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 (https=) |
| EP (1) | EP4185332A1 (https=) |
| JP (1) | JP2023535576A (https=) |
| KR (1) | KR20230039669A (https=) |
| CN (1) | CN116171325A (https=) |
| AU (1) | AU2021312320A1 (https=) |
| CA (1) | CA3186700A1 (https=) |
| IL (1) | IL299955A (https=) |
| MX (1) | MX2023000995A (https=) |
| WO (2) | WO2022017630A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3021572A1 (en) | 2016-04-20 | 2017-10-26 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas | Compositions and methods for enhanced gene expression of pklr |
| EP3697452A4 (en) | 2017-10-16 | 2021-11-24 | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. | LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS |
| BR112020020841A2 (pt) | 2018-04-11 | 2021-01-19 | Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus | Composições e métodos para transplante de célulastronco |
| CA3096293A1 (en) | 2018-04-27 | 2019-10-31 | Rocket Pharmaceuticals, Ltd. | Gene therapy for cns degeneration |
| JP2023538519A (ja) | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| WO2026006785A2 (en) * | 2024-06-28 | 2026-01-02 | Adverum Biotechnologies, Inc. | Melanopsin variants for vision restoration |
| WO2026044087A1 (en) | 2024-08-21 | 2026-02-26 | Shape Therapeutics Inc. | Increased cellular stability for aav production |
| WO2026073032A1 (en) | 2024-09-27 | 2026-04-02 | Shape Therapeutics Inc. | Constructs for improved aav production |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| US7122350B2 (en) * | 2003-12-12 | 2006-10-17 | Zymogenetics, Inc. | Methods for enhancing expression of recombinant proteins |
| WO2009012176A2 (en) | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| JP2013523776A (ja) | 2010-04-02 | 2013-06-17 | セレジーン インコーポレイテッド | パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法 |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| AU2016324317A1 (en) * | 2015-09-17 | 2018-03-08 | Coda Biotherapeutics, Inc. | Compositions and methods for treating neurological disorders |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| WO2020014523A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| CN113227126A (zh) * | 2018-11-26 | 2021-08-06 | 巴塞罗那自治大学 | 成纤维细胞生长因子21(fgf21)基因治疗 |
-
2020
- 2020-07-24 WO PCT/EP2020/071028 patent/WO2022017630A1/en not_active Ceased
-
2021
- 2021-07-21 KR KR1020237003445A patent/KR20230039669A/ko active Pending
- 2021-07-21 MX MX2023000995A patent/MX2023000995A/es unknown
- 2021-07-21 EP EP21755896.4A patent/EP4185332A1/en active Pending
- 2021-07-21 IL IL299955A patent/IL299955A/en unknown
- 2021-07-21 CN CN202180056421.4A patent/CN116171325A/zh active Pending
- 2021-07-21 WO PCT/EP2021/070455 patent/WO2022018171A1/en not_active Ceased
- 2021-07-21 AU AU2021312320A patent/AU2021312320A1/en active Pending
- 2021-07-21 JP JP2023504517A patent/JP2023535576A/ja active Pending
- 2021-07-21 CA CA3186700A patent/CA3186700A1/en active Pending
- 2021-07-21 US US18/017,502 patent/US20230330265A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022017630A1 (en) | 2022-01-27 |
| WO2022018171A1 (en) | 2022-01-27 |
| AU2021312320A1 (en) | 2023-03-16 |
| IL299955A (en) | 2023-03-01 |
| CA3186700A1 (en) | 2022-01-27 |
| JP2023535576A (ja) | 2023-08-18 |
| EP4185332A1 (en) | 2023-05-31 |
| US20230330265A1 (en) | 2023-10-19 |
| CN116171325A (zh) | 2023-05-26 |
| KR20230039669A (ko) | 2023-03-21 |
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