regenxbio Inc. | Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-L-iduronidase (IDUS) |
| EP3894864A2 (en) * | 2018-12-10 | 2021-10-20 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| AU2019428629A1 (en) * | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| AU2020369570A1 (en) * | 2019-10-23 | 2022-05-12 | 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) |
| WO2021155337A1 (en) * | 2020-02-02 | 2021-08-05 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| JP2023540054A (ja) * | 2020-08-26 | 2023-09-21 | 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 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| EP4225906A1 (en) | 2020-10-09 | 2023-08-16 | The Trustees of The University of Pennsylvania | Compositions and methods for treatment of fabry disease |
| EP4236968A4 (en) * | 2020-10-30 | 2024-03-27 | Immusoft Corporation | METHOD FOR ADMINISTRATION OF GENETICALLY MODIFIED B CELLS FOR IN VIVO ADMINISTRATION OF THERAPEUTICS |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| 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 |
| WO2023172975A1 (en) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Methods of raav packaging |
| 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 |
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| 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 |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| JP2002514429A (ja) | 1998-05-13 | 2002-05-21 | ハーバー−ユーシーエルエー | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法 |
| 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 |
| SG10201912509RA (en) | 2001-11-13 | 2020-02-27 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| 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 |
| CN102212558B (zh) | 2003-09-30 | 2016-08-03 | 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 |
| CN106906241B (zh) | 2005-04-07 | 2020-09-11 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| 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 |
| EP2553106A2 (en) | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system 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 |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | 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 | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
| US9193956B2 (en) | 2011-04-22 | 2015-11-24 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| HUE030799T2 (en) | 2012-07-11 | 2017-06-28 | Sangamo Biosciences Inc | Methods and preparations for the treatment of lysosomal storage diseases |
| 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 |
| PL2984166T3 (pl) * | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| BR112015028605A8 (pt) | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
| EP3044318B1 (en) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Selective recovery |
| WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US20170304466A1 (en) | 2014-10-06 | 2017-10-26 | Arthrogen B.V. | AAV-Based Gene Therapy |
| MX2017014443A (es) | 2015-05-15 | 2018-08-01 | Univ Minnesota | Adeno-asociado para administracion terapeutica al sistema nervioso central. |
| 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 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP3992283A1 (en) | 2015-12-11 | 2022-05-04 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| CA3010738A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
-
2017
- 2017-02-02 SG SG11201806270XA patent/SG11201806270XA/en unknown
- 2017-02-02 CA CA3012195A patent/CA3012195A1/en active Pending
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/ja active Active
- 2017-02-02 SG SG10201912758TA patent/SG10201912758TA/en unknown
- 2017-02-02 WO PCT/US2017/016133 patent/WO2017136500A1/en active Application Filing
- 2017-02-02 IL IL260959A patent/IL260959B1/en unknown
- 2017-02-02 MA MA043968A patent/MA43968A/fr unknown
- 2017-02-02 BR BR112018015751A patent/BR112018015751A2/pt unknown
- 2017-02-02 KR KR1020187025463A patent/KR20180121899A/ko unknown
- 2017-02-02 AU AU2017215211A patent/AU2017215211C1/en active Active
- 2017-02-02 US US16/075,056 patent/US10973929B2/en active Active
- 2017-02-02 EP EP17706915.0A patent/EP3411488A1/en active Pending
-
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 active Pending
- 2023-09-27 US US18/475,999 patent/US20240024510A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MA43968A (fr) | 2018-12-12 |
| JP2019512005A (ja) | 2019-05-09 |
| US20190038772A1 (en) | 2019-02-07 |
| SG11201806270XA (en) | 2018-08-30 |
| JP7360241B2 (ja) | 2023-10-12 |
| US10973929B2 (en) | 2021-04-13 |
| BR112018015751A2 (pt) | 2019-02-05 |
| AU2017215211C1 (en) | 2023-11-16 |
| CA3012195A1 (en) | 2017-08-10 |
| AU2017215211A1 (en) | 2018-08-09 |
| AU2023202680A1 (en) | 2023-07-13 |
| JP2023062088A (ja) | 2023-05-02 |
| EP3411488A1 (en) | 2018-12-12 |
| US20210228740A1 (en) | 2021-07-29 |
| US20240024510A1 (en) | 2024-01-25 |
| AU2017215211B2 (en) | 2023-05-18 |
| IL260959A (en) | 2018-09-20 |
| WO2017136500A1 (en) | 2017-08-10 |
| KR20180121899A (ko) | 2018-11-09 |
| IL260959B1 (en) | 2024-05-01 |
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