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
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)
- 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)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Obesity (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)
- Medicinal Preparation (AREA)
- Enzymes And Modification Thereof (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 |
|---|---|
| SG10201912758TA true SG10201912758TA (en) | 2020-02-27 |
Family
ID=58108727
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201912758TA SG10201912758TA (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
| SG11201806270XA SG11201806270XA (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Family Applications After (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 (en) |
| EP (1) | EP3411488A1 (en) |
| JP (2) | JP7360241B2 (en) |
| KR (1) | KR20180121899A (en) |
| AU (2) | AU2017215211C1 (en) |
| BR (1) | BR112018015751A2 (en) |
| CA (1) | CA3012195A1 (en) |
| IL (1) | IL260959B2 (en) |
| MA (1) | MA43968A (en) |
| SG (2) | SG10201912758TA (en) |
| WO (1) | WO2017136500A1 (en) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6591956B2 (en) | 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 | Compositions and methods for treating MPS1 |
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JOP20190200A1 (en) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | Compositions useful in treatment of spinal muscular atrophy |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| CA3061655A1 (en) | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| SG11202002457RA (en) | 2017-09-22 | 2020-04-29 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| CN107699590A (en) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | A kind of method of Prepare restructuring people α L iduronases |
| KR20200104864A (en) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Gene therapy for mucopolysaccharide type IIIB |
| JP7389744B2 (en) | 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 | Gene therapy for mucopolysaccharidosis type IIIA |
| IL280198B1 (en) * | 2018-07-18 | 2025-09-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
| EP4306961A3 (en) * | 2018-10-25 | 2024-04-17 | Regeneron Pharmaceuticals, Inc. | Methods for analysis of viral capsid protein composition |
| MA54450A (en) * | 2018-12-10 | 2021-10-20 | Denali Therapeutics Inc | LYSOSOMAL DISEASE BIOMARKERS AND THEIR METHODS OF USE |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CA3106693A1 (en) * | 2019-02-06 | 2020-08-13 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| KR20210131370A (en) | 2019-02-22 | 2021-11-02 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Recombinant adeno-associated virus for the treatment of GRN-associated adult-onset neurodegeneration |
| IL292372A (en) * | 2019-10-23 | 2022-06-01 | Univ Pennsylvania | Preparations for the reduction of transgene expression specifically drg |
| WO2021150570A1 (en) * | 2020-01-22 | 2021-07-29 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| CA3165057A1 (en) * | 2020-02-02 | 2021-08-05 | James M. Wilson | Compositions useful for treating gm1 gangliosidosis |
| CN115803064A (en) | 2020-05-12 | 2023-03-14 | 宾夕法尼亚州大学信托人 | Compositions for drg-specific reduction of transgene expression |
| JP2023540054A (en) * | 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 | Recombinant adeno-associated virus for the treatment of GRN-associated adult-onset neurodegeneration |
| KR20230118075A (en) | 2020-10-09 | 2023-08-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Compositions and methods for the treatment of Fabry disease |
| CN116490203A (en) * | 2020-10-30 | 2023-07-25 | 益缪索夫特公司 | Methods of administering genetically modified B cells for in vivo delivery of therapeutic agents |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4405469A4 (en) * | 2021-09-24 | 2025-09-10 | Univ Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| EP4423264A4 (en) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | AAV-IDUA VECTOR FOR THE 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 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| US20250188490A1 (en) * | 2022-03-08 | 2025-06-12 | 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 |
| KR20250007064A (en) | 2022-04-06 | 2025-01-13 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Compositions and methods for treating HER2 positive metastatic breast cancer and other cancers |
| CN115838709A (en) * | 2022-11-29 | 2023-03-24 | 武汉禾元生物科技股份有限公司 | Method for expressing and preparing recombinant human iduronidase by genetic engineering rice |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
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 |
| JP2001500015A (en) | 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 | Method for producing inducible recombinant adeno-associated virus using T7 polymerase |
| CA2328518A1 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| JP4827353B2 (en) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | Increased expression of single-stranded heterologous nucleotide sequences from recombinant viral vectors by designing sequences to form 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 (en) | 2000-04-19 | 2003-01-20 | Государственный новосибирский областной клинический диагностический центр | Method for diagnostics of mucopolysaccharidosis |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| JP4810062B2 (en) | 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 | Sequence of adeno-associated virus (AAV) serotype 8 |
