CO2024006328A2 - Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd) - Google Patents
Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd)Info
- Publication number
- CO2024006328A2 CO2024006328A2 CONC2024/0006328A CO2024006328A CO2024006328A2 CO 2024006328 A2 CO2024006328 A2 CO 2024006328A2 CO 2024006328 A CO2024006328 A CO 2024006328A CO 2024006328 A2 CO2024006328 A2 CO 2024006328A2
- Authority
- CO
- Colombia
- Prior art keywords
- cdd
- raav
- treatment
- compositions useful
- deficiency disorder
- Prior art date
Links
Classifications
-
- 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
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- A61K48/005—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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in 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
- A61K48/0075—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
-
- 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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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)
-
- 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)
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/60—Vectors containing traps for, e.g. exons, promoters
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163256827P | 2021-10-18 | 2021-10-18 | |
| PCT/US2022/078327 WO2023069967A2 (en) | 2021-10-18 | 2022-10-18 | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2024006328A2 true CO2024006328A2 (es) | 2024-07-18 |
Family
ID=86058644
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2024/0006328A CO2024006328A2 (es) | 2021-10-18 | 2024-05-17 | Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd) |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240425882A1 (https=) |
| EP (1) | EP4419690A4 (https=) |
| JP (1) | JP2024542950A (https=) |
| KR (1) | KR20240100490A (https=) |
| CN (1) | CN118401667A (https=) |
| AU (1) | AU2022369293A1 (https=) |
| CA (1) | CA3235593A1 (https=) |
| CO (1) | CO2024006328A2 (https=) |
| IL (1) | IL312241A (https=) |
| MX (1) | MX2024004723A (https=) |
| WO (1) | WO2023069967A2 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20230003569A (ko) * | 2020-04-27 | 2023-01-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물 |
| WO2025050032A1 (en) * | 2023-08-31 | 2025-03-06 | Ultragenyx Pharmaceutical Inc. | Methods of producing cdkl5 proteins and uses of the same |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE070436T2 (hu) * | 2017-10-23 | 2025-06-28 | Stoke Therapeutics Inc | Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CA3133889A1 (en) * | 2019-04-24 | 2020-10-29 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| WO2021081217A1 (en) * | 2019-10-23 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20230043046A1 (en) * | 2019-10-30 | 2023-02-09 | Amicus Therapeutics, Inc. | Recombinant CDKL5 Proteins, Gene Therapy and Production Methods |
| KR20230003569A (ko) * | 2020-04-27 | 2023-01-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물 |
-
2022
- 2022-10-18 MX MX2024004723A patent/MX2024004723A/es unknown
- 2022-10-18 CN CN202280083857.7A patent/CN118401667A/zh active Pending
- 2022-10-18 KR KR1020247014950A patent/KR20240100490A/ko active Pending
- 2022-10-18 IL IL312241A patent/IL312241A/en unknown
- 2022-10-18 WO PCT/US2022/078327 patent/WO2023069967A2/en not_active Ceased
- 2022-10-18 CA CA3235593A patent/CA3235593A1/en active Pending
- 2022-10-18 EP EP22884659.8A patent/EP4419690A4/en active Pending
- 2022-10-18 AU AU2022369293A patent/AU2022369293A1/en active Pending
- 2022-10-18 JP JP2024523457A patent/JP2024542950A/ja active Pending
- 2022-10-18 US US18/702,258 patent/US20240425882A1/en active Pending
-
2024
- 2024-05-17 CO CONC2024/0006328A patent/CO2024006328A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4419690A4 (en) | 2026-01-21 |
| US20240425882A1 (en) | 2024-12-26 |
| AU2022369293A1 (en) | 2024-05-16 |
| WO2023069967A3 (en) | 2023-11-02 |
| CN118401667A (zh) | 2024-07-26 |
| WO2023069967A9 (en) | 2023-08-24 |
| IL312241A (en) | 2024-06-01 |
| MX2024004723A (es) | 2024-07-19 |
| KR20240100490A (ko) | 2024-07-01 |
| EP4419690A2 (en) | 2024-08-28 |
| CA3235593A1 (en) | 2023-04-27 |
| WO2023069967A8 (en) | 2024-05-16 |
| JP2024542950A (ja) | 2024-11-19 |
| WO2023069967A2 (en) | 2023-04-27 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CO2021016323A2 (es) | Composiciones útiles en el tratamiento de leucodistrofia metacromática | |
| CO2024006328A2 (es) | Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd) | |
| MX2020001593A (es) | Moleculas de acido nucleico y usos de las mismas. | |
| EA202091712A1 (ru) | МОДИФИЦИРОВАННЫЙ КАПСИДНЫЙ БЕЛОК rAAV ДЛЯ ГЕННОЙ ТЕРАПИИ | |
| CO2019009525A2 (es) | Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este | |
| CO2020004738A2 (es) | Variantes de cápsides de virus adenoasociados y métodos de uso de estas | |
| CO2022016956A2 (es) | Composiciones útiles en el tratamiento del trastorno de deficiencia de cdkl5 (cdd) | |
| CL2018003196A1 (es) | Cápsides variantes de virus adenoasociados y métodos de uso de estas. | |
| BR112019000015A2 (pt) | distribuição por vírus oncolítico pseudotipado de polipeptídeos terapêuticos | |
| CO2021011040A2 (es) | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio en adultos asociada a grn | |
| BR112021020957A2 (pt) | Capsídeos de vírus adenoassociado (aav) variante para liberação intravítrea | |
| CO2020002283A2 (es) | Métodos de terapia génica del factor viii (fviii) | |
| MX2024007432A (es) | Virus oncoliticos y metodo | |
| MX2022011394A (es) | Composiciones y metodos para la induccion de una respuesta inmune. | |
| MX2022000551A (es) | Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica. | |
| WO2020219766A8 (en) | Compositions useful in treatment of rett syndrome | |
| CL2025002970A1 (es) | Composiciones y métodos para el tratamiento de trastornos relacionados con la deficiencia de cdkl5 | |
| EA202091194A1 (ru) | Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки | |
| BR112023023249A2 (pt) | Fator neurotrófico de codificação de vírion aav e usos do mesmo | |
| AR126839A1 (es) | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta | |
| AR122378A1 (es) | Terapia génica para el tratamiento del trastorno por deficiencia de cdkl5 | |
| AR132580A1 (es) | Composiciones y métodos para el tratamiento de trastornos relacionados con la deficiencia de cdkl5 | |
| AR134214A1 (es) | Redireccionamiento de cápsides de aav para dirigirlas al sistema nervioso central | |
| AR112362A1 (es) | Polinucleótidos de aadc (l-aminoácido aromático descarboxilasa) para el tratamiento de la enfermedad de parkinson | |
| AR133298A1 (es) | Arn guía manipulados por ingeniería o polinucleótidos dirigidos a dux4 |