MX2019005266A - Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas. - Google Patents
Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas.Info
- Publication number
- MX2019005266A MX2019005266A MX2019005266A MX2019005266A MX2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- gene transfer
- neurodegenerative diseases
- treating neurodegenerative
- transfer compositions
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title 1
- 230000004770 neurodegeneration Effects 0.000 title 1
- 208000015122 neurodegenerative disease Diseases 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 241000124008 Mammalia Species 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 2
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 210000004556 brain Anatomy 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 abstract 1
- 238000002347 injection Methods 0.000 abstract 1
- 239000007924 injection Substances 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 210000000278 spinal cord Anatomy 0.000 abstract 1
Classifications
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- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/365—Lactones
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
-
- 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
-
- 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
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/12—Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
- A61K38/13—Cyclosporins
-
- 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
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
-
- 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
- A61K48/0058—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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Mycology (AREA)
- Psychology (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662418033P | 2016-11-04 | 2016-11-04 | |
| PCT/US2017/059986 WO2018085688A1 (en) | 2016-11-04 | 2017-11-03 | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019005266A true MX2019005266A (es) | 2019-09-27 |
Family
ID=62075988
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2019005266A MX2019005266A (es) | 2016-11-04 | 2017-11-03 | Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas. |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190269797A1 (enExample) |
| EP (1) | EP3534892A4 (enExample) |
| JP (2) | JP2019537576A (enExample) |
| CN (1) | CN110198712A (enExample) |
| AU (1) | AU2017355502B2 (enExample) |
| BR (1) | BR112019009074A2 (enExample) |
| CA (1) | CA3041548A1 (enExample) |
| MX (1) | MX2019005266A (enExample) |
| WO (1) | WO2018085688A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9818239B2 (en) | 2015-08-20 | 2017-11-14 | Zendrive, Inc. | Method for smartphone-based accident detection |
| ES3065816T3 (en) | 2017-06-07 | 2026-05-08 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| EP3698340A4 (en) | 2017-10-20 | 2021-07-14 | Zendrive, Inc. | PROCEDURE AND SYSTEM FOR VEHICLE ASSOCIATED COMMUNICATION |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| SG11202103102WA (en) | 2018-09-26 | 2021-04-29 | California Inst Of Techn | Adeno-associated virus compositions for targeted gene therapy |
| IL327339A (en) * | 2018-11-14 | 2026-05-01 | Regenxbio Inc | Gene therapy for neural ceroid lipofuscinosis |
| CN113891712A (zh) * | 2019-02-01 | 2022-01-04 | 星火治疗有限公司 | 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法 |
| EP4042297A4 (en) | 2019-12-03 | 2023-11-22 | Zendrive, Inc. | METHOD AND SYSTEM FOR DETERMINING THE RISK OF A ROUTE |
| WO2021180118A1 (en) * | 2020-03-11 | 2021-09-16 | Shanghai Belief-Delivery Biomed Co., Ltd. | Novel use of aspirin compound in increasing nucleic acid expression |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003221745A1 (en) * | 2002-04-19 | 2003-11-03 | University Of Florida | rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| KR20070004637A (ko) * | 2004-01-22 | 2007-01-09 | 가부시키가이샤 디나벡크 겐큐쇼 | 바이러스 벡터의 제조방법 |
| ES2700048T3 (es) * | 2006-02-08 | 2019-02-13 | Genzyme Corp | Terapia génica par la enfermedad de Niemann-Pick tipo A |
| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| CA2832151C (en) * | 2011-03-31 | 2021-06-15 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| PT3024498T (pt) * | 2013-07-22 | 2020-03-06 | Childrens Hospital Philadelphia | Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos |
| PT3024497T (pt) * | 2013-07-26 | 2021-08-23 | Univ Iowa Res Found | Métodos e composições para o tratamento de doenças cerebrais |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| IL245703B (en) * | 2013-11-20 | 2022-07-01 | Univ Iowa Res Found | Methods and compositions for treating amyloid deposits |
| SG11201803218PA (en) * | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| BR112019023303A2 (pt) * | 2017-05-11 | 2020-06-16 | The Trustees Of The University Of Pennsylvania | Terapia de gene para lipofuscinoses ceroides neuronais |
-
2017
- 2017-11-03 AU AU2017355502A patent/AU2017355502B2/en not_active Ceased
- 2017-11-03 CN CN201780067919.4A patent/CN110198712A/zh active Pending
- 2017-11-03 MX MX2019005266A patent/MX2019005266A/es unknown
- 2017-11-03 US US16/344,298 patent/US20190269797A1/en active Pending
- 2017-11-03 WO PCT/US2017/059986 patent/WO2018085688A1/en not_active Ceased
- 2017-11-03 EP EP17867272.1A patent/EP3534892A4/en not_active Withdrawn
- 2017-11-03 BR BR112019009074-6A patent/BR112019009074A2/pt not_active IP Right Cessation
- 2017-11-03 JP JP2019522377A patent/JP2019537576A/ja active Pending
- 2017-11-03 CA CA3041548A patent/CA3041548A1/en active Pending
-
2022
- 2022-10-21 JP JP2022169285A patent/JP2023002721A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023002721A (ja) | 2023-01-10 |
| CN110198712A (zh) | 2019-09-03 |
| US20190269797A1 (en) | 2019-09-05 |
| EP3534892A4 (en) | 2020-05-27 |
| RU2019117062A3 (enExample) | 2021-03-11 |
| AU2017355502A1 (en) | 2019-05-16 |
| BR112019009074A2 (pt) | 2019-07-16 |
| EP3534892A1 (en) | 2019-09-11 |
| AU2017355502B2 (en) | 2023-08-31 |
| WO2018085688A1 (en) | 2018-05-11 |
| RU2019117062A (ru) | 2020-12-04 |
| CA3041548A1 (en) | 2018-05-11 |
| JP2019537576A (ja) | 2019-12-26 |
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