HRP20251506T1 - Genska terapija za lizosomske poremećaje - Google Patents
Genska terapija za lizosomske poremećajeInfo
- Publication number
- HRP20251506T1 HRP20251506T1 HRP20251506TT HRP20251506T HRP20251506T1 HR P20251506 T1 HRP20251506 T1 HR P20251506T1 HR P20251506T T HRP20251506T T HR P20251506TT HR P20251506 T HRP20251506 T HR P20251506T HR P20251506 T1 HRP20251506 T1 HR P20251506T1
- Authority
- HR
- Croatia
- Prior art keywords
- raav
- use according
- itr
- administered
- transgenic insert
- Prior art date
Links
Classifications
-
- 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
- C07K14/475—Growth factors; Growth regulators
-
- 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
- A61K48/0083—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 administration regime
-
- 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
-
- 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
-
- 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
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/18—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with another compound as one donor, and incorporation of one atom of oxygen (1.14.18)
- C12Y114/18001—Tyrosinase (1.14.18.1)
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6803—General methods of protein analysis not limited to specific proteins or families of proteins
-
- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Urology & Nephrology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Food Science & Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- General Physics & Mathematics (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831846P | 2019-04-10 | 2019-04-10 | |
| US201962934450P | 2019-11-12 | 2019-11-12 | |
| US201962954089P | 2019-12-27 | 2019-12-27 | |
| US202062960471P | 2020-01-13 | 2020-01-13 | |
| US202062988665P | 2020-03-12 | 2020-03-12 | |
| PCT/US2020/027764 WO2020210698A1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
| EP20724262.9A EP3953377B1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20251506T1 true HRP20251506T1 (hr) | 2026-01-02 |
Family
ID=70554180
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20251506TT HRP20251506T1 (hr) | 2019-04-10 | 2020-04-10 | Genska terapija za lizosomske poremećaje |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US11999974B2 (https=) |
| EP (2) | EP3953377B1 (https=) |
| JP (2) | JP7571044B2 (https=) |
| KR (1) | KR20210150486A (https=) |
| CN (1) | CN114174324A (https=) |
| AU (2) | AU2020273182B2 (https=) |
| BR (1) | BR112021020183A2 (https=) |
| CA (1) | CA3136004A1 (https=) |
| DK (1) | DK3953377T3 (https=) |
| ES (1) | ES3055985T3 (https=) |
| FI (1) | FI3953377T3 (https=) |
| HR (1) | HRP20251506T1 (https=) |
| IL (1) | IL286903A (https=) |
| LT (1) | LT3953377T (https=) |
| MX (1) | MX2021011958A (https=) |
| PL (1) | PL3953377T3 (https=) |
| PT (1) | PT3953377T (https=) |
| RS (1) | RS67476B1 (https=) |
| SI (1) | SI3953377T1 (https=) |
| WO (1) | WO2020210698A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110506057B (zh) | 2017-02-17 | 2023-09-29 | 百时美施贵宝公司 | Alpha突触核蛋白抗体及其应用 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| US20230346979A1 (en) * | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| WO2023202637A1 (en) | 2022-04-19 | 2023-10-26 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating neurodegenerative disorders |
| KR20250131772A (ko) * | 2022-11-13 | 2025-09-03 | 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 | 전두측두엽 치매에 대한 유전자 요법 |
| EP4669361A2 (en) * | 2023-02-24 | 2025-12-31 | The Board of Trustees of the Leland Stanford Junior University | BMP SYNTHASE ACTIVATORS AND INHIBITORS |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6538119B2 (en) | 1997-11-18 | 2003-03-25 | Abbott Laboratories | Reagents and methods useful for detecting diseases of the breast |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2001290984A1 (en) | 2000-09-18 | 2002-04-02 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| AU2002359284A1 (en) * | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| EP2687223B2 (en) | 2006-05-30 | 2024-11-20 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| CA2653974A1 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2007146046A2 (en) | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| WO2009079399A2 (en) | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| WO2009120978A2 (en) | 2008-03-27 | 2009-10-01 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
