MX2021013275A - Terapia genica intratecal e intravenosa para el tratamiento de la enfermedad de batten infantil. - Google Patents
Terapia genica intratecal e intravenosa para el tratamiento de la enfermedad de batten infantil.Info
- Publication number
- MX2021013275A MX2021013275A MX2021013275A MX2021013275A MX2021013275A MX 2021013275 A MX2021013275 A MX 2021013275A MX 2021013275 A MX2021013275 A MX 2021013275A MX 2021013275 A MX2021013275 A MX 2021013275A MX 2021013275 A MX2021013275 A MX 2021013275A
- Authority
- MX
- Mexico
- Prior art keywords
- polynucleotide
- intrathecal
- disease
- treatment
- gene therapy
- Prior art date
Links
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
- A61K48/0075—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
- A61K48/005—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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- C12N15/63—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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962840360P | 2019-04-29 | 2019-04-29 | |
| PCT/US2020/030427 WO2020223322A1 (en) | 2019-04-29 | 2020-04-29 | Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021013275A true MX2021013275A (es) | 2022-03-17 |
Family
ID=73029200
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021013275A MX2021013275A (es) | 2019-04-29 | 2020-04-29 | Terapia genica intratecal e intravenosa para el tratamiento de la enfermedad de batten infantil. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220193268A1 (enExample) |
| EP (1) | EP3963081A4 (enExample) |
| JP (1) | JP7788594B2 (enExample) |
| KR (1) | KR20220046513A (enExample) |
| CN (1) | CN114269935A (enExample) |
| AU (1) | AU2020264438A1 (enExample) |
| BR (1) | BR112021021632A8 (enExample) |
| CA (1) | CA3138274A1 (enExample) |
| IL (1) | IL287608A (enExample) |
| MX (1) | MX2021013275A (enExample) |
| SG (1) | SG11202111908XA (enExample) |
| WO (1) | WO2020223322A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023303015A1 (en) * | 2022-07-06 | 2025-01-23 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus delivery of cln1 polynucleotide |
| WO2024015794A2 (en) * | 2022-07-12 | 2024-01-18 | The Research Institute At Nationwide Children's Hospital | Adeno-associated virus gene therapy products and methods |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2550359B1 (en) * | 2010-03-23 | 2017-08-09 | Intrexon Corporation | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof |
| JP6754361B2 (ja) * | 2014-12-16 | 2020-09-09 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| JP2019517274A (ja) * | 2016-06-13 | 2019-06-24 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ | 最適化されたｃｌｎ１遺伝子および発現カセットおよびそれらの使用 |
| WO2019011817A1 (en) * | 2017-07-08 | 2019-01-17 | Genethon | TREATMENT OF SPINAL AMYOTROPHY |
| US20210115453A1 (en) | 2017-08-31 | 2021-04-22 | Poseida Therapeutics, Inc. | Transposon system and methods of use |
-
2020
- 2020-04-29 CN CN202080047719.4A patent/CN114269935A/zh active Pending
- 2020-04-29 US US17/607,315 patent/US20220193268A1/en not_active Abandoned
- 2020-04-29 JP JP2021564486A patent/JP7788594B2/ja active Active
- 2020-04-29 WO PCT/US2020/030427 patent/WO2020223322A1/en not_active Ceased
- 2020-04-29 BR BR112021021632A patent/BR112021021632A8/pt not_active Application Discontinuation
- 2020-04-29 KR KR1020217038864A patent/KR20220046513A/ko not_active Withdrawn
- 2020-04-29 AU AU2020264438A patent/AU2020264438A1/en active Pending
- 2020-04-29 CA CA3138274A patent/CA3138274A1/en not_active Abandoned
- 2020-04-29 EP EP20798198.6A patent/EP3963081A4/en active Pending
- 2020-04-29 MX MX2021013275A patent/MX2021013275A/es unknown
- 2020-04-29 SG SG11202111908XA patent/SG11202111908XA/en unknown
-
2021
- 2021-10-27 IL IL287608A patent/IL287608A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| BR112021021632A8 (pt) | 2022-06-28 |
| US20220193268A1 (en) | 2022-06-23 |
| JP2022530264A (ja) | 2022-06-28 |
| CA3138274A1 (en) | 2020-11-05 |
| JP7788594B2 (ja) | 2025-12-19 |
| WO2020223322A1 (en) | 2020-11-05 |
| KR20220046513A (ko) | 2022-04-14 |
| AU2020264438A1 (en) | 2021-12-16 |
| BR112021021632A2 (enExample) | 2021-12-21 |
| IL287608A (en) | 2021-12-01 |
| CN114269935A (zh) | 2022-04-01 |
| EP3963081A1 (en) | 2022-03-09 |
| SG11202111908XA (en) | 2021-11-29 |
| EP3963081A4 (en) | 2023-07-26 |
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