MX2023000995A - Vector de genoterapia para eef1a2 y usos del mismo. - Google Patents
Vector de genoterapia para eef1a2 y usos del mismo.Info
- Publication number
- MX2023000995A MX2023000995A MX2023000995A MX2023000995A MX2023000995A MX 2023000995 A MX2023000995 A MX 2023000995A MX 2023000995 A MX2023000995 A MX 2023000995A MX 2023000995 A MX2023000995 A MX 2023000995A MX 2023000995 A MX2023000995 A MX 2023000995A
- Authority
- MX
- Mexico
- Prior art keywords
- eef1a2
- gene therapy
- raav
- virion
- therapy vector
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 3
- 210000002845 virion Anatomy 0.000 abstract 3
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 208000012902 Nervous system disease Diseases 0.000 abstract 1
- 208000025966 Neurological disease Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 210000002569 neuron Anatomy 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
-
- 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
- A61K48/0041—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 the non-active part being polymeric
-
- 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
- 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
- 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
- C07K14/575—Hormones
- C07K14/61—Growth hormone [GH], i.e. somatotropin
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Endocrinology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063055775P | 2020-07-23 | 2020-07-23 | |
| PCT/EP2021/070455 WO2022018171A1 (en) | 2020-07-23 | 2021-07-21 | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023000995A true MX2023000995A (es) | 2023-06-16 |
Family
ID=71786970
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023000995A MX2023000995A (es) | 2020-07-23 | 2021-07-21 | Vector de genoterapia para eef1a2 y usos del mismo. |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230330265A1 (enExample) |
| EP (1) | EP4185332A1 (enExample) |
| JP (1) | JP2023535576A (enExample) |
| KR (1) | KR20230039669A (enExample) |
| CN (1) | CN116171325A (enExample) |
| AU (1) | AU2021312320A1 (enExample) |
| CA (1) | CA3186700A1 (enExample) |
| IL (1) | IL299955A (enExample) |
| MX (1) | MX2023000995A (enExample) |
| WO (2) | WO2022017630A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3445406T3 (pl) | 2016-04-20 | 2022-02-07 | Centro De Investigaciones Energéticas, Medioambientales Y Tecnológicas, O.A., M.P. | Kompozycje i sposoby zwiększonej ekspresji genów PKLR |
| 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 |
| RU2020134965A (ru) | 2018-04-27 | 2022-04-27 | Спейскрафт Севен, Ллк | Генная терапия при дегенерации цнс |
| IL300263A (en) | 2020-08-07 | 2023-03-01 | Spacecraft Seven Llc | Plakophilin-2 (PKP2) gene therapy using an AAV vector |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE386131T1 (de) | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | 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 | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| ATE317916T1 (de) | 2001-11-13 | 2006-03-15 | Univ Pennsylvania | Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode |
| US7122350B2 (en) * | 2003-12-12 | 2006-10-17 | Zymogenetics, Inc. | Methods for enhancing expression of recombinant proteins |
| US8299215B2 (en) | 2007-07-14 | 2012-10-30 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| WO2011123842A2 (en) | 2010-04-02 | 2011-10-06 | Ceregene, Inc. | Methods for treating parkinson's disease and other disorders of dopaminergic neurons of the brain |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| EP3561062A1 (en) | 2013-09-13 | 2019-10-30 | California Institute of Technology | Selective recovery |
| AU2015252797C1 (en) | 2014-05-02 | 2021-04-22 | Genzyme Corporation | AAV vectors for retinal and CNS gene therapy |
| JP2018531926A (ja) * | 2015-09-17 | 2018-11-01 | コーダ バイオセラピューティクス， インコーポレイテッド | 神経学的障害を処置するための組成物および方法 |
| ES2869284T3 (es) | 2015-12-11 | 2021-10-25 | California Inst Of Techn | Péptidos de diana para dirigir virus adenoasociados (AAV) |
| AU2019299970B2 (en) * | 2018-07-12 | 2025-12-11 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of Danon disease |
-
2020
- 2020-07-24 WO PCT/EP2020/071028 patent/WO2022017630A1/en not_active Ceased
-
2021
- 2021-07-21 MX MX2023000995A patent/MX2023000995A/es unknown
- 2021-07-21 JP JP2023504517A patent/JP2023535576A/ja active Pending
- 2021-07-21 KR KR1020237003445A patent/KR20230039669A/ko active Pending
- 2021-07-21 CN CN202180056421.4A patent/CN116171325A/zh active Pending
- 2021-07-21 US US18/017,502 patent/US20230330265A1/en active Pending
- 2021-07-21 IL IL299955A patent/IL299955A/en unknown
- 2021-07-21 CA CA3186700A patent/CA3186700A1/en active Pending
- 2021-07-21 EP EP21755896.4A patent/EP4185332A1/en active Pending
- 2021-07-21 AU AU2021312320A patent/AU2021312320A1/en active Pending
- 2021-07-21 WO PCT/EP2021/070455 patent/WO2022018171A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| CA3186700A1 (en) | 2022-01-27 |
| WO2022017630A1 (en) | 2022-01-27 |
| WO2022018171A1 (en) | 2022-01-27 |
| EP4185332A1 (en) | 2023-05-31 |
| JP2023535576A (ja) | 2023-08-18 |
| KR20230039669A (ko) | 2023-03-21 |
| IL299955A (en) | 2023-03-01 |
| CN116171325A (zh) | 2023-05-26 |
| US20230330265A1 (en) | 2023-10-19 |
| AU2021312320A1 (en) | 2023-03-16 |
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