| 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 |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| JP4495210B2 (en) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | Amplitude error compensator and orthogonality error compensator |
| 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 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP3540055A1 (en) | 2010-04-23 | 2019-09-18 | 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 |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| LT3254703T (en) | 2011-04-22 | 2020-05-25 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| WO2014125647A1 (en) | 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 | Photoreceptor device |
| JP6591956B2 (en) | 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 | Compositions and methods for treating MPS1 |
| CA2912678C (en) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| EP3800260A1 (en) | 2014-09-24 | 2021-04-07 | City of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| AU2015330092A1 (en) | 2014-10-06 | 2017-04-13 | Arthrogen B.V. | AAV-based gene therapy |
| SI3294323T1 (en) | 2015-05-15 | 2022-06-30 | Regenxbio Inc. | Adeno-associated virus for therapeutic delivery to the central nervous system |
| WO2017024204A1 (en) | 2015-08-06 | 2017-02-09 | 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 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | 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 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| ES2918998T3 (en) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Scalable purification method for AAVrh10 |
| WO2017123757A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis 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 EP EP17706915.0A patent/EP3411488A1/en active Pending
- 2017-02-02 AU AU2017215211A patent/AU2017215211C1/en active Active
- 2017-02-02 IL IL260959A patent/IL260959B2/en unknown
- 2017-02-02 WO PCT/US2017/016133 patent/WO2017136500A1/en not_active Ceased
- 2017-02-02 SG SG10201912758TA patent/SG10201912758TA/en unknown
- 2017-02-02 MA MA043968A patent/MA43968A/en unknown
- 2017-02-02 CA CA3012195A patent/CA3012195A1/en active Pending
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/en active Active
- 2017-02-02 SG SG11201806270XA patent/SG11201806270XA/en unknown
- 2017-02-02 US US16/075,056 patent/US10973929B2/en active Active
- 2017-02-02 BR BR112018015751-1A patent/BR112018015751A2/en not_active Application Discontinuation
- 2017-02-02 KR KR1020187025463A patent/KR20180121899A/en 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/en 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 |
|---|---|
| BR112018015751A2 (en) | 2019-02-05 |
| JP2019512005A (en) | 2019-05-09 |
| CA3012195A1 (en) | 2017-08-10 |
| US20210228740A1 (en) | 2021-07-29 |
| JP2023062088A (en) | 2023-05-02 |
| AU2017215211A1 (en) | 2018-08-09 |
| SG11201806270XA (en) | 2018-08-30 |
| WO2017136500A1 (en) | 2017-08-10 |
| US20240024510A1 (en) | 2024-01-25 |
| US20190038772A1 (en) | 2019-02-07 |
| IL260959B1 (en) | 2024-05-01 |
| EP3411488A1 (en) | 2018-12-12 |
| JP7360241B2 (en) | 2023-10-12 |
| US10973929B2 (en) | 2021-04-13 |
| IL260959B2 (en) | 2024-09-01 |
| IL260959A (en) | 2018-09-20 |
| AU2017215211B2 (en) | 2023-05-18 |
| AU2017215211C1 (en) | 2023-11-16 |
| KR20180121899A (en) | 2018-11-09 |
| AU2023202680A1 (en) | 2023-07-13 |
| MA43968A (en) | 2018-12-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL260959A (en) | Gene therapy for treating mucopolysaccharidosis type i | |
| IL262211B1 (en) | Gene therapy for treating mucopolysaccharidosis type ii | |
| IL271780A (en) | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i | |
| IL304084B1 (en) | Gene therapy | |
| IL273427A (en) | Gene therapy for treating mucopolysaccharidosis type ii | |
| SG10202009849WA (en) | Gene therapy for treating hemophilia a | |
| IL274972A (en) | Gene therapy for mucopolysaccharidosis iiib | |
| LT3612624T (en) | Gene therapy | |
| GB201420139D0 (en) | Factor IX gene therapy | |
| IL258005A (en) | Methods and materials for galgt2 gene therapy | |
| IL259850A (en) | Gene therapy for treating familial hypercholesterolemia | |
| IL274939A (en) | Gene therapy for mucopolysaccharidosis iiia | |
| IL267057A (en) | Gene therapy for mucopolysaccharidosis, type i | |
| GB201407322D0 (en) | Gene therapy | |
| HUE056570T2 (en) | Gene therapy | |
| GB201707212D0 (en) | Gene therapy for ciliopathies | |
| IL267060A (en) | Gene therapy for mucopolysaccharidosis, type ii | |
| GB201701968D0 (en) | Gene therapy | |
| GB201513151D0 (en) | Functional gene replacement therapy | |
| HK40100628A (en) | Gene therapy | |
| HK40096184A (en) | Gene therapy | |
| HK40095843A (en) | Gene therapy | |
| HK40001467A (en) | Gene therapy for treating mucopolysaccharidosis type i | |
| HK40031706A (en) | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i | |
| HK1262142A1 (en) | Gene therapy for treating mucopolysaccharidosis type ii |