MX2021009696A - Vectores de genoterapia para el tratamiento de la enfermedad de danon. - Google Patents
Vectores de genoterapia para el tratamiento de la enfermedad de danon.Info
- Publication number
- MX2021009696A MX2021009696A MX2021009696A MX2021009696A MX2021009696A MX 2021009696 A MX2021009696 A MX 2021009696A MX 2021009696 A MX2021009696 A MX 2021009696A MX 2021009696 A MX2021009696 A MX 2021009696A MX 2021009696 A MX2021009696 A MX 2021009696A
- Authority
- MX
- Mexico
- Prior art keywords
- treatment
- gene therapy
- lamp
- therapy vectors
- danon disease
- Prior art date
Links
Classifications
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/06—Antiarrhythmics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- Toxicology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962804521P | 2019-02-12 | 2019-02-12 | |
| US201962934928P | 2019-11-13 | 2019-11-13 | |
| PCT/US2020/017987 WO2020167996A1 (en) | 2019-02-12 | 2020-02-12 | Gene therapy vectors for treatment of danon disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021009696A true MX2021009696A (es) | 2021-09-23 |
Family
ID=72043957
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021009696A MX2021009696A (es) | 2019-02-12 | 2020-02-12 | Vectores de genoterapia para el tratamiento de la enfermedad de danon. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220143215A1 (enExample) |
| EP (1) | EP3924371A4 (enExample) |
| JP (2) | JP2022520232A (enExample) |
| KR (1) | KR20210125999A (enExample) |
| CN (2) | CN120324641A (enExample) |
| AU (3) | AU2020221842A1 (enExample) |
| BR (1) | BR112021015751A2 (enExample) |
| CA (1) | CA3128514A1 (enExample) |
| IL (1) | IL285238A (enExample) |
| MX (1) | MX2021009696A (enExample) |
| SG (1) | SG11202107744SA (enExample) |
| WO (1) | WO2020167996A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102673203B1 (ko) | 2016-04-20 | 2024-06-10 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | Pklr의 유전자 발현 증강을 위한 조성물 및 방법 |
| AU2018352583B2 (en) | 2017-10-16 | 2025-04-17 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| IL278213B2 (en) | 2018-04-27 | 2026-04-01 | Rocket Pharmaceuticals Ltd | A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd) |
| EP3820537A4 (en) * | 2018-07-12 | 2022-04-13 | Spacecraft Seven, LLC | GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE |
| IL280465B2 (en) | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| WO2022032226A1 (en) * | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
| CN116887868A (zh) * | 2020-12-07 | 2023-10-13 | 太空飞船七有限责任公司 | 达农病的治疗 |
| CN114874990A (zh) * | 2021-02-05 | 2022-08-09 | 中国科学院苏州纳米技术与纳米仿生研究所 | 一种功能化外泌体及其制备方法和应用 |
| US12609410B2 (en) | 2021-09-23 | 2026-04-21 | Lg Energy Solution, Ltd. | High heat-resistant connector, and battery module, battery pack, and vehicle including the same |
| CN114457112A (zh) * | 2022-02-07 | 2022-05-10 | 苏州市立医院 | 一种特异性神经靶向mr分子探针及其制备方法和应用 |
| CN115975051B (zh) * | 2022-11-17 | 2025-09-12 | 东南大学 | 靶向外泌体及制备方法、应用、药物和药物递送系统 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003038049A2 (en) * | 2001-10-29 | 2003-05-08 | Renovis, Inc. | Method for isolating cell-type specific mrnas |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US9434928B2 (en) * | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| EP3405215B1 (en) * | 2016-01-19 | 2022-06-15 | The Regents of the University of California | Methods for the treatment of danon disease and other disorders of autophagy |
| CA3011939A1 (en) * | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| WO2019028273A1 (en) * | 2017-08-02 | 2019-02-07 | Dna2.0, Inc. Dba Atum | DNA VECTORS AND ELEMENTS FOR SUSTAINED GENE EXPRESSION IN EUKARYOTIC CELLS |
| EP3820537A4 (en) * | 2018-07-12 | 2022-04-13 | Spacecraft Seven, LLC | GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE |
| WO2020033842A1 (en) * | 2018-08-10 | 2020-02-13 | Regenxbio Inc. | Scalable method for recombinant aav production |
-
2020
- 2020-02-12 US US17/430,107 patent/US20220143215A1/en active Pending
- 2020-02-12 BR BR112021015751A patent/BR112021015751A2/pt unknown
- 2020-02-12 CN CN202510460177.8A patent/CN120324641A/zh active Pending
- 2020-02-12 KR KR1020217022811A patent/KR20210125999A/ko active Pending
- 2020-02-12 CN CN202080011825.7A patent/CN113508130A/zh active Pending
- 2020-02-12 MX MX2021009696A patent/MX2021009696A/es unknown
- 2020-02-12 WO PCT/US2020/017987 patent/WO2020167996A1/en not_active Ceased
- 2020-02-12 JP JP2021546891A patent/JP2022520232A/ja active Pending
- 2020-02-12 AU AU2020221842A patent/AU2020221842A1/en not_active Abandoned
- 2020-02-12 CA CA3128514A patent/CA3128514A1/en active Pending
- 2020-02-12 SG SG11202107744SA patent/SG11202107744SA/en unknown
- 2020-02-12 EP EP20754942.9A patent/EP3924371A4/en active Pending
-
2021
- 2021-07-29 IL IL285238A patent/IL285238A/en unknown
-
2023
- 2023-03-01 AU AU2023201237A patent/AU2023201237B2/en active Active
-
2025
- 2025-01-20 JP JP2025007633A patent/JP2025063228A/ja active Pending
- 2025-07-09 AU AU2025205318A patent/AU2025205318A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2025205318A1 (en) | 2025-07-31 |
| CA3128514A1 (en) | 2020-08-20 |
| CN120324641A (zh) | 2025-07-18 |
| AU2023201237B2 (en) | 2025-04-10 |
| US20220143215A1 (en) | 2022-05-12 |
| WO2020167996A1 (en) | 2020-08-20 |
| AU2020221842A1 (en) | 2021-08-12 |
| JP2022520232A (ja) | 2022-03-29 |
| KR20210125999A (ko) | 2021-10-19 |
| BR112021015751A2 (pt) | 2022-02-08 |
| IL285238A (en) | 2021-09-30 |
| AU2023201237A1 (en) | 2023-04-13 |
| EP3924371A1 (en) | 2021-12-22 |
| JP2025063228A (ja) | 2025-04-15 |
| EP3924371A4 (en) | 2023-01-11 |
| CN113508130A (zh) | 2021-10-15 |
| SG11202107744SA (en) | 2021-08-30 |
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