MX2022009982A - Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. - Google Patents
Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.Info
- Publication number
- MX2022009982A MX2022009982A MX2022009982A MX2022009982A MX2022009982A MX 2022009982 A MX2022009982 A MX 2022009982A MX 2022009982 A MX2022009982 A MX 2022009982A MX 2022009982 A MX2022009982 A MX 2022009982A MX 2022009982 A MX2022009982 A MX 2022009982A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- deficiency disorder
- cdkl5 deficiency
- treating
- raav
- Prior art date
Links
Classifications
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- 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
- A61K48/005—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
-
- 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
- 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
- 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
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
- C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- 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
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11022—Cyclin-dependent kinase (2.7.11.22)
Abstract
Esta presente descripción provee vectores virales adeno-asociados, virus recombinantes adeno-asociados (rAAV) y métodos para usarse en terapia génica para tratamiento de trastorno por deficiencia de CDKL5(CDD); también se proporcionan composiciones farmacéuticas que comprenden un rAAV de la invención y un portador o excipiente farmacéuticamente aceptable; estas composiciones farmacéuticas pueden ser útiles en terapia génica para el tratamiento de CDD causado por mutaciones en CDKL.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062976483P | 2020-02-14 | 2020-02-14 | |
US202063090492P | 2020-10-12 | 2020-10-12 | |
PCT/US2021/017656 WO2021163322A1 (en) | 2020-02-14 | 2021-02-11 | Gene therapy for treating cdkl5 deficiency disorder |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022009982A true MX2022009982A (es) | 2022-09-12 |
Family
ID=74873804
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022009982A MX2022009982A (es) | 2020-02-14 | 2021-02-11 | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
Country Status (12)
Country | Link |
---|---|
US (1) | US20230054144A1 (es) |
EP (1) | EP4103724A1 (es) |
JP (1) | JP2023514204A (es) |
KR (1) | KR20220140537A (es) |
CN (1) | CN115151648A (es) |
AU (1) | AU2021218703A1 (es) |
BR (1) | BR112022015921A2 (es) |
CA (1) | CA3164714A1 (es) |
IL (1) | IL294781A (es) |
MX (1) | MX2022009982A (es) |
TW (1) | TW202144577A (es) |
WO (1) | WO2021163322A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023023590A1 (en) * | 2021-08-18 | 2023-02-23 | Ultragenyx Pharmaceutical Inc. | Recombinant aav formulations |
EP4349365A1 (en) * | 2022-10-07 | 2024-04-10 | Hansa Biopharma AB | Co-treatment for gene therapy |
CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
CN116377050A (zh) * | 2022-12-16 | 2023-07-04 | 湖南家辉生物技术有限公司 | 一种发育性癫痫性脑病2型致病基因cdkl5突变位点的应用及其检测试剂和应用 |
Family Cites Families (39)
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GB2140408B (en) | 1982-12-23 | 1987-03-18 | Suwa Seikosha Kk | Process for producing quartz glass |
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 |
PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
PT1625210E (pt) | 2003-05-21 | 2011-03-15 | Genzyme Corp | Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios |
DK3235827T3 (da) | 2003-06-19 | 2021-04-19 | Genzyme Corp | Aav-virioner med reduceret immunreaktivitet og anvendelser deraf |
US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
ES2648241T3 (es) | 2003-09-30 | 2017-12-29 | The Trustees Of The University Of Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
PL2191001T3 (pl) | 2007-04-09 | 2017-01-31 | University Of Florida Research Foundation, Inc. | Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania |
ES2751999T3 (es) | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
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-
2021
- 2021-02-11 BR BR112022015921A patent/BR112022015921A2/pt unknown
- 2021-02-11 US US17/796,064 patent/US20230054144A1/en active Pending
- 2021-02-11 MX MX2022009982A patent/MX2022009982A/es unknown
- 2021-02-11 IL IL294781A patent/IL294781A/en unknown
- 2021-02-11 AU AU2021218703A patent/AU2021218703A1/en active Pending
- 2021-02-11 CA CA3164714A patent/CA3164714A1/en active Pending
- 2021-02-11 EP EP21711979.1A patent/EP4103724A1/en active Pending
- 2021-02-11 CN CN202180013079.XA patent/CN115151648A/zh active Pending
- 2021-02-11 KR KR1020227029963A patent/KR20220140537A/ko unknown
- 2021-02-11 WO PCT/US2021/017656 patent/WO2021163322A1/en unknown
- 2021-02-11 JP JP2022548652A patent/JP2023514204A/ja active Pending
- 2021-02-17 TW TW110105391A patent/TW202144577A/zh unknown
Also Published As
Publication number | Publication date |
---|---|
IL294781A (en) | 2022-09-01 |
CN115151648A (zh) | 2022-10-04 |
CA3164714A1 (en) | 2021-08-19 |
TW202144577A (zh) | 2021-12-01 |
BR112022015921A2 (pt) | 2022-10-04 |
WO2021163322A1 (en) | 2021-08-19 |
KR20220140537A (ko) | 2022-10-18 |
AU2021218703A1 (en) | 2022-07-28 |
EP4103724A1 (en) | 2022-12-21 |
JP2023514204A (ja) | 2023-04-05 |
US20230054144A1 (en) | 2023-02-23 |
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