MX2024004119A - Composiciones y metodos para el tratamiento de enfermedades de repeticion cag - Google Patents
Composiciones y metodos para el tratamiento de enfermedades de repeticion cagInfo
- Publication number
- MX2024004119A MX2024004119A MX2024004119A MX2024004119A MX2024004119A MX 2024004119 A MX2024004119 A MX 2024004119A MX 2024004119 A MX2024004119 A MX 2024004119A MX 2024004119 A MX2024004119 A MX 2024004119A MX 2024004119 A MX2024004119 A MX 2024004119A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- methods
- diseases
- treatment
- cag repetitive
- Prior art date
Links
Classifications
-
- 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
- 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
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Psychology (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163253070P | 2021-10-06 | 2021-10-06 | |
| US202263339363P | 2022-05-06 | 2022-05-06 | |
| PCT/US2022/077572 WO2023060104A1 (en) | 2021-10-06 | 2022-10-05 | Compositions and methods for treating cag repeat diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2024004119A true MX2024004119A (es) | 2024-12-06 |
Family
ID=85804745
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2024004119A MX2024004119A (es) | 2021-10-06 | 2024-04-03 | Composiciones y metodos para el tratamiento de enfermedades de repeticion cag |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20240425856A1 (enExample) |
| EP (1) | EP4413137A4 (enExample) |
| JP (1) | JP2024543782A (enExample) |
| AU (1) | AU2022358732A1 (enExample) |
| CA (1) | CA3232737A1 (enExample) |
| IL (1) | IL311638A (enExample) |
| MX (1) | MX2024004119A (enExample) |
| TW (1) | TW202329985A (enExample) |
| WO (1) | WO2023060104A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2026515707A (ja) * | 2023-04-05 | 2026-05-19 | アイリス メディスン, インコーポレイテッド | Cagリピート疾患を治療するための組成物及び方法 |
| US20240360443A1 (en) * | 2023-04-09 | 2024-10-31 | Dystrogen Gene Therapies Inc. | Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats |
| WO2025050048A2 (en) * | 2023-08-31 | 2025-03-06 | Iris Medicine, Inc. | Methods and compositions for treating ggggcc repeat expansion diseases |
| WO2025137623A1 (en) * | 2023-12-22 | 2025-06-26 | Iris Medicine, Inc. | Methods and compositions for treating ctg repeat expansion diseases |
| WO2025213123A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL243776B1 (pl) * | 2013-09-02 | 2023-10-09 | Inst Chemii Bioorganicznej Polskiej Akademii Nauk | Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG |
| AU2016335032B2 (en) * | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
| US11613754B2 (en) * | 2017-02-20 | 2023-03-28 | Northwestern University | Toxic RNAi active seed sequences for killing cancer cells |
-
2022
- 2022-10-05 IL IL311638A patent/IL311638A/en unknown
- 2022-10-05 US US18/699,523 patent/US20240425856A1/en active Pending
- 2022-10-05 WO PCT/US2022/077572 patent/WO2023060104A1/en not_active Ceased
- 2022-10-05 AU AU2022358732A patent/AU2022358732A1/en active Pending
- 2022-10-05 CA CA3232737A patent/CA3232737A1/en active Pending
- 2022-10-05 EP EP22879440.0A patent/EP4413137A4/en active Pending
- 2022-10-05 JP JP2024521180A patent/JP2024543782A/ja active Pending
- 2022-10-05 TW TW111137904A patent/TW202329985A/zh unknown
-
2024
- 2024-04-03 MX MX2024004119A patent/MX2024004119A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2022358732A1 (en) | 2024-04-11 |
| TW202329985A (zh) | 2023-08-01 |
| EP4413137A1 (en) | 2024-08-14 |
| EP4413137A4 (en) | 2026-02-11 |
| JP2024543782A (ja) | 2024-11-26 |
| CA3232737A1 (en) | 2023-04-13 |
| IL311638A (en) | 2024-05-01 |
| WO2023060104A1 (en) | 2023-04-13 |
| US20240425856A1 (en) | 2024-12-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CL2021001042A1 (es) | Nuevos compuestos antihelmínticos | |
| CO2020015758A2 (es) | Compuestos de purinona y su uso en el tratamiento del cáncer | |
| CO2022001094A2 (es) | Pirrolo[2,3-b]pirazinas como inhibidores de hpk1 y uso de las mismas | |
| DOP2019000005A (es) | 3-metil pirazinas 2,5-disustituidas y 3-metil pirazinas 2,5,6-trisustituidas como inhibidores alostéricos de shp2 | |
| CO2019007810A2 (es) | Compuestos de amino-triazolopiridina y su uso en el tratamiento del cáncer | |
| MX379543B (es) | Purinonas como inhibidores de proteasa específica de ubiquitina 1. | |
| CO2017011851A2 (es) | Compuestos novedosos | |
| CO2021002977A2 (es) | Dimetilaminoazetidinaminas como inhibidores de jak | |
| CO2017004525A2 (es) | Dihidropirrolopiridinas inhibidoras de ror-gamma | |
| CO6630139A2 (es) | Aptámeros par b-bgf y su uso en el tratamiento de enfermedades y transtornos mediados por b-ngf | |
| CO6751275A2 (es) | Triterpenoides c-17 y c-3 modificados con la actividad inhibidora de la maduración del virus de inmunodeficiencia humana | |
| CO2021007908A2 (es) | Derivados de oxopiridina sustituidos | |
| CL2023003518A1 (es) | Nuevas sales y formas cristalinas de sales de n,n-dimetiltriptamina | |
| CO2020014677A2 (es) | Compuestos tetrahidro-1h-pirazino[2,1-a]isoindolilquinolina para el tratamiento de enfermedades autoinmunes | |
| UA120927C2 (uk) | Тритерпеноїди, заміщені в положенні 3 неароматичним кільцем, яке несе галогеналкільний замісник, з інгібуючою активністю щодо дозрівання віл | |
| CO2019011227A2 (es) | N-ariletil-2-aminoquinolin-4-carboxamidas sustituidas y su uso | |
| ECSP099807A (es) | (oxazolidinon-5-il-metil)-2-tiofen-carboxamidas sustituidas y su uso en el campo de la coagulación sanguínea | |
| MX2022003398A (es) | Composicion micelar inmunoestimulante. | |
| ECSP22083772A (es) | Compuestos y composiciones para inhibir la actividad de hif2-alfa y sus métodos de uso | |
| DOP2019000221A (es) | Derivados de pirazol como inhibidores de bromodominio | |
| MX2024005777A (es) | Isoxazolidinas como inhibidores de ripk1 y uso de estas. | |
| ECSP24024658A (es) | Derivados de s-alaninato sustituidos | |
| MX2021009443A (es) | Composiciones que contienen bacterias productoras de bacillaeno o preparaciones de las mismas. | |
| UY29189A1 (es) | Nonadepsipeptidos acilados ii | |
| MX2019009909A (es) | Composiciones farmaceuticas para terapia de combinacion. |