CA2607173C - Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease - Google Patents
Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease Download PDFInfo
- Publication number
- CA2607173C CA2607173C CA2607173A CA2607173A CA2607173C CA 2607173 C CA2607173 C CA 2607173C CA 2607173 A CA2607173 A CA 2607173A CA 2607173 A CA2607173 A CA 2607173A CA 2607173 C CA2607173 C CA 2607173C
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- 239000013598 vector Substances 0.000 title claims abstract description 8
- 241000702421 Dependoparvovirus Species 0.000 title claims abstract description 4
- 208000015439 Lysosomal storage disease Diseases 0.000 title claims abstract 6
- 208000024827 Alzheimer disease Diseases 0.000 title claims abstract 5
- 210000000234 capsid Anatomy 0.000 title claims abstract 3
- 241000124008 Mammalia Species 0.000 claims abstract 9
- 210000003169 central nervous system Anatomy 0.000 claims abstract 7
- 239000013607 AAV vector Substances 0.000 claims abstract 5
- 239000003937 drug carrier Substances 0.000 claims abstract 3
- 230000003376 axonal effect Effects 0.000 claims abstract 2
- 210000003591 cerebellar nuclei Anatomy 0.000 claims abstract 2
- 239000002253 acid Substances 0.000 claims 7
- 230000002132 lysosomal effect Effects 0.000 claims 4
- 102000004157 Hydrolases Human genes 0.000 claims 3
- 108090000604 Hydrolases Proteins 0.000 claims 3
- 108090000131 Metalloendopeptidases Proteins 0.000 claims 2
- 102000003843 Metalloendopeptidases Human genes 0.000 claims 2
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 2
- ZSLZBFCDCINBPY-ZSJPKINUSA-N acetyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZSLZBFCDCINBPY-ZSJPKINUSA-N 0.000 claims 2
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 102000006772 Acid Ceramidase Human genes 0.000 claims 1
- 108020005296 Acid Ceramidase Proteins 0.000 claims 1
- 102100022146 Arylsulfatase A Human genes 0.000 claims 1
- 102100031491 Arylsulfatase B Human genes 0.000 claims 1
- 108010023546 Aspartylglucosylaminase Proteins 0.000 claims 1
- 101100080277 Caenorhabditis elegans ncr-1 gene Proteins 0.000 claims 1
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 1
- 102000003779 Dipeptidyl-peptidases and tripeptidyl-peptidases Human genes 0.000 claims 1
- 108090000194 Dipeptidyl-peptidases and tripeptidyl-peptidases Proteins 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 102100021496 Insulin-degrading enzyme Human genes 0.000 claims 1
- 108090000828 Insulysin Proteins 0.000 claims 1
- 102000004882 Lipase Human genes 0.000 claims 1
- 108090001060 Lipase Proteins 0.000 claims 1
- 102100021003 N(4)-(beta-N-acetylglucosaminyl)-L-asparaginase Human genes 0.000 claims 1
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 1
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 claims 1
- 101710099863 N-acetylgalactosamine-6-sulfatase Proteins 0.000 claims 1
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 1
- 101710202061 N-acetyltransferase Proteins 0.000 claims 1
- 102000003729 Neprilysin Human genes 0.000 claims 1
- 108090000028 Neprilysin Proteins 0.000 claims 1
- 101100459404 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) npc-1 gene Proteins 0.000 claims 1
- 102000005327 Palmitoyl protein thioesterase Human genes 0.000 claims 1
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 claims 1
- 102100031293 Thimet oligopeptidase Human genes 0.000 claims 1
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims 1
- 235000018417 cysteine Nutrition 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims 1
- 210000001767 medulla oblongata Anatomy 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
- 235000018102 proteins Nutrition 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 210000003523 substantia nigra Anatomy 0.000 claims 1
- 108010073106 thimet oligopeptidase Proteins 0.000 claims 1
- 108091005703 transmembrane proteins Proteins 0.000 claims 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a 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
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CA2998603A CA2998603C (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US67705705P | 2005-05-02 | 2005-05-02 | |
| US60/677,057 | 2005-05-02 | ||