| JP2016503405A (ja) | 2012-11-05 | 2016-02-04 | ジェンザイム・コーポレーション | タンパク質症を処置するための組成物および方法 |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| WO2017151884A1 (en) | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| WO2017178826A1 (en) * | 2016-04-14 | 2017-10-19 | The University Of Liverpool | Diagnosing acute bacterial meningitis |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| MX2020004830A (es) * | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CA3136117A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20230346979A1 (en) | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
-
2020
- 2020-04-10 PL PL20724262.9T patent/PL3953377T3/pl unknown
- 2020-04-10 US US16/846,065 patent/US11999974B2/en active Active
- 2020-04-10 EP EP20724262.9A patent/EP3953377B1/en active Active
- 2020-04-10 LT LTEPPCT/US2020/027764T patent/LT3953377T/lt unknown
- 2020-04-10 PT PT207242629T patent/PT3953377T/pt unknown
- 2020-04-10 BR BR112021020183A patent/BR112021020183A2/pt unknown
- 2020-04-10 HR HRP20251506TT patent/HRP20251506T1/hr unknown
- 2020-04-10 CA CA3136004A patent/CA3136004A1/en active Pending
- 2020-04-10 FI FIEP20724262.9T patent/FI3953377T3/fi active
- 2020-04-10 KR KR1020217036234A patent/KR20210150486A/ko active Pending
- 2020-04-10 CN CN202080041413.8A patent/CN114174324A/zh active Pending
- 2020-04-10 RS RS20251192A patent/RS67476B1/sr unknown
- 2020-04-10 WO PCT/US2020/027764 patent/WO2020210698A1/en not_active Ceased
- 2020-04-10 JP JP2021559727A patent/JP7571044B2/ja active Active
- 2020-04-10 EP EP25202531.7A patent/EP4707400A2/en active Pending
- 2020-04-10 DK DK20724262.9T patent/DK3953377T3/da active
- 2020-04-10 AU AU2020273182A patent/AU2020273182B2/en active Active
- 2020-04-10 MX MX2021011958A patent/MX2021011958A/es unknown
- 2020-04-10 ES ES20724262T patent/ES3055985T3/es active Active
- 2020-04-10 SI SI202030683T patent/SI3953377T1/sl unknown
-
2021
- 2021-10-03 IL IL286903A patent/IL286903A/en unknown
-
2024
- 2024-04-26 US US18/646,957 patent/US12516298B2/en active Active
- 2024-10-09 JP JP2024176952A patent/JP2025016496A/ja active Pending
-
2026
- 2026-02-17 AU AU2026201184A patent/AU2026201184A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL286903A (en) | 2021-10-31 |
| KR20210150486A (ko) | 2021-12-10 |
| RS67476B1 (sr) | 2025-12-31 |
| CA3136004A1 (en) | 2020-10-15 |
| AU2026201184A1 (en) | 2026-03-26 |
| PT3953377T (pt) | 2025-11-27 |
| CN114174324A (zh) | 2022-03-11 |
| EP4707400A2 (en) | 2026-03-11 |
| JP2022526823A (ja) | 2022-05-26 |
| US20240287471A1 (en) | 2024-08-29 |
| AU2020273182B2 (en) | 2025-11-20 |
| EP3953377A1 (en) | 2022-02-16 |
| JP7571044B2 (ja) | 2024-10-22 |
| PL3953377T3 (pl) | 2026-02-16 |
| US11999974B2 (en) | 2024-06-04 |
| SI3953377T1 (sl) | 2026-01-30 |
| BR112021020183A2 (pt) | 2022-03-03 |
| US12516298B2 (en) | 2026-01-06 |
| FI3953377T3 (fi) | 2025-11-26 |
| EP3953377B1 (en) | 2025-09-17 |
| ES3055985T3 (en) | 2026-02-17 |
| WO2020210698A1 (en) | 2020-10-15 |
| AU2020273182A1 (en) | 2021-12-02 |
| US20200332265A1 (en) | 2020-10-22 |
| JP2025016496A (ja) | 2025-02-04 |
| MX2021011958A (es) | 2021-12-15 |
| DK3953377T3 (da) | 2025-12-01 |
| LT3953377T (lt) | 2025-12-10 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HRP20251506T1 (hr) | Genska terapija za lizosomske poremećaje | |
| ES3052719T3 (en) | Polynucleotides and vectors for the expression of transgenes | |
| JP2022060228A5 (https=) | ||
| ES3050313T3 (en) | Liver-specific tropism of adeno-associated viruses | |
| CN109715650B (zh) | 新颖腺相关病毒衣壳蛋白 | |
| JP2022078208A5 (https=) | ||
| US8067014B2 (en) | Chimeric AAV capsid proteins | |
| CN101511373B (zh) | 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体 | |
| JP2020537544A5 (https=) | ||
| JP2020537542A5 (https=) | ||
| AU2017257169B2 (en) | Evasion of neutralizing antibodies by a recombinant adeno-associated virus | |
| JP2020537543A5 (https=) | ||
| CN112424217A (zh) | 肝脏靶向的腺病毒相关病毒载体 | |
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| US20230049066A1 (en) | Novel aav3b variants that target human hepatocytes in the liver of humanized mice | |
| US20240350671A1 (en) | Engineered itr sequences and methods of use | |
| US20220243201A1 (en) | Engineered itr sequences and methods of use | |
| JPWO2021067448A5 (https=) | ||
| JPWO2019222136A5 (https=) | ||
| JPWO2022076803A5 (https=) | ||
| KR20250040030A (ko) | 아데노 관련 바이러스 역가를 향상시킬 수 있는 아데노 관련 바이러스 구조 플라스미드 | |
| CN117836312A (zh) | 分离的修饰的aav9衣壳蛋白vp1 | |
| WO2022268110A1 (zh) | 用于治疗b型血友病的组合物和方法 | |
| JP2025508988A (ja) | 副甲状腺ホルモン融合物をコードするウイルスベクター及び副甲状腺機能低下症の治療におけるその使用 | |
| Li et al. | 110. Mechanistic Study of Recombinant Adeno-Associated Virus (AAV) DNA End-Joining |