| US68580805P | 2005-05-31 | 2005-05-31 | |
| US60/685,808 | 2005-05-31 | ||
| PCT/US2006/017242 WO2006119458A1 (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2998603A Division CA2998603C (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2607173A1 CA2607173A1 (en) | 2006-11-09 |
| CA2607173C true CA2607173C (en) | 2018-04-24 |
Family
ID=37308324
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2607173A Active CA2607173C (en) | 2005-05-02 | 2006-05-02 | Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease |
| CA2998603A Active CA2998603C (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2998603A Active CA2998603C (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US10632213B2 (enExample) |
| EP (4) | EP2420256B1 (enExample) |
| JP (3) | JP5829372B2 (enExample) |
| CN (4) | CN104306986A (enExample) |
| AT (1) | ATE525092T1 (enExample) |
| AU (1) | AU2006243776A1 (enExample) |
| BR (1) | BRPI0611379A2 (enExample) |
| CA (2) | CA2607173C (enExample) |
| ES (1) | ES2887076T3 (enExample) |
| IL (2) | IL187078A (enExample) |
| PL (4) | PL3058959T3 (enExample) |
| PT (4) | PT2420256T (enExample) |
| WO (1) | WO2006119458A1 (enExample) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3302529B2 (ja) | 1995-04-28 | 2002-07-15 | 株式会社クボタ | ポンプ吸水槽の整流装置 |
| DK1879623T3 (da) * | 2005-05-02 | 2012-12-17 | Genzyme Corp | Genterapi til rygmarvssygdomme |
| EP2420256B1 (en) | 2005-05-02 | 2016-06-22 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| EP2489733B1 (en) | 2006-06-07 | 2019-02-13 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| EP3252161B1 (en) | 2007-06-06 | 2021-11-17 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
| EP2687609B1 (en) | 2008-11-10 | 2017-01-04 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Method for treating solid tumor |
| PL4342992T3 (pl) | 2009-05-02 | 2025-09-22 | Genzyme Corporation | Terapia genowa dla zaburzeń neurodegeneracyjnych |
| US9150926B2 (en) | 2010-12-06 | 2015-10-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Diagnosis and treatment of adrenocortical tumors using human microRNA-483 |
| WO2012145646A1 (en) * | 2011-04-20 | 2012-10-26 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| KR20230169197A (ko) | 2014-11-14 | 2023-12-15 | 보이저 테라퓨틱스, 인크. | 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법 |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| US11319555B2 (en) | 2014-11-20 | 2022-05-03 | Duke University | Compositions, systems and methods for cell therapy |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| HUE057272T2 (hu) * | 2015-02-10 | 2022-04-28 | Genzyme Corp | Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe |
| CN112472796B (zh) * | 2015-03-26 | 2024-12-24 | 上海澳宗生物科技有限公司 | 使用p75ecd和/或p75诊断或治疗神经障碍的方法 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| US11027024B2 (en) | 2015-05-29 | 2021-06-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| CN117904112A (zh) | 2016-05-18 | 2024-04-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN119491003A (zh) | 2017-05-05 | 2025-02-21 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| IL271193B2 (en) | 2017-06-07 | 2025-01-01 | Regeneron Pharma | Preparations and methods for internalizing enzymes |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018352236B2 (en) | 2017-10-16 | 2025-04-10 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019157224A1 (en) | 2018-02-07 | 2019-08-15 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| AU2019269685B2 (en) | 2018-05-17 | 2025-12-04 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| KR20240126916A (ko) * | 2023-02-14 | 2024-08-22 | 경북대학교 산학협력단 | Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE8303626D0 (sv) | 1983-06-23 | 1983-06-23 | Kabigen Ab | A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced |
| US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| WO1995000655A1 (en) | 1993-06-24 | 1995-01-05 | Mc Master University | Adenovirus vectors for gene therapy |
| ATE437232T1 (de) | 1993-10-25 | 2009-08-15 | Canji Inc | Rekombinanter adenoviren-vektor und verfahren zur verwendung |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